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Ensaio (s) clínico (s) nacional (is)

ILE-DE-FRANCE
PARIS
MAGMAT : Interest of Magnesium Sulfate in Thrombotic Thrombocytopenic Purpura in Intensive Care: Multicentric Randomized Controlled Trial (Phase III)
CHU Paris-GH St-Louis Lariboisière F.Widal - Hôpital Saint-Louis
Service de réanimation médicale

Baden-Württemberg
HEIDELBERG
MPN-SG 01-09 (MPN Pomalidomide): Multicenter Phase II Study with Pomalidomide in patients with Myeloproliferative Neoplasms in fibrotic Stage
Nationales Centrum für Tumorerkrankungen (NCT)
Nationales Centrum für Tumorerkrankungen (CCC) Heidelberg

Berlin
ADDRESS: NOT PROVIDED - DE

A prospective, open-label, phase I/III study investigating pharmacokinetic properties of BT524 and efficacy and safety of BT524 in the treatment and prophylaxis of bleeding in patients with congenital fibrinogen deficiency -DE-
Institution: Information not provided - DE

Hamburg
HAMBURG

Ruxolitinib versus allogeneic stem cell transplantation for patients with myelofibrosis according to donor availability: A prospective phase II trial (MMM 02 study) - DE
UKE - Universitätsklinikum Hamburg-Eppendorf
Interdisziplinäre Klinik und Poliklinik für Stammzelltransplantation

Nordrhein-Westfalen
AACHEN

The RUXO-BEAT Trial : RUXOlitinib versus BEst Available Therapy in patients with high-risk polycythemia vera or high-risk essential thrombocythemia (Phase 3) - DE
Universitätsklinikum Aachen
Klinik für Onkologie, Hämatologie und Stammzelltransplantation (Med. Klinik IV)

Nordrhein-Westfalen
ESSEN
CP14B015: A phase II trial to evaluate the activity of Imetelstat (GRN163L) in patients with Essential Thrombocythemia or Polycythemia vera who require cytoreduction and have failed or are intolerant to previous therapy, or who refuse standard therapy
Westdeutsches Tumorzentrum Essen (WTZ)
Klinik für Hämatologie

Nordrhein-Westfalen
MINDEN
JHTB(a): A Phase 2 Study of LY2784544 in Patients With Myeloproliferative Neoplasms - DE
Johannes Wesling Klinikum Minden
Klinik für Hämatologie, Onkologie und Palliativmedizin

SALZBURG
SALZBURG

An Open-Label Phase 2 Study of Itacitinib (INCB039110) in Combination With Low-Dose Ruxolitinib or Itacitinib Alone Following Ruxolitinib in Subjects With Myelofibrosis - AT
Universitätsklinikum der Paracelsus Medizinischen Privatuniversität
Universitätsklinik für Innere Medizin III

TIROL
INNSBRUCK

A Double-blind, Placebo-controlled Pilot Trial to Investigate the Administration of Von Willebrand Factor Concentrate (Willfact®, LFB France) in Adult Patients During Extracorporeal Membrane Oxygenation (Phase 2)
Medizinische Universität Innsbruck
Universitätsklinik für Anästhesie und Intensivmedizin

WIEN
ADDRESS: NOT PROVIDED - AT
A Phase Ib, open-label, dose-finding study of the JAK inhibitor INC424 tablets administered orally to patients with Primary Myelofibrosis (PMF), Post-Polycythemia Vera-Myelofibrosis (PPV-MF) or Post-Essential Thrombocythemia-Myelofibrosis (PET-MF) and baseline platelet counts > 50 x109/L and <100 x109/L - AT
Institution: Information not provided - AT

WIEN
ADDRESS: NOT PROVIDED - AT
Very low doses of Rituximab for off-label treatment - a Pilot Trial (Phase 2)
Institution: Information not provided - AT

WIEN
ADDRESS: NOT PROVIDED - AT

FREEDOM2: A Phase 3, multicenter, open-label, randomized study to evaluate the efficacy and safety of fedratinib compared to best available therapy in subjects with DIPSS - intermediate or high-risk primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis and previously treated with ruxolitinib - AT
Institution: Information not provided - AT

WIEN
ADDRESS: NOT PROVIDED - AT

MOMENTUM: A Randomized, Double-Blind, Phase 3 Study of Momelotinib vs Danazol in Symptomatic, Anemic Subjects With Previously JAKi Treated Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post Essential Thrombocythemia Myelofibrosis - AT
Institution: Information not provided - AT

WIEN
WIEN
A Phase 2 Study of LY2784544 in Patients with Myeloproliferative Neoplasms - AT
Allgemeines Krankenhaus der Stadt Wien
Klinische Abteilung für Hämatologie und Hämostaseologie

WIEN
WIEN
Post-HERCULES: Prospective Follow-up Study for Patients Who Completed Study ALX0681-C301 (HERCULES) to Evaluate Long-term Safety and Efficacy of Caplacizumab (Phase III) - AT
Allgemeines Krankenhaus der Stadt Wien
Klinische Abteilung für Hämatologie und Hämostaseologie

WIEN
WIEN

A Phase 3, Prospective, Randomized, Controlled, Open-label, Multicenter, 2-period Crossover Study With a Single Arm Continuation Evaluating the Safety and Efficacy of BAX 930 (rADAMTS13) in the Prophylactic and On-demand Treatment of Subjects With Severe Congenital Thrombotic Thrombocytopenic Purpura - AT
Allgemeines Krankenhaus der Stadt Wien
Klinische Abteilung für Hämatologie und Hämostaseologie

WIEN
WIEN

An Open-Label Phase 2 Study of Itacitinib (INCB039110) in Combination With Low-Dose Ruxolitinib or Itacitinib Alone Following Ruxolitinib in Subjects With Myelofibrosis - AT
Hanusch Krankenhaus
3. Medizinische Abteilung (Hämatologie und Onkologie)

Andalucía
MÁLAGA

An open-label, phase 2a/2b study of KRT-232 in subjects with primary myelofibrosis (PMF), post-polycythemia vera MF (Post-PV-MF), or post-essential thrombocythemia MF (Post-ET-MF) who have failed ruxolitinib - ES
Hospital Universitario Virgen de la Victoria
Unidad de Hematología

Andalucía
SEVILLA

Evaluation of the pharmacokinetic profile, clinical efficacy and safety of the Von Willebrand factor contained in Fanhdi® (double-inactivated human anti-hemophilic factor) in pediatric patients with Von Willebrand disease (Phase IV) - ES
Hospital Universitario Virgen del Rocío
Unidad de Hematología

Aragón
ZARAGOZA

Extended Access of Momelotinib for Subjects with Primary Myelofibrosis (PMF) or Post-polycythemia Vera or Post-essential Thrombocythemia Myelofibrosis (Post-PV/ET MF) (Phase II) - ES
Hospital Quirónsalud Zaragoza
Servicio de Hematología

Canarias
LAS PALMAS

An open-label, phase 2a/2b study of KRT-232 in subjects with primary myelofibrosis (PMF), post-polycythemia vera MF (Post-PV-MF), or post-essential thrombocythemia MF (Post-ET-MF) who have failed ruxolitinib - ES
Hospital Universitario de Gran Canaria Dr. Negrín
Servicio de Hematología

Castilla - León
SALAMANCA

An open-label, phase 2a/2b study of KRT-232 in subjects with primary myelofibrosis (PMF), post-polycythemia vera MF (Post-PV-MF), or post-essential thrombocythemia MF (Post-ET-MF) who have failed ruxolitinib - ES
Hospital Clínico Universitario de Salamanca
Servicio de Hematología

Cataluña
BADALONA

Extended Access of Momelotinib for Subjects with Primary Myelofibrosis (PMF) or Post-polycythemia Vera or Post-essential Thrombocythemia Myelofibrosis (Post-PV/ET MF) (Phase II) - ES
ICO Badalona - Hospital Germans Trias i Pujol
Servicio de Hematología Clínica

Cataluña
BADALONA

An open-label, phase 2a/2b study of KRT-232 in subjects with primary myelofibrosis (PMF), post-polycythemia vera MF (Post-PV-MF), or post-essential thrombocythemia MF (Post-ET-MF) who have failed ruxolitinib - ES
ICO Badalona - Hospital Germans Trias i Pujol
Servicio de Hematología Clínica

Cataluña
BARCELONA

Extended Access of Momelotinib for Subjects with Primary Myelofibrosis (PMF) or Post-polycythemia Vera or Post-essential Thrombocythemia Myelofibrosis (Post-PV/ET MF) (Phase II) - ES
Hospital Clínic de Barcelona
Servicio de Hematología

Cataluña
BARCELONA

Extended Access of Momelotinib for Subjects with Primary Myelofibrosis (PMF) or Post-polycythemia Vera or Post-essential Thrombocythemia Myelofibrosis (Post-PV/ET MF) (Phase II) - ES
Hospital Universitari Vall d'Hebron
Servicio de Hematología y Hemoterapia

Comunidad Valenciana
ALICANTE
A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF With or Without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics of rVWF in Children Diagnosed With Severe Von Willebrand Disease - ES
Hospital General Universitario de Alicante
Servicio de Hematología

Comunidad Valenciana
VALENCIA

An open-label, phase 2a/2b study of KRT-232 in subjects with primary myelofibrosis (PMF), post-polycythemia vera MF (Post-PV-MF), or post-essential thrombocythemia MF (Post-ET-MF) who have failed ruxolitinib - ES
Hospital Clínico Universitario de Valencia
Servicio de Hematología

Comunidad Valenciana
VALENCIA
A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF With or Without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics of rVWF in Children Diagnosed With Severe Von Willebrand Disease - ES
Hospital Universitario y Politécnico La Fe
Servicio de Hematología y Hemoterapia

Madrid
ADDRESS: NOT PROVIDED - ES
A Phase 2 Study of LY278544 in Patients With Myeloproliferative Neoplasms - ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES


A Phase 3, Prospective, Randomized, Controlled, Open-label, Multicenter, 2-period Crossover Study With a Single Arm Continuation Evaluating the Safety and Efficacy of BAX 930 (rADAMTS13) in the Prophylactic and On-demand Treatment of Subjects With Severe Congenital Thrombotic Thrombocytopenic Purpura - ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

FREEDOM2: A Phase 3, multicenter, open-label, randomized study to evaluate the efficacy and safety of fedratinib compared to best available therapy in subjects with DIPSS - intermediate or high-risk primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis and previously treated with ruxolitinib - ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

MOMENTUM: A Randomized, Double-Blind, Phase 3 Study of Momelotinib vs Danazol in Symptomatic, Anemic Subjects With Previously JAKi Treated Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post Essential Thrombocythemia Myelofibrosis - ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

REFINE: A Phase 2 Open-Label Study Evaluating Tolerability and Efficacy of Navitoclax Alone or in Combination with Ruxolitinib in Subjects with Myelofibrosis - ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

SOAR-HI: A Phase 2, multicenter, randomized, placebo-controlled, double-blind study in patients with acquired thrombotic thrombocytopenic purpura (aTTP) to evaluate the pharmacokinetics, safety, and efficacy of rADAMTS-13 (SHP655) administered in addition to standard of care (SoC) treatment - ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

PACIFICA Phase 3: A Randomized, Controlled Phase 3 Study of Pacritinib Versus Physician's Choice in Patients with Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post Essential Thrombocythemia Myelofibrosis with Severe Thrombocytopenia (Platelet Counts <50,000) - ES
Institution: Information not provided - ES

Madrid
MADRID

Extended Access of Momelotinib for Subjects with Primary Myelofibrosis (PMF) or Post-polycythemia Vera or Post-essential Thrombocythemia Myelofibrosis (Post-PV/ET MF) (Phase II) - ES
Hospital Universitario 12 de Octubre
Servicio de Hematología y Hemoterapia

Madrid
MADRID

An open-label, phase 2a/2b study of KRT-232 in subjects with primary myelofibrosis (PMF), post-polycythemia vera MF (Post-PV-MF), or post-essential thrombocythemia MF (Post-ET-MF) who have failed ruxolitinib - ES
Hospital Universitario Ramón y Cajal
Servicio de Hematología y Hemoterapia

Madrid
MAJADAHONDA

Extended Access of Momelotinib for Subjects with Primary Myelofibrosis (PMF) or Post-polycythemia Vera or Post-essential Thrombocythemia Myelofibrosis (Post-PV/ET MF) (Phase II) - ES
Hospital Universitario Puerta de Hierro - Majadahonda
Servicio de Hematología y Hemoterapia

Madrid
MAJADAHONDA

An open-label, phase 2a/2b study of KRT-232 in subjects with primary myelofibrosis (PMF), post-polycythemia vera MF (Post-PV-MF), or post-essential thrombocythemia MF (Post-ET-MF) who have failed ruxolitinib - ES
Hospital Universitario Puerta de Hierro - Majadahonda
Servicio de Hematología y Hemoterapia

Navarra
PAMPLONA

Extended Access of Momelotinib for Subjects with Primary Myelofibrosis (PMF) or Post-polycythemia Vera or Post-essential Thrombocythemia Myelofibrosis (Post-PV/ET MF) (Phase II) - ES
Clínica Universidad de Navarra
Servicio de Hematología y Hemoterapia

Washington
ADDRESS: NOT PROVIDED - US


A Phase 2, Double-blind, Randomized Safety And Efficacy Study Of Glasdegib (Pf-04449913) Versus Placebo In Patients With Myelofibrosis Previously Treated With Ruxolitinib - US
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
PAC203: An Open-Label, Randomized, Phase 2 Dose-Finding Study of Pacritinib in Patients with Thrombocytopenia and Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis Previously Treated with Ruxolitinib - GB
Institution: Information not provided - US

Washington
SEATTLE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
RESUME - A phase-3, multi-center, randomized, double-blind, placebo-controlled, parallel-group study to compare efficacy and safety of pomalidomide in subjects with myeloproliferative neoplasm -associated myelofibrosis and red blood cell-transfusion-dependence - FR
Institution: Information not provided - FR

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

A randomized controlled phase III study of oral Pacritinib versus best available therapy in patients with Primary Myelofibrosis, Post-Polycythemia vera Myelofibrosis, or Post-Essential thrombocythemia Myelofibrosis - FR
Institution: Information not provided - FR

ILE-DE-FRANCE
LE KREMLIN-BICÊTRE
A Multi-Centre, Open-Label, Single-Arm, and Multiple Dosing Trial on Safety of Monthly Replacement Therapy with Recombinant Factor XIII (rFXIII) in Subjects with Congenital Factor XIII Deficiency (Safety Extension Trial to F13CD-1725) (Phase III) - FR
CHU Paris-Sud - Hôpital de Bicêtre
Service d'Hématologie

County Offaly
TULLAMORE

A Phase IIIb, randomised, open label study to compare the safety, efficacy and tolerability of anagrelide hydrochloride versus hydroxyurea in high-risk essential thrombocythaemia patients - IE
Midland Regional Hospital
Department of Haematology

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
Evaluation of clinical response to rituximab treatment in patients with acquired autoimmune recurrent thrombotic thrombocytopenic purpura (TTP) - RTXTTP2010
Institution: Information not provided - IT

Greater London
LONDON

MAJIC - A RandoMised study of best Available therapy versus JAK Inhibition in patients with high risk Polycythaemia Vera or Essential Thrombocythaemia who are resistant or intolerant to HydroxyCarbamide (Phase II)
Guy's Hospital
Department of Haematology

Greater London
LONDON
A Randomized Phase II, Open-Label study of the Efficacy and Safety of Orally Administered SAR302503 in patients with polycythemia vera (PV) or essential thrombocythemia (ET) who are resistant or intolerant to hydroxyurea - UK
Guy's Hospital
Department of Haematology

Greater London
LONDON

A randomized controlled phase III study of oral Pacritinib versus best available therapy in patients with Primary Myelofibrosis, Post-Polycythemia vera Myelofibrosis, or Post-Essential thrombocythemia Myelofibrosis - UK
National Cancer Research Institute (NCRI)
Cancer Research UK

Greater London
LONDON

Forma-01: A prospective, controlled, randomised, cross-over study investigating the pharmacokinetic properties, surrogate efficacy and safety of Octafibrin compared to Haemocomplettan® P/ RiaSTAPTM in subjects with congenital fibrinogen deficiency - UK
St Thomas' Hospital
Molecular Haemostasis & Thrombosis

Greater Manchester
ADDRESS: NOT PROVIDED - UK
A UK open-label, multicentre, exploratory Phase II study of INC424 for patients with primary myelofibrosis (PMF) or post polycythemia myelofibrosis (PPV MF) or post-essential thrombocythemia myelofibrosis (PET-MF)
Institution: Information not provided - UK

South Glamorgan
CARDIFF
Extended Access of Momelotinib for Subjects With Primary Myelofibrosis (PMF) or Post-polycythemia Vera or Post-essential Thrombocythemia Myelofibrosis (Post-PV/ET MF)GB
University Hospital of Wales

West Midlands
BIRMINGHAM

TAMARIN:Effects of TAMoxifen on the Mutant Allele Burden and Disease Course in Patients with MyeloprolifeRatIve Neoplasms- GB
University of Birmingham
University of Birmingham HQ

Berlin
ADDRESS: NOT PROVIDED - DE
A 4-part Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of M254 in Healthy Volunteers and in Patients With Immune Thrombocytopenic Purpura - DE-
Institution: Information not provided - DE

Berlin
ADDRESS: NOT PROVIDED - DE

FRONTIER1: Safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple subcutaneous doses of NNC0365-3769 (Mim8) in healthy subjects and in subjects with haemophilia A with or without factor VIII inhibitors -DE-
Institution: Information not provided - DE

Berlin
BERLIN


ATLAS-A/B: A Phase 3 study to evaluate the efficacy and safety of fitusiran in patients with hemophilia A or B, without inhibitory antibodies to factor VIII or IX -DE-
Vivantes Klinikum im Friedrichshain
Zentrum für Hämophilie und Hämostaseologie

Hessen
FRANKFURT AM MAIN


ATLAS-A/B: A Phase 3 study to evaluate the efficacy and safety of fitusiran in patients with hemophilia A or B, without inhibitory antibodies to factor VIII or IX -DE-
Klinikum der Johann Wolfgang Goethe-Universität Frankfurt

Hessen
MARBURG
A Randomized, Double-blind, Placebo-controlled, Phase II Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients With Primary Immune Thrombocytopenia - DE
Universitätsklinikum Gießen und Marburg GmbH, Standort Marburg
Klinik für Dermatologie und Allergologie

Niedersachsen
HANNOVER


A prospective, phase 3, open-label, international multicenter study on efficacy and safety of prophylaxis with rVWF in severe Von Willebrand disease - DE
Medizinische Hochschule Hannover
Medizinische Hochschule Hannover (MHH)

Niedersachsen
HANNOVER

Sachsen
LEIPZIG


ATLAS-A/B: A Phase 3 study to evaluate the efficacy and safety of fitusiran in patients with hemophilia A or B, without inhibitory antibodies to factor VIII or IX -DE-
Universitätsklinikum Leipzig AöR
Klinik und Poliklinik für Hämatologie, Zelltherapie und Hämostaseologie

WIEN
ADDRESS: NOT PROVIDED - AT

A Randomized, Double-blind, Placebo-controlled, Phase II Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients With Primary Immune Thrombocytopenia - AT
Institution: Information not provided - AT

WIEN
ADDRESS: NOT PROVIDED - AT

A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF With or Without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics of rVWF in Children Diagnosed With Severe Von Willebrand Disease - AT
Institution: Information not provided - AT

WIEN
ADDRESS: NOT PROVIDED - AT

TAPER: A Phase II, Open-label, Prospective, Single-arm, Study to Assess Ability of Eltrombopag to Induce Sustained Remission in Subjects With ITP Who Are Refractory or Relapsed After First-line Steroids - AT
Institution: Information not provided - AT

WIEN
ADDRESS: NOT PROVIDED - AT
ALIFE2: Anticoagulants for living fetuses for women with recurrent miscarriage and inherited blood clotting disorders (phase 3) - AT
Institution: Information not provided - AT

WIEN
ADDRESS: NOT PROVIDED - AT
A Phase 3b, prospective, open-label, uncontrolled, multicenter study on longterm safety and efficacy of rVWF in pediatric and adult subjects with severe Von Willebrand disease - AT
Institution: Information not provided - AT

WIEN
ADDRESS: NOT PROVIDED - AT

myOpportunITy1: A Phase 3 Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP) - AT
Institution: Information not provided - AT

WIEN
ADDRESS: NOT PROVIDED - AT
ADVANCE: A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Efgartigimod (ARGX 113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia (ITP) - AT
Institution: Information not provided - AT

WIEN
ADDRESS: NOT PROVIDED - AT

myOpportunITy2: A Phase 3 Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP) - AT
Institution: Information not provided - AT

WIEN
WIEN
A Phase 2a Multiple Dose "Basket Design" Study Of The Safety, Tolerability, And Pharmacologic Activity Of BT200 In Patients With Hereditary Bleeding Disorders
Allgemeines Krankenhaus der Stadt Wien
Universitätsklinik für klinische Pharmakologie

WIEN
WIEN
A Phase 3 Open Label Extension Study of Fostamatinib Disodium in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura - AT
Hanusch Krankenhaus
3. Medizinische Abteilung (Hämatologie und Onkologie)

ARRONDISSEMENT BRUSSELS-CAPITAL
ADDRESS: NOT PROVIDED - BE
MOTIVATE: MOdern Treatment of Inhibitor-PositiVe PATiEnts with Haemophilia A - An International Observational Study - BE
Institution: Information not provided - BE

NAMUR
YVOIR
A 4-part Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of M254 in Healthy Volunteers and in Patients With Immune Thrombocytopenic Purpura - BE
Cliniques Universitaires UCL de Mont-Godinne
CHU Dinant-Godinne UCL Namur

VLAAMS BRABANT
LEUVEN

A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF With or Without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics of rVWF in Children Diagnosed With Severe Von Willebrand Disease - BE
UZ Leuven - Campus Gasthuisberg
Hemofiliecentrum

Ontario
TORONTO
A Randomized Study of IVIG vs. IVIG With High Dose Methylprednisolone in Rapidly Augmenting Platelet Counts in Childhood ITP (Phase III)
The Hospital for Sick Children and University of Toronto
Division of Haematology/Oncology

Andalucía
MÁLAGA

A 4-part Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of M254 in Healthy Volunteers and in Patients With Immune Thrombocytopenic Purpura - ES
Hospital Regional Universitario de Málaga - Hospital General
Servicio de Inmunología

Andalucía
SEVILLA

myOpportunITy1: A Phase 3 Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP) - ES
Hospital Universitario Virgen Macarena
Unidad de Hematología

Andalucía
SEVILLA

ADVANCE: A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Efgartigimod (ARGX 113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia (ITP) - ES
Hospital Universitario Virgen del Rocío

Baleares
PALMA DE MALLORCA

Castilla - León
BURGOS

ADVANCE: A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Efgartigimod (ARGX 113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia (ITP) - ES
Hospital Universitario de Burgos

Castilla - León
SALAMANCA

myOpportunITy1: A Phase 3 Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP) - ES
Hospital Clínico Universitario de Salamanca
Servicio de Hematología

Cataluña
BARCELONA

ADVANCE: A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Efgartigimod (ARGX 113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia (ITP) - ES
Hospital Universitari Vall d'Hebron

Cataluña
BARCELONA

A 4-part Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of M254 in Healthy Volunteers and in Patients With Immune Thrombocytopenic Purpura - ES
Hospital Universitari Vall d'Hebron
Servicio de Hematología y Hemoterapia

Cataluña
BARCELONA

Comunidad Valenciana
ALICANTE

A Phase 3b, prospective, open-label, uncontrolled, multicenter study on longterm safety and efficacy of rVWF in pediatric and adult subjects with severe Von Willebrand disease - ES
Hospital General Universitario de Alicante

Comunidad Valenciana
MANISES

Comunidad Valenciana
VALENCIA

A 4-part Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of M254 in Healthy Volunteers and in Patients With Immune Thrombocytopenic Purpura - ES
Hospital Universitario Doctor Peset
Servicio de Inmunología

Comunidad Valenciana
VALENCIA

A Phase 3b, prospective, open-label, uncontrolled, multicenter study on longterm safety and efficacy of rVWF in pediatric and adult subjects with severe Von Willebrand disease - ES
Hospital Universitario y Politécnico La Fe

Comunidad Valenciana
VALENCIA

ADVANCE: A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Efgartigimod (ARGX 113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia (ITP) - ES
Hospital Universitario y Politécnico La Fe

Comunidad Valenciana
VALENCIA

myOpportunITy1: A Phase 3 Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP) - ES
Hospital Universitario y Politécnico La Fe
Servicio de Hematología y Hemoterapia

Madrid
ADDRESS: NOT PROVIDED - ES
ATLAS-PPX trial: an open-label, multinational, switching study to describe the efficacy and safety of fitusiran prophylaxis in hemophilia A and B patients previously receiving factor or bypassing agent prophylaxis (Phase III) - ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES
MOTIVATE: Modern Treatment of Inhibitor - Positive Patients with haemophilia A - An International Low - Interventional Pragmatic Investigator Initiated Trial - ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

ADVANCE+: A Phase 3, Multicenter, Open-label, Long-term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX-113) 10 mg/kg Intravenous in Adult Patients with Primary Immune Thrombocytopenia - ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

myOpportunITy2: A Phase 3 Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP) - ES
Institution: Information not provided - ES

Madrid
MADRID

A Phase 3b, prospective, open-label, uncontrolled, multicenter study on longterm safety and efficacy of rVWF in pediatric and adult subjects with severe Von Willebrand disease - ES
Hospital Universitario La Paz

Madrid
MADRID

ADVANCE: A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Efgartigimod (ARGX 113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia (ITP) - ES
Hospital Universitario La Paz

Madrid
MADRID

ADVANCE: A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Efgartigimod (ARGX 113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia (ITP) - ES
Hospital Universitario Ramón y Cajal

Madrid
POZUELO DE ALARCÓN

ADVANCE: A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Efgartigimod (ARGX 113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia (ITP) - ES
Hospital Universitario Quirónsalud Madrid

ILE-DE-FRANCE
CRÉTEIL

A phase Ib open-label, dose escalation study of PRTX-100 in adult patients with persistent/chronic immune thrombocytopenia - FR -
CHU Henri Mondor
Service de Médecine interne

ILE-DE-FRANCE
CRÉTEIL

DAPS-ITP : Prospective Multicenter Randomized Open-label Controlled Trial Assessing Efficacy and Safety of DAPSone as a Second-line Option in Adult Immune Thrombocytopenia (Phase III)
CHU Henri Mondor
Service de Médecine interne

County Dublin
DUBLIN

ATLAS-PPX trial: an open-label, multinational, switching study to describe the efficacy and safety of fitusiran prophylaxis in hemophilia A and B patients previously receiving factor or bypassing agent prophylaxis (Phase III) - IE
Children's Health Ireland @ Crumlin
Haematology Department

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT

A 4-part Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics of M254 in healthy volunteers and in patients with immune thrombocytopenic purpura - IT
Institution: Information not provided - IT

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT

MOTIVATE: MOdern Treatment of Inhibitor-PositiVe PATiEnts with Haemophilia A - An International Low-Interventional Pragmatic Investigator Initiated Trial - IT
Institution: Information not provided - IT

Zuid-Holland
DEN HAAG
STIP study: Stop TPO-receptor agonist in ITP Patients
Haga Ziekenhuis, locatie Leyweg
Afdeling Hematologie

Cambridgeshire
CAMBRIDGE

Treatment of thromBocytopenia With EltRombopag or Intravenous Immune Globulin (IVIG) Before and DurING Invasive Procedures in Patients With Immune ThrombocytoPenia- BRIDGING ITP Study (Phase III)
University of Cambridge
Department of Medicine

Cornwall
TRURO
A Multicenter, Open-label, Multiple-dose Study to Evaluate the Safety, Tolerability, and Efficacy of UCB7665 in Subjects With Primary Immune Thrombocytopenia-GB
Royal Cornwall Hospital

Essex
LONDON

Open-label, Multicenter Evaluation of Safety, Pharmacokinetics, and Efficacy of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in the Prevention and Treatment of Bleeding Episodes in Pediatric Subjects With Hemophilia B - UK
Great Ormond Street Hospital
Great Ormond Street Haemophilia Comprehensive Care Centre

Greater London
UXBRIDGE

Greater Manchester
ADDRESS: NOT PROVIDED - UK

A Phase I/II, Open Label, Multicentre, Ascending Single Dose, Safety Study of a Novel Adeno- Associated Viral Vector (FLT180a) in Patients With Haemophilia B
Institution: Information not provided - UK

Greater Manchester
ADDRESS: NOT PROVIDED - UK


ATLAS-INH: A Phase 3 study to evaluate the efficacy and safety of fitusiran in patients with hemophilia A or B, with inhibitory antibodies to factor VIII or IX -GB
Institution: Information not provided - UK

Greater Manchester
ADDRESS: NOT PROVIDED - UK

ATLAS-A/B: A Phase 3 study to evaluate the efficacy and safety of fitusiran in patients with hemophilia A or B, without inhibitory antibodies to factor VIII or IX-CA
Institution: Information not provided - UK

Suisse Romande
ADDRESS: NOT PROVIDED - CH
ATLAS-PPX trial: an open-label, multinational, switching study to describe the efficacy and safety of fitusiran prophylaxis in hemophilia A and B patients previously receiving factor or bypassing agent prophylaxis - CH
Institution: Information not provided - CH

Berlin
ADDRESS: NOT PROVIDED - DE
A phase I/II, open-label, uncontrolled, single-dose, dose-ascending, multi-centre trial investigating an adeno-associated viral vector containing a codon-optimized human factor IX gene (AAV5-hFIX) administered to adult patients with severe or moderately severe haemophilia B -DE-
Institution: Information not provided - DE

Berlin
ADDRESS: NOT PROVIDED - DE
A low-interventional Multicentre Post-Authorisation Safety Study for Voncento/Biostate/Aleviate for Routine Prophylaxis, Treatment of Bleeding Events and/or Surgery in Male Subjects with Haemophilia A
Institution: Information not provided - DE

Berlin
ADDRESS: NOT PROVIDED - DE
XTEND-1: A Phase 3 Open-Label, Multicenter Study of the Safety, Efficacy, and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients aged 12 and over With Severe Hemophilia A - DE
Institution: Information not provided - DE

Berlin
BERLIN
Get8: A phase 1/2 open-label safety and dose-finding study of BAY 2599023 (DTX201), an adeno-associated virus (AAV) hu37-mediated gene transfer of B-domain deleted human factor VIII, in adults with severe hemophilia A -DE-
Vivantes
Vivantes - Netzwerk für Gesundheit GmbH

Berlin
BERLIN

STASEY: A single-arm, multicenter Phase IIIb clinical trial to evaluate the safety and tolerability of prophylactic Emicizumab in Hemophilia A patients with inhibitors -DE-
Vivantes Klinikum im Friedrichshain
Klinik für Angiologie und Hämostaseologie

Hessen
FRANKFURT AM MAIN
Get8: A phase 1/2 open-label safety and dose-finding study of BAY 2599023 (DTX201), an adeno-associated virus (AAV) hu37-mediated gene transfer of B-domain deleted human factor VIII, in adults with severe hemophilia A -DE-
Klinikum der Johann Wolfgang Goethe-Universität Frankfurt

Hessen
MÖRFELDEN-WALLDORF

STASEY: A single-arm, multicenter Phase IIIb clinical trial to evaluate the safety and tolerability of prophylactic Emicizumab in Hemophilia A patients with inhibitors -DE-
Hämophilie-Zentrum Rhein Main GmbH

Niedersachsen
HANNOVER

A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects With Transfusion-dependent-Thalassemia, Who Have a beta 0/beta 0 Genotype, by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral A-T87Q-Globin Vector in Subjects = 50 Years of Age -DE-
Medizinische Hochschule Hannover
Medizinische Hochschule Hannover (MHH)

Nordrhein-Westfalen
BONN

STASEY: A single-arm, multicenter Phase IIIb clinical trial to evaluate the safety and tolerability of prophylactic Emicizumab in Hemophilia A patients with inhibitors -DE-
Universitätsklinikum Bonn (AöR)
Institut für Experimentelle Hämatologie und Transfusionsmedizin

Saarland
HOMBURG
Get8: A phase 1/2 open-label safety and dose-finding study of BAY 2599023 (DTX201), an adeno-associated virus (AAV) hu37-mediated gene transfer of B-domain deleted human factor VIII, in adults with severe hemophilia A -DE-
Universitätsklinikum des Saarlandes

Sachsen
LEIPZIG

STASEY: A single-arm, multicenter Phase IIIb clinical trial to evaluate the safety and tolerability of prophylactic Emicizumab in Hemophilia A patients with inhibitors -DE-
Universitätsklinikum Leipzig AöR
Klinik und Poliklinik für Hämatologie, Zelltherapie und Hämostaseologie

WIEN
ADDRESS: NOT PROVIDED - AT

Paradigm 6: An Open-label Single-arm Multicentre Non-controlled Phase 3a Trial Investigating Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Prophylaxis and Treatment of Bleeding Episodes in Previously Untreated Patients With Haemophilia B (FIX Activity Below or Equal to 2 Percent) - AT
Institution: Information not provided - AT

WIEN
ADDRESS: NOT PROVIDED - AT
Pathfinder 6: An Open-label Single-arm Multicentre Non-controlled Phase 3a Trial Investigating Safety and Efficacy of N8-GP in Prophylaxis and Treatment of Bleeding Episodes in Previously Untreated Paediatric Patients With Severe Haemophilia A - AT
Institution: Information not provided - AT

WIEN
ADDRESS: NOT PROVIDED - AT
explorer7: Efficacy and safety of Concizumab prophylaxis in patients with haemophilia A or B with inhibitors (phase 3) - AT
Institution: Information not provided - AT

WIEN
ADDRESS: NOT PROVIDED - AT
An Open-Label, Multinational, Phase 1/2 Study of the Safety and Dose Escalation of SHP648, an Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing FIX Padua in Hemophilia B Subjects - AT
Institution: Information not provided - AT

WIEN
ADDRESS: NOT PROVIDED - AT

FRONTIER1: Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Subcutaneous Doses of NNC0365-3769 (Mim8) in Healthy Subjects and in Subjects With Haemophilia A With or Without Factor VIII Inhibitors (Phase 2) - AT
Institution: Information not provided - AT

WIEN
WIEN

A Phase 3b Open-label, Multicenter, Safety and Efficacy Extension Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects with Hemophilia B - AT
Allgemeines Krankenhaus der Stadt Wien
Klinische Abteilung für Hämatologie und Hämostaseologie

WIEN
WIEN
A Phase III Open Label, Multicenter, Extension Study to Assess the Safety and Efficacy of Recombinant Coagulation Factor VIII (rVIII-SingleChain, CSL627) in Subjects with Severe Hemophilia A - AT
Allgemeines Krankenhaus der Stadt Wien
Klinische Abteilung für Hämatologie und Hämostaseologie

WIEN
WIEN


A Global, Open-Label, Multicenter, Phase 1/2 Study of the Safety and Dose Escalation of BAX 888, an Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing B-Domain Deleted Factor VIII (BDD-FVIII) in Severe Hemophilia A Subjects Administered a Single Intravenous Infusion - AT
Allgemeines Krankenhaus der Stadt Wien
Klinische Abteilung für Hämatologie und Hämostaseologie

WIEN
WIEN

A Phase 3b Open-label, Multicenter, Safety and Efficacy Extension Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects with Hemophilia B - AT
Allgemeines Krankenhaus der Stadt Wien
Klinische Abteilung für Pädiatrische Kardiologie - Kinderherzzentrum Wien

WIEN
WIEN


Phase 3, Prospective, Multi-center, Open Label Study to Investigate Safety, Immunogenicity, and Hemostatic Efficacy of PEGylated Factor VIII (BAX 855) in Previously Untreated Patients (PUPs) and Minimally Treated Patients (MTPs) < 6 Years With Severe Hemophilia A (FVIII < 1%) - AT
Allgemeines Krankenhaus der Stadt Wien
Klinische Abteilung für Pädiatrische Kardiologie - Kinderherzzentrum Wien

WIEN
WIEN
LEOPOLD KIDS: A Multicenter Phase III Uncontrolled Open-label Trial to Evaluate Safety and Efficacy of BAY81-8973 in Children With Severe Hemophilia A Under Prophylaxis Therapy - AT
Allgemeines Krankenhaus der Stadt Wien
Klinische Abteilung für Pädiatrische Kardiologie - Kinderherzzentrum Wien

WIEN
WIEN
PROTECT KIDS: A Multi-center, Phase III, Non-controlled, Open-label Trial to Evaluate the Pharmacokinetics, Safety, and Efficacy of BAY94-9027 for Prophylaxis and Treatment of Bleeding in Previously Treated Children (Age <12 Years) With Severe Hemophilia A - AT
Allgemeines Krankenhaus der Stadt Wien
Klinische Abteilung für Pädiatrische Kardiologie - Kinderherzzentrum Wien

WIEN
WIEN

ARRONDISSEMENT BRUSSELS-CAPITAL
ADDRESS: NOT PROVIDED - BE
XTEND-1: A Phase 3 Open-Label, Multicenter Study of the Safety, Efficacy, and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients aged 12 and over With Severe Hemophilia A - BE
Institution: Information not provided - BE

ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS

Does the Thrombin Generation Test Performed During the Pharmacokinetic Profile of the Substitutive Factor VIII Bring Benefits to the Personalized Treatment of Pediatric Patients and Adult Hemophilia A Patients Under Prophylaxis ?
CHU Brugmann
Hémato-oncologie

VLAAMS BRABANT
LEUVEN

STASEY: A single-arm, multicenter Phase IIIb clinical trial to evaluate the safety and tolerability of prophylactic Emicizumab in Hemophilia A patients with inhibitors - BE
UZ Leuven - Campus Gasthuisberg
Hemofiliecentrum

Ontario
HAMILTON
A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) - CA
McMaster University Medical Centre Neuromuscular and Neurometabolic Clinic
Neuromuscular and Neurometabolic Clinic

Andalucía
MÁLAGA
A phase 3 open-label, single-arm study to evaluate the efficacy and safety of BMN 270, an adeno-associated virus vector-mediated gene transfer of human factor VIII in hemophilia A patients with residual FVIII levels less or equal to 1 IU/dL receiving prophylactic FVIII infusions - ES
Hospital Regional Universitario de Málaga - Hospital General
Unidad de Hematología

Andalucía
MÁLAGA
A Global, Open-Label, Multicenter, Phase 1/2 Study of the Safety and Dose Escalation of BAX 888, an Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing B-Domain Deleted Factor VIII (BDD-FVIII) in Severe Hemophilia A Subjects Administered a Single Intravenous Infusion - ES
Hospital Regional Universitario de Málaga - Hospital General
Unidad de Hematología

Andalucía
SEVILLA

HAVEN 3: A randomized, multicenter, open-label, phase III clinical trial to evaluate the efficacy, safety, and pharmacokinetics of prophylactic emicizumab versus no prophylaxis in hemophilia A patients without inhibitors - ES
Hospital Universitario Virgen del Rocío
Unidad de Hematología

Andalucía
SEVILLA
A phase 3 open-label, single-arm study to evaluate the efficacy and safety of BMN 270, an adeno-associated virus vector-mediated gene transfer of human factor VIII in hemophilia A patients with residual FVIII levels less or equal to 1 IU/dL receiving prophylactic FVIII infusions - ES
Hospital Universitario Virgen del Rocío
Unidad de Hematología

Baleares
PALMA DE MALLORCA

ATLAS-PEDS: An open-label, multinational study of fitusiran prophylaxis in male pediatric subjects aged 1 to less than 12 years with hemophilia A or B (Phase II-III) - ES
Hospital Universitario Son Espases
Servicio de Hematología

Castilla - León
VALLADOLID
Phase 3, Prospective, Multi-center, Open Label Study to Investigate Safety, Immunogenicity, and Hemostatic Efficacy of PEGylated Factor VIII (BAX 855) in Previously Untreated Patients (PUPs) and Minimally Treated Patients (MTPs) < 6 Years With Severe Hemophilia A (FVIII < 1%) - ES
Hospital Universitario Río Hortega
Servicio de Hematología y Hemoterapia

Castilla - León
VALLADOLID

An Open-Label, [...] Lead-In Study To Evaluate At Least 6 Months Of Prospective Efficacy And Selected Safety Data Of Factor IX (FIX) Prophylaxis Replacement Therapy [...] Of Moderately Severe To Severe Adult Hemophilia B Subjects (FIX:C=2%) Who Are Negative For Neutralizing Antibodies (Nab) To Adeno-Associated Virus Vector (Aav)-Spark100 (Phase III) - ES
Hospital Universitario Río Hortega
Servicio de Hematología y Hemoterapia

Cataluña
BARCELONA

An Open-Label, [...] Lead-In Study To Evaluate At Least 6 Months Of Prospective Efficacy And Selected Safety Data Of Factor IX (FIX) Prophylaxis Replacement Therapy [...] Of Moderately Severe To Severe Adult Hemophilia B Subjects (FIX:C=2%) Who Are Negative For Neutralizing Antibodies (Nab) To Adeno-Associated Virus Vector (Aav)-Spark100 (Phase III) - ES
Hospital Universitari Vall d'Hebron
Unidad de Hemofilia y coagulopatías congénitas

Cataluña
BARCELONA
A Global, Open-Label, Multicenter, Phase 1/2 Study of the Safety and Dose Escalation of BAX 888, an Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing B-Domain Deleted Factor VIII (BDD-FVIII) in Severe Hemophilia A Subjects Administered a Single Intravenous Infusion - ES
Hospital Universitari Vall d'Hebron
Unidad de Hemofilia y coagulopatías congénitas

Cataluña
BARCELONA
verITI-8 Study: A Non-controlled, Open-Label, Multicenter, Study of Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Subjects with Inhibitors Undergoing the First ITI Treatment (Phase IV) - ES
Hospital Universitari Vall d'Hebron

Cataluña
BARCELONA

ATLAS-PEDS: An open-label, multinational study of fitusiran prophylaxis in male pediatric subjects aged 1 to less than 12 years with hemophilia A or B (Phase II-III) - ES
Hospital Universitari Vall d'Hebron
Servicio de Hematología y Hemoterapia

Comunidad Valenciana
VALENCIA
verITI-8 Study: A Non-controlled, Open-Label, Multicenter, Study of Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Subjects with Inhibitors Undergoing the First ITI Treatment (Phase IV) - ES
Hospital Universitario y Politécnico La Fe

Comunidad Valenciana
VALENCIA

An Open-Label, [...] Lead-In Study To Evaluate At Least 6 Months Of Prospective Efficacy And Selected Safety Data Of Factor IX (FIX) Prophylaxis Replacement Therapy [...] Of Moderately Severe To Severe Adult Hemophilia B Subjects (FIX:C=2%) Who Are Negative For Neutralizing Antibodies (Nab) To Adeno-Associated Virus Vector (Aav)-Spark100 (Phase III) - ES
Hospital Universitario y Politécnico La Fe
Servicio de Hematología y Hemoterapia

Comunidad Valenciana
VALENCIA

Paradigm 6: An open-label single-arm multicentre non-controlled phase 3a trial investigating safety and efficacy of nonacog beta pegol (N9-GP) in prophylaxis and treatment of bleeding episodes in previously untreated patients with haemophilia B (FIX activity ?2%) - ES
Hospital Universitario y Politécnico La Fe
Unidad de Hemostasia y Trombosis

Galicia
A CORUÑA
A phase 3 open-label, single-arm study to evaluate the efficacy and safety of BMN 270, an adeno-associated virus vector-mediated gene transfer of human factor VIII in hemophilia A patients with residual FVIII levels less or equal to 1 IU/dL receiving prophylactic FVIII infusions - ES
Hospital Teresa Herrera
Departamento de Hematología y Hemoterapia

Madrid
ADDRESS: NOT PROVIDED - ES

Get8: A phase 1/2 open-label safety and dose-finding study of BAY 2599023 (DTX201), an adeno-associated virus (AAV) hu37-mediated gene transfer of B-domain deleted human factor VIII, in adults with severe hemophilia A - ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES
An Open-Label, Multinational, Phase 1/2 Study of the Safety and Dose Escalation of SHP648, an Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing FIX Padua in Hemophilia B Subjects - ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES


An Open-Label Study in Adolescent and Adult Severe (Coagulation Factor Activity <1%) Hemophilia A or B Patients With or Without Inhibitors Comparing Standard Treatment to PF-06741086 Prophylaxis - ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

FRONTIER1: Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Subcutaneous Doses of NNC0365-3769 (Mim8) in Healthy Subjects and in Subjects With Haemophilia A With or Without Factor VIII Inhibitors - ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

HAVEN 6: A Multicenter, Open-Label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Emicizumab in Patients With Mild or Moderate Hemophilia A Without FVIII Inhibitors - ES
Institution: Information not provided - ES

Madrid
MADRID
Phase 3, Prospective, Multi-center, Open Label Study to Investigate Safety, Immunogenicity, and Hemostatic Efficacy of PEGylated Factor VIII (BAX 855) in Previously Untreated Patients (PUPs) and Minimally Treated Patients (MTPs) < 6 Years With Severe Hemophilia A (FVIII < 1%) - ES
Hospital Universitario La Paz
Servicio de Hematología

Madrid
MADRID

ATLAS-PEDS: An open-label, multinational study of fitusiran prophylaxis in male pediatric subjects aged 1 to less than 12 years with hemophilia A or B (Phase II-III) - ES
Hospital Universitario La Paz
Servicio de Hematología

Madrid
MADRID

XTEND-1: Phase 3 Open-Label, Multicenter Study of the Safety, Efficacy, and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients >=12 Years of Age With Severe Hemophilia A - ES
Hospital Universitario La Paz
Servicio de Hematología

Madrid
MADRID

HAVEN 3: A randomized, multicenter, open-label, phase III clinical trial to evaluate the efficacy, safety, and pharmacokinetics of prophylactic emicizumab versus no prophylaxis in hemophilia A patients without inhibitors - ES
Hospital Universitario La Paz
Servicio de Hematología

Madrid
MADRID
verITI-8 Study: A Non-controlled, Open-Label, Multicenter, Study of Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Subjects with Inhibitors Undergoing the First ITI Treatment (Phase IV) - ES
Hospital Universitario La Paz
Servicio de Hematología

Madrid
MADRID
A phase 3 open-label, single-arm study to evaluate the efficacy and safety of BMN 270, an adeno-associated virus vector-mediated gene transfer of human factor VIII in hemophilia A patients with residual FVIII levels less or equal to 1 IU/dL receiving prophylactic FVIII infusions - ES
Hospital Universitario La Paz
Servicio de Hematología

Madrid
MADRID

An Open-Label, [...] Lead-In Study To Evaluate At Least 6 Months Of Prospective Efficacy And Selected Safety Data Of Factor IX (FIX) Prophylaxis Replacement Therapy [...] Of Moderately Severe To Severe Adult Hemophilia B Subjects (FIX:C=2%) Who Are Negative For Neutralizing Antibodies (Nab) To Adeno-Associated Virus Vector (Aav)-Spark100 (Phase III) - ES
Hospital Universitario La Paz
Servicio de Hematología

Madrid
MADRID
A Global, Open-Label, Multicenter, Phase 1/2 Study of the Safety and Dose Escalation of BAX 888, an Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing B-Domain Deleted Factor VIII (BDD-FVIII) in Severe Hemophilia A Subjects Administered a Single Intravenous Infusion - ES
Hospital Universitario La Paz
Servicio de Hematología

Massachusetts
CAMBRIDGE

Massachusetts
CAMBRIDGE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
PROTECT-VIII: A phase II/III, multicenter, partially randomized, open label trial investigating safety and efficacy of on-demand and prophylactic treatment with BAY94-9027 in Severe hemophilia A - FR
Institution: Information not provided - FR

AUVERGNE-RHONE-ALPES
BRON

Phase I/II/III pharmacokinetic and outcome study of Inspiration's recombinant Factor IX product, IB1001, in subjects with hemophilia B - FR
CHU de Lyon HCL - GH Est-Hôpital Femme Mère Enfant
Service d'hématologie biologique et d'hémostase clinique

ILE-DE-FRANCE
PARIS

County Dublin
DUBLIN

8HA01EXT: An Open-Label, Multicenter Evaluation of the Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) in the Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects With Hemophilia A (Phase III) - IE
Children's Health Ireland @ Crumlin
Haematology Department

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
A Phase I/II, Open-label, Uncontrolled, Single-dose, Dose-ascending, Multi-centre Trial Investigating an Adeno-associated Viral Vector Containing a Codon-optimized Human Factor IX Gene (AAV5-hFIX) Administered to Adult Patients With Severe or Moderately Severe Hemophilia B - IT
Institution: Information not provided - IT

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT

XTEND-1: Phase 3 Open-Label, Multicenter Study of the Safety, Efficacy, and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients greater than or equal to 12 Years of Age With Severe Hemophilia A - IT
Institution: Information not provided - IT

EMILIA ROMAGNA
FERRARA
Gentamycin treatment of congenital coagulation factor VII or factor IX deficiency caused by nonsense mutations (phase II)
Università degli Studi di Ferrara
Laboratorio di Biologia Molecolare dell'Emostasi e Trombosi

LAZIO
ROMA
PROTECT-VIII: A Phase II/III, multicenter, partially randomized, open label trial investigating safety and efficacy of on-demand and prophylactic treatment with BAY 94-9027 in Severe Hemophilia A - IT
Sapienza Università di Roma - Clinica Ematologica
U.O.S. Diagnosi Speciale e Terapia dell'Emostasi e della Trombosi - U.O.C Ematologia - Sezione Ematologia

LOMBARDIA
MILANO

A Multicenter Phase III Uncontrolled Open-label Trial to Evaluate Safety and Efficacy of BAY81-8973 in Children With Severe Hemophilia A Under Prophylaxis Therapy - IT
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico
U.O.S.D. Diagnosi e Terapia delle Coagulopatie

LOMBARDIA
MILANO
A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) - IT
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico
Dipartimento di Scienze Neurologiche

Zuid-Holland
ROTTERDAM
Little DAVID: Desmopressin and FVIII concentrate combination treatment in non-severe hemophilia A patients undergoing minor interventions
Erasmus MC - Erasmus Medisch Centrum
Afdeling Hematologie

Zuid-Holland
ROTTERDAM

DAVID study: DDAVP treatment combined with FVIII clotting factor concentrates in patients with mild haemophilia A
Erasmus MC - Erasmus Medisch Centrum
Afdeling Hematologie

Avon
BRISTOL
A Phase IIIb Continuation study of the Safety and Efficacy of PEGylated Recombinant Factor VIII (PEG-rFVIII; BAX-855) in Prophylaxis of Bleeding in Previously Treated Patients with Severe Hemophilia A - UK
Bristol Royal Hospital for Children
Bristol Haematology and Oncology Centre

Essex
LONDON

MCRN127 (GENA-03): Prospective Clinical Study in Children with Severe Haemophilia A to Investiagte Clinical Efficacy, Immunogenicity, Pharmacokinetics, and Safety of OF Human-CL RHFVIII (Phase III) - UK
Great Ormond Street Hospital
Great Ormond Street Haemophilia Comprehensive Care Centre

Essex
LONDON
A phase III Extension Study for Patients who completed GENA 05 (NuProtect) to Investigate Immunogenicity, Efficacy and Safety of Treatment with Human-cl rhFVIII - UK
Great Ormond Street Hospital
Great Ormond Street Haemophilia Comprehensive Care Centre

Greater London
LONDON
A Phase 3, Multi-Center, Open Label Study of Efficacy and Safety of PEGylated rFVIII (BAX 855) in Previously Treated Patients With Severe Hemophilia A Undergoing Surgical or Other Invasive Procedures - UK
Royal Free Hospital
The Katharine Dormandy Haemophilia Centre and Thrombosis Unit

Greater London
LONDON


GO-8: Gene Therapy for Haemophilia A Using a Novel Serotype 8 Capsid Pseudotyped Adeno-associated Viral Vector Encoding Factor VIII-V3
Royal Free Hospital
The Katharine Dormandy Haemophilia Centre and Thrombosis Unit

Greater London
LONDON

An Open-Label, Multicentre, Long-Term Follow-Up Study to Investigate the Safety and Durability of Response Following Dosing of a Novel Adeno-Associated Viral Vector (FLT180a) in Patients With Haemophilia B -GB
Royal Free Hospital
The Katharine Dormandy Haemophilia Centre and Thrombosis Unit

Greater London
LONDON
An open label dose-escalation study of a self complementary adeno-associated viral vector (scAAV2/8-LP1-hFIXCO) for gene therapy of haemophilia B (Phase I/II)
UCL Cancer Institute, University College London
Research Department of Haematology

Greater Manchester
ADDRESS: NOT PROVIDED - UK

B-YOND: An Open-Label, Multicenter Evaluation of the Long-Term Safety and Efficacy of Recombinant, Human Coagulation Factor IX Fusion Protein (rfixfc) in the Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects With Hemophilia B (Phase III) - UK
Institution: Information not provided - UK

Greater Manchester
ADDRESS: NOT PROVIDED - UK

PROTECT VIII: A phase II/III, multicenter, partially randomized, open label trial investigating safety and efficacy of on-demand and prophylactic treatment with BAY94-9027 in Severe hemophilia A - UK
Institution: Information not provided - UK

Greater Manchester
ADDRESS: NOT PROVIDED - UK

8HA01EXT: An Open-Label, Multicenter Evaluation of the Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) in the Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects With Hemophilia A (Phase III) - UK
Institution: Information not provided - UK

Greater Manchester
ADDRESS: NOT PROVIDED - UK

Immunogenicity, Efficacy and Safety of Treatment with Human-cl rhFVIII in Previously Untreated Patients with Severe Haemophilia A (phase III) - UK
Institution: Information not provided - UK

Greater Manchester
ADDRESS: NOT PROVIDED - UK
A randomized, open label, phase 2 study of Rituximab and Bendamustine with or without Brentuximab Vedotin for relapsed or refractory cd30-positive diffuse large B cell lymphoma - GB
Institution: Information not provided - UK

Greater Manchester
ADDRESS: NOT PROVIDED - UK
An Open-label Extension Study of Subcutaneously Administered ALN-AT3SC in Patients with Moderate or Severe Hemophilia A or B who have Participated in a Previous Clinical Study with ALN-AT3SC - GB
Institution: Information not provided - UK

Greater Manchester
MANCHESTER

Get8: A Phase 1/2 Open-label Safety and Dose-finding Study of BAY2599023 (DTX201), an Adeno-associated Virus (AAV) hu37-mediated Gene Transfer of B-domain Deleted Human Factor VIII, in Adults With Severe Hemophilia A-GB
Manchester University NHS Foundation Trust - Manchester Royal Infirmary
Manchester Haemophilia Comprehensive Care Centre, Adult's

Hampshire
BASINGSTOKE
CCRN 756: A Multi-national Trial Evaluating Safety and Efficacy, including Pharmacokinetics, of NNC 0129-0000-1003 (N8-GP) when Administered for Treatment and Prophylaxis of Bleeding in Patients with Haemophilia A (Phase III) - UK
Basingstoke and North Hampshire Hospital NHS Foundation Trust
North Hampshire Haemophilia Centre

Hampshire
BASINGSTOKE
CCRN 472: Safety and Efficacy of NNC-0156-0000-0009 After Long-Term Exposure in Patients With Haemophilia B (Phase III) - UK
Basingstoke and North Hampshire Hospital NHS Foundation Trust
North Hampshire Haemophilia Centre

Hampshire
BASINGSTOKE

Pathfinder 3: Efficacy and Safety of NNC 0129-0000-1003 (N8-GP) during Surgical Procedures in Patients with Haemophilia A (Phase III) - UK
Basingstoke and North Hampshire Hospital NHS Foundation Trust
North Hampshire Haemophilia Centre

Hampshire
BASINGSTOKE
A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients with Severe Haemophilia A (phase III) - UK
Basingstoke and North Hampshire Hospital NHS Foundation Trust
North Hampshire Haemophilia Centre

Oxfordshire
OXFORD

An open label Safety and Efficacy extension study of GreenGene (TM) F in Previously Treated Patients Diagnosed with Severe Hemophilia A (Phase III) - UK
Churchill Hospital
Oxford Haemophilia and Thrombosis Centre (OHTC)

Oxfordshire
OXFORD

GreenGene F_P3: Determination of Safety, Efficacy, and Pharmacokinetics of GreenGeneTM F in Previously Treated Patients 12 years of age or older Diagnosed with Severe Hemophilia A (Phase III) - UK
Churchill Hospital
Oxford Haemophilia and Thrombosis Centre (OHTC)

Tyne & Wear
NEWCASTLE UPON TYNE

PROTECT KIDS: A Multi-center, Phase III, Non-controlled, Open-label Trial to Evaluate the Pharmacokinetics, Safety, and Efficacy of BAY94-9027 for Prophylaxis and Treatment of Bleeding in Previously Treated Children (Age <12 Years) With Severe Hemophilia A - UK
Institute of Cellular Medicine, Newcastle University, Medical School
Institute of Cellular Medicine

Tyne & Wear
NEWCASTLE UPON TYNE
A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) - UK
Newcastle upon Tyne Hospitals NHS Trust
John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine

West Midlands
BIRMINGHAM
PARADIGM 5: Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 (N9-GP) in Previously Treated Children with Haemophilia B (phase III) - UK
Birmingham Children's Hospital NHS Foundation Trust
Haemophilia Comprehensive Comprehensive Care

West Midlands
BIRMINGHAM
STASEY: A single-arm, multicenter phase IIIB clinical trial to evaluate the safety and tolerability of prophylactic Emicizumab in Hemophilia A patients with inhibitors.GB
Birmingham Children's Hospital NHS Foundation Trust
Haemophilia Comprehensive Comprehensive Care

Suisse Alémanique
LACHEN

Suisse Romande
ADDRESS: NOT PROVIDED - CH

A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects With Transfusion-dependent-Thalassemia, Who Have a beta 0/beta 0 Genotype, by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral A-T87Q-Globin Vector in Subjects = 50 Years of Age - GB
Institution: Information not provided - CH

WIEN
WIEN


HCQ4Surfdefect: Hydroxychloroquine (HCQ) in pediatric ILD (interstitial lung disease) - AT
Allgemeines Krankenhaus der Stadt Wien
Klinische Abteilung für Pädiatrische Pulmologie, Allergologie und Endokrinologie

Andalucía
SEVILLA

HCQ-chILD-EU: Hydroxychloroquine in pediatric ILD - START randomized controlled in parallel-group, then switch placebo to active drug, and STOP randomized controlled in parallel-group to evaluate the efficacy and safety of hydroxychloroquine (HCQ) (Phase II) - ES
Hospital Universitario Virgen del Rocío
Unidad de Pediatría

Cataluña
BARCELONA

HCQ-chILD-EU: Hydroxychloroquine in pediatric ILD - START randomized controlled in parallel-group, then switch placebo to active drug, and STOP randomized controlled in parallel-group to evaluate the efficacy and safety of hydroxychloroquine (HCQ) (Phase II) - ES
Hospital Universitari Vall d'Hebron
Unidad de Neumología Pediátrica y Fibrosis Quística

Comunidad Valenciana
VALENCIA

HCQ-chILD-EU: Hydroxychloroquine in pediatric ILD - START randomized controlled in parallel-group, then switch placebo to active drug, and STOP randomized controlled in parallel-group to evaluate the efficacy and safety of hydroxychloroquine (HCQ) (Phase II) - ES
Hospital Clínico Universitario de Valencia
Servicio de Pediatría

Madrid
MADRID

HCQ-chILD-EU: Hydroxychloroquine in pediatric ILD - START randomized controlled in parallel-group, then switch placebo to active drug, and STOP randomized controlled in parallel-group to evaluate the efficacy and safety of hydroxychloroquine (HCQ) (Phase II) - ES
Hospital Universitario 12 de Octubre
Servicio de Pediatría

Madrid
MADRID

HCQ-chILD-EU: Hydroxychloroquine in pediatric ILD - START randomized controlled in parallel-group, then switch placebo to active drug, and STOP randomized controlled in parallel-group to evaluate the efficacy and safety of hydroxychloroquine (HCQ) (Phase II) - ES
Hospital Universitario La Paz
Servicio de Pediatría

Pennsylvania
PHILADELPHIA


Reinstituting Natural Killer Cell Cytotoxicity and Cytoskeletal Dynamics in Wiskott-Aldrich Syndrome With IL-2 Therapy (Phase 1) - US
The Children's Hospital of Philadelphia
Department of Allergy and Asthma

ILE-DE-FRANCE
PARIS


WAS FUP : Long Term Safety Follow up of Patients Enrolled in the Phase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome (GTG002-07 and GTG003-08) - FR
Hôpital Necker-Enfants Malades
Département de Biothérapie - Unité d'Hémaphérèse Thérapeutique

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT

A Single Arm, Open-label Clinical Trial of Hematopoietic Stem Cell Gene Therapy With Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Encoding WAS cDNA in Subjects With Wiskott-Aldrich Syndrome (WAS)
Institution: Information not provided - IT

LOMBARDIA
MILANO

TIGET-WAS - A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Wiskott-Aldrich Syndrome
Istituto San Raffaele Telethon per la Terapia Genica - TIGET
Istituto San Raffaele Telethon per la Terapia Genica

Greater London
LONDON

Phase I/II clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome - UK
Great Ormond Street Hospital
Molecular and Cellular Immunology Unit

Greater London
LONDON

Long Term Safety Follow up of Patients Enrolled in the Phase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome (GTG002-07 and GTG003-08) - GB
Great Ormond Street Hospital
Molecular and Cellular Immunology Unit
Ensaio (s) clínico (s) multinacional (ais)

Berlin
ADDRESS: NOT PROVIDED - DE
A PHASE 3 CLINICAL STUDY TO DETERMINE THE PHARMACOKINETICS, SAFETY AND EFFICACY OF rVWF:rFVIII and rVWF IN THE TREATMENT OF BLEEDING EPISODES IN SUBJECTS DIAGNOSED WITH VON WILLEBRAND DISEASE (Coordination)
Institution: Information not provided - DE

Berlin
ADDRESS: NOT PROVIDED - DE
A Phase 3, Prospective, Multicenter Study to Evaluate Efficacy and Safety of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Elective Surgical Procedures in Subjects With Severe Von Willebrand Disease
Institution: Information not provided - DE

WIEN
WIEN
ANAHYDRET: A single blind, multi-center, randomised multinational phase III study to compare the efficacy and tolerability of Anagrelide versus Hydroxyurea in patients with essential thrombocythaemia
AOP Orphan Pharmaceuticals AG

WIEN
WIEN

OOST-VLAANDEREN
ZWIJNAARDE

Jylland
BAGSVAERD

Delaware
WILMINGTON
An Open-Label Phase 2 Study of Itacitinib (INCB039110) in Combination With Low-Dose Ruxolitinib or Itacitinib Alone Following Ruxolitinib in Subjects With Myelofibrosis
Incyte Corporation

New York
NEW YORK
Single Arm Salvage Therapy With Pegylated Interferon Alfa-2a for Patients With High Risk Polycythemia Vera or High Risk Essential Thrombocythemia Who Are Either Hydroxyurea Resistant or Intolerant or Have Had Abdominal Vein Thrombosis
Icahn School of Medicine at Mount Sinai

Pennsylvania
WAYNE
SOAR-HI: A Phase 2, Multicenter, Randomized, Placebo-Controlled, Double-blind Study in Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP) to Evaluate the Pharmacokinetics,Safety and Efficacy of rADAMTS-13 (SHP655) Administered in Addition to Standard Of Care (SoC) Treatment
Shire Development LLC and International Affiliates

Washington
ADDRESS: NOT PROVIDED - US
PAC203: An Open-Label, Randomized, Phase 2 Dose-Finding Study of Pacritinib in Patients with Thrombocytopenia and Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis Previously Treated with Ruxolitinib
Institution: Information not provided - US

Washington
SEATTLE

Suisse Alémanique
BASEL
REALISE: A Multicenter Phase II, Open Label, Single Arm Study to Evaluate the Efficacy and Safety of Ruxolitinib in the Treatment of Anemic Myelofibrosis Patients
Novartis Pharma Services AG

Suisse Romande
ADDRESS: NOT PROVIDED - CH
The COMFORT-II Trial: Randomized Study of INCB018424 Tablets Compared to Best Available Therapy in Subjects With Primary Myelofibrosis, Post-Polycythemia Vera-Myelofibrosis or Post-Essential Thrombocythemia Myelofibrosis (Phase III) (coordination)
Institution: Information not provided - CH

Suisse Romande
ADDRESS: NOT PROVIDED - CH
JUMP: An Open-label, Multicenter, Expanded Access Study of INC424 for Patients With Primary Myelofibrosis (PMF) or Post Polycythemia Myelofibrosis (PPV MF) or Post-essential Thrombocythemia Myelofibrosis (PET-MF)
Institution: Information not provided - CH

Hessen
MÖRFELDEN-WALLDORF
MOTIVATE: MOdern Treatment of Inhibitor-PositiVe PATiEnts with Haemophilia A - An International Low-Interventional Pragmatic Investigator Initiated Trial
Hämophilie-Zentrum Rhein Main GmbH

WIEN
ADDRESS: NOT PROVIDED - AT
A prospective, phase 3, open-label, international multicenter study on efficacy and safety of prophylaxis with rVWF in severe von willebrand disease
Institution: Information not provided - AT

ARRONDISSEMENT BRUSSELS-CAPITAL
ADDRESS: NOT PROVIDED - BE
A Randomized, Double-blind, Placebo-controlled, Phase II Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients With Primary Immune Thrombocytopenia
Institution: Information not provided - BE

Jylland
BAGSVAERD

Massachusetts
CAMBRIDGE
ATLAS-OLE: An Open-label, Long-term Safety and Efficacy Study of Fitusiran in Patients with Hemophilia A or B, with or without Inhibitory Antibodies to Factor VIII or IX
Genzyme Corporation

Massachusetts
CAMBRIDGE
A 4-part Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of M254 in Healthy Volunteers and in Patients With Immune Thrombocytopenic Purpura
Momenta Pharmaceuticals, Inc.

Washington
ADDRESS: NOT PROVIDED - US
ATLAS-INH: A Phase 3 study to evaluate the efficacy and safety of fitusiran in patients with hemophilia A or B, with inhibitory antibodies to factor VIII or IX
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
ATLAS-A/B: A Phase 3 study to evaluate the efficacy and safety of fitusiran in patients with hemophilia A or B, without inhibitory antibodies to factor VIII or IX
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
ATLAS-PPX trial: an open-label, multinational, switching study to describe the efficacy and safety of fitusiran prophylaxis in hemophilia A and B patients previously receiving factor or bypassing agent prophylaxis.
Institution: Information not provided - US

Suisse Alémanique
BASEL
A Multicenter, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Subcutaneous Administration of Emicizumab in Hemophilia A Pediatric Patients With Inhibitors
Hoffmann-La Roche
Hoffmann - La Roche Ltd.

Suisse Alémanique
BASEL
HAVEN 3: A Randomized, Multicenter, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Patients Without Inhibitors
Hoffmann-La Roche
Hoffmann - La Roche Ltd.

Berlin
ADDRESS: NOT PROVIDED - DE
A Phase II/III Open-label, Multicenter, Safety and Efficacy Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects With Hemophilia B (Coordination)
Institution: Information not provided - DE

Berlin
ADDRESS: NOT PROVIDED - DE
PROTECT VIII: A Phase II/III, Multicenter, Partially Randomized, Open Label Trial Investigating Safety and Efficacy of On-demand and Prophylactic Treatment With BAY94-9027 in Severe Hemophilia A
Institution: Information not provided - DE

Berlin
ADDRESS: NOT PROVIDED - DE
PROTECT KIDS: A Multi-center, Phase III, Non-controlled, Open-label Trial to Evaluate the Pharmacokinetics, Safety, and Efficacy of BAY94-9027 for Prophylaxis and Treatment of Bleeding in Previously Treated Children (Age <12 Years) With Severe Hemophilia A
Institution: Information not provided - DE

WIEN
WIEN

WIEN
WIEN

Jylland
ADDRESS: NOT PROVIDED - DK
A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A (Phase III)
Institution: Information not provided - DK

Jylland
BAGSVAERD

Jylland
BAGSVAERD

Jylland
BAGSVAERD

Jylland
BAGSVAERD

Jylland
BAGSVAERD
explorer7: Efficacy and Safety of Concizumab Prophylaxis in Patients With Haemophilia A or B With Inhibitors
Novo Nordisk A/S

Massachusetts
CAMBRIDGE

Massachusetts
CAMBRIDGE

Massachusetts
CAMBRIDGE
ATLAS-PEDS: An open-label, multinational study of fitusiran prophylaxis in male pediatric subjects aged 1 to less than 12 years with hemophilia A or B
Genzyme Corporation

New York
NEW YORK

Washington
ADDRESS: NOT PROVIDED - US
A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects With Transfusion-dependent-Thalassemia, Who Have a beta 0/beta 0 Genotype, by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral A-T87Q-Globin Vector in Subjects = 50 Years of Age
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Phase 3B Open-Label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
Institution: Information not provided - US

ILE-DE-FRANCE
PARIS

ILE-DE-FRANCE
EVRY