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Sperimentazioni cliniche nazionali
STATI UNITI
California
WESTLAKE VILLAGE
Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects with Primary Immunodeficiency Diseases -GB
Baxalta US, Inc.
FRANCIA
HAUTS-DE-FRANCE
LILLE
Assessment of the IgG trough level in subjects with primary immunodeficiency switching from standard subcutaneous immunoglobulin (SCIG) to every other week HyQvia (Phase IV)
CHRU de Lille - Hôpital Claude Huriez
Service de médecine interne
AUSTRIA
TIROL
INNSBRUCK
FURI: Open-Label Study to Evaluate the Efficacy and Safety of SCY-078 in Patients With Fungal Diseases That Are Refractory to or Intolerant of Standard Antifungal Treatment (Phase 3) - AT
Medizinische Universität Innsbruck
Universitätsklinik für Innere Medizin II
AUSTRIA
WIEN
ADDRESS: NOT PROVIDED - AT
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients With WHIM Syndrome With Open-Label Extension - AT
Institution: Information not provided - AT
AUSTRIA
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
AUSTRIA
WIEN
WIEN
A Multicenter Study for the Long-term Follow-up of HLH Patients Who Received Treatment With NI-0501, an Anti-interferon Gamma Monoclonal Antibody (Phase II) - AT
St. Anna Kinderspital
Zentrum für Kinder- und Jugendheilkunde
BELGIO
VLAAMS BRABANT
LEUVEN
ReSPECT: A Phase 3, Multicenter, Randomized, Double-Blind Study of the Efficacy and Safety of Rezafungin for Injection Versus the Standard Antimicrobial Regimen to Prevent Invasive Fungal Diseases in Adults Undergoing Allogeneic Blood and Marrow Transplantation
UZ Leuven - Campus Gasthuisberg
Department of Hematology
FRANCIA
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
A one-year open-label, multicenter trial to assess efficacy, safety and tolerability of canakinumab (ACZ885) and the efficacy and safety of childhood vaccinations in patients aged 4 years or younger with Cryopyrin Associated Periodic Syndromes (CAPS) (Phase III) - FR
Institution: Information not provided - FR
FRANCIA
AUVERGNE-RHONE-ALPES
GRENOBLE
DexEnceph : Dexamethasone in Herpes Simplex Virus Encephalitis Open Label Randomized Controlled Trial With an Observer-blinded Evaluation at 6 Months (Phase III)
CHU Grenoble Alpes - Site Nord - Hôpital Couple Enfant
Service d'Infectiologie - Maladies infectieuses
FRANCIA
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
GERMANIA
Baden-Württemberg
TÜBINGEN
TOFFIFE: Tocilizumab for the Treatment of Familial Mediterranean Fever - A randomized, doubleblind, phase II proof of concept study
Interdisziplinäres Rheumazentrum (INDIRA)
Zentrum für Interdisziplinäre Rheumatologie, klinische Immunologie und Autoimmunerkrankungen
GERMANIA
Berlin
ADDRESS: NOT PROVIDED - DE
Safety of Abatacept in patients with interstitial lung disease and common variable immunodeficiency (CVID) and related disease
Institution: Information not provided - DE
GERMANIA
Nordrhein-Westfalen
MÜNSTER
A Pilot, open-label, single arm, multicentre study to explore safety, tolerability, pharmacokinetics and efficacy of intravenous multiple administrations of NI-0501, an Anti-interferon Gamma (Anti-IFNy) monoclonal antibody, in paediatric patients with Primary Haemophagocytic Lymphohistiocytosis which has reactivated (Phase II) - DE
Universitätsklinikum Münster
Klinik für Kinder- und Jugendmedizin - Pädiatrische Hämatologie und Onkologie
ITALIA
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
ITALIA
LOMBARDIA
MILANO
TIGET-WAS - Sperimentazione clinica di fase I / II di terapia genica con cellule staminali ematopoietiche per la sindrome di Wiskott-Aldrich
Istituto San Raffaele Telethon per la Terapia Genica - TIGET
Istituto San Raffaele Telethon per la Terapia Genica
ITALIA
LOMBARDIA
MONZA
Studio pilota, in aperto, a singolo braccio, multicentrico, per esplorare la sicurezza, tollerabilità, farmacocinetica ed efficacia di somministrazioni intravenose multiple di NI-0501, un anticorpo monoclonale anti-interferone gamma (anti-IFNgamma), in pazienti pediatrici con Linfoistiocitosi emofagocitica primaria (HLH) riattivata
ASST Monza - Ospedale San Gerardo
Centro Ricerca "M.Tettamanti"
ITALIA
TOSCANA
FIRENZE
Studio pilota, in aperto, a singolo braccio, multicentrico, per esplorare la sicurezza, tollerabilità, farmacocinetica ed efficacia di somministrazioni intravenose multiple di NI-0501, un anticorpo monoclonale anti-interferone gamma (anti-IFNgamma), in pazienti pediatrici con Linfoistiocitosi emofagocitica primaria (HLH) riattivata
Azienda Ospedaliera Universitaria Anna Meyer
D.A.I. Oncoematologia
REGNO UNITO; GRAN BRETAGNA
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
SPAGNA
Cataluña
ESPLUGUES DE LLOBREGAT
A multicentre study for the long-term follow-up of HLH patients who received treatment with NI-0501, an anti-interferon gamma monoclonal antibody (Phase II) - ES
Hospital Sant Joan de Déu Barcelona
Servicio de Alergia e Inmunología clínica
SPAGNA
Madrid
ADDRESS: NOT PROVIDED - ES
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients With WHIM Syndrome With Open-Label Extension - ES
Institution: Information not provided - ES
STATI UNITI
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
AUSTRIA
WIEN
ADDRESS: NOT PROVIDED - AT
TALAPRO-1: A phase 2, open-label, response rate study of talazoparib in men with DNA repair defects metastatic castration-resistant prostate cancer who previously received taxane-based chemotherapy and progressed on at least 1 novel hormonal agent (enzalutamide and/or abiraterone acetate/prednisone) - AT
Institution: Information not provided - AT
BELGIO
VLAAMS BRABANT
LEUVEN
OLE-IEDAT: Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study - BE
UZ Leuven - Campus Gasthuisberg
Laboratory of Pediatric Immunology
FINLANDIA
Finland
HELSINKI
Immunodeficiency in cartilage-hair hypoplasia: sub-project on safety of vaccination against chickenpox -FI
HUS - Helsinki University Hospital
New Children's Hospital
FRANCIA
ILE-DE-FRANCE
PARIS
A Phase I/II, Non Randomized, Monocentric Open-label Study of Autologous CD34+ Cells Transduced With the G1XCGD Lentiviral Vector in Patients With X-Linked Chronic Granulomatous Disease
Hôpital Necker-Enfants Malades
Service d'Immuno-hématologie pédiatrique
FRANCIA
ILE-DE-FRANCE
PARIS
C-HLH : First Line Treatment of Familiar Lymphohistiocytosis by Alemtuzumab (CAMPATH®) (Phase I-II)
Hôpital Necker-Enfants Malades
Service d'Immuno-hématologie pédiatrique
GERMANIA
Baden-Württemberg
FREIBURG
ABACHAI: Safety and effectiveness of Abatacept (s.c.) in patients with CTLA4 insufficiency or LRBA deficiency (study of the GAIN-Network)
CCI am Universitätsklinikum Freiburg
Centrum für Chronische Immundefizienz
GERMANIA
Bayern
MÜNCHEN
ABACHAI: Safety and effectiveness of Abatacept (s.c.) in patients with CTLA4 insufficiency or LRBA deficiency (study of the GAIN-Network)
Medizinische Klinik und Poliklinik IV - Standort Ziemssenstraße
Medizinische Klinik und Poliklinik IV
GERMANIA
Berlin
ADDRESS: NOT PROVIDED - DE
A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients with Ataxia Telangiectasia -DE-
Institution: Information not provided - DE
GERMANIA
Berlin
ADDRESS: NOT PROVIDED - DE
Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study -DE-
Institution: Information not provided - DE
GERMANIA
Hessen
FRANKFURT AM MAIN
OLE-IEDAT: Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study -DE-
Klinikum der Johann Wolfgang Goethe-Universität Frankfurt
Schwerpunkt Allergologie, Pneumologie und Mukoviszidose
GERMANIA
Hessen
FRANKFURT AM MAIN
gene therapy for X-CGD (X-CGD-Version2): A Phase I/II Gene Therapy Trial for X-CGD With a SIN Gammaretroviral Vector - DE
Klinikum der Johann Wolfgang Goethe-Universität Frankfurt
Medizinische Klinik II - Hämatologie, Onkologie, Hämostaseologie, Rheumatologie, Infektiologie
GERMANIA
Hessen
FRANKFURT AM MAIN
Phase I/II G1XCGD.01 Sudy: Phase I/II, Non Randomized, Multicenter, Open-label Study of g1xcgd (Lentiviral Vector Transduced cd34+ Cells) in Patients with X-linked Chronic Granulomatous Disease - DE
Klinikum der Johann Wolfgang Goethe-Universität Frankfurt
Medizinische Klinik II - Hämatologie, Onkologie, Hämostaseologie, Rheumatologie, Infektiologie
GERMANIA
Schleswig-Holstein
KIEL
ABACHAI: Safety and effectiveness of Abatacept (s.c.) in patients with CTLA4 insufficiency or LRBA deficiency (study of the GAIN-Network)
Universitätsklinikum Schleswig-Holstein - Campus Kiel
Klinik für Innere Medizin I
ITALIA
TOSCANA
FIRENZE
EURO-HIT-HLH: Studio cooperativo pilota per la valutazione della terapia immunologica combinata della Linfoistiocitosi Emofagocitica Familiare
Azienda Ospedaliera Universitaria Anna Meyer
D.A.I. Oncoematologia
REGNO UNITO; GRAN BRETAGNA
Greater London
LONDON
A phase I/II, non randomized, multicenter, open-label study of G1XCGD (lentiviral vector transduced CD34+ cells) in patients with X-Linked Chronic Granulomatous Disease - UK
Great Ormond Street Hospital
Molecular and Cellular Immunology Unit
SPAGNA
Cataluña
BARCELONA
An Open-label, Single Arm, Multicenter Study to Broaden Access to Emapalumab, an Anti-Interferon Gamma (Anti-IFN gamma) Monoclonal Antibody, and to Assess its Efficacy, Safety, Impact on Quality of Life, and Long-term Outcome in Pediatric Patients with Primary Hemophagocytic Lymphohistiocytosis (Phase III) - ES
Hospital Universitari Vall d'Hebron
SPAGNA
Madrid
MADRID
An Open-label, Single Arm, Multicenter Study to Broaden Access to Emapalumab, an Anti-Interferon Gamma (Anti-IFN gamma) Monoclonal Antibody, and to Assess its Efficacy, Safety, Impact on Quality of Life, and Long-term Outcome in Pediatric Patients with Primary Hemophagocytic Lymphohistiocytosis (Phase III) - ES
Hospital Infantil Universitario Niño Jesús
SPAGNA
Madrid
MADRID
OLE-IEDAT: Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study (Phase III) - ES
Hospital Universitario La Paz
Servicio de Neurología
BELGIO
ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS
GLYCO-1B: Evaluation of the Safety and Efficacy of Empagliflozin Administration as a Treatment for Neutropenia in Patients With Glycogenosis Type 1b and G6PC3 Deficiency
Cliniques universitaires Saint-Luc - UCLouvain
Cliniques Universitaires Saint-Luc
FRANCIA
ILE-DE-FRANCE
PARIS
SCID2 - Protocol No. 2 of gene therapy for X-linked severe combined immunodeficiency (SCID-X1) using a self retroviral vector.
Hôpital Necker-Enfants Malades
Développement normal et pathologique du système immunitaire
Sperimentazioni cliniche internazionali
FRANCIA
ILE-DE-FRANCE
EVRY
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)
Généthon
Centre de Recherche Généthon
STATI UNITI
New Jersey
JERSEY CITY
STATI UNITI
Pennsylvania
PHILADELPHIA
An Open-label, Parallel-group, Multiple-Dose, Pharmacokinetic and Safety Study of Colchicine Pediatric Formulation in Pediatric and Adult Patients With FMF
Mutual Pharmaceutical Company, Inc.
SVIZZERA
Suisse Alémanique
BASEL
An Open-label Extension Study to Assess Efficacy, Safety and Tolerability of Canakinumab and the Efficacy and Safety of Childhood Vaccinations in Patients With Cryopyrin Associated Periodic Syndromes (CAPS) (Phase III) (Coordination)
Novartis Pharma Services AG
SVIZZERA
Suisse Romande
PLAN-LES-OUATES
FIGHT-HLH: First Targeted Therapy to FIGHT Hemophagocytic Lymphohistiocytosis (HLH): A novel approach to HLH
NovImmune SA
SVIZZERA
Suisse Romande
PLAN-LES-OUATES
FRANCIA
ILE-DE-FRANCE
EVRY
NET4CGD: Gene Therapy for X-linked Chronic Granulomatous Disease (CGD)
Généthon
Centre de Recherche Généthon
ITALIA
EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
OLE-IEDAT: Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study
Institution: Information not provided - IT
REGNO UNITO; GRAN BRETAGNA
South Yorkshire
SHEFFIELD
TAIN: Treatment of Adrenal Insufficiency in neonates- Development of a Hydrocortisone Preparation for the treatment of Adrenal Insufficiency in neonatesand infants
TAIN Project, University of Sheffield
TAIN Project
SVIZZERA
Suisse Romande
ADDRESS: NOT PROVIDED - CH