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Sperimentazioni cliniche nazionali

NF1-EXCEL: The Effect of Lamotrigine on Cognitive Deficits Associated With Neurofibromatosis Type 1: a Phase II Randomized Controlled Multi-centre Trial - BE UZ Leuven - Campus Gasthuisberg Department of Human Genetics

Neuropsychological Impairment and Quality of Life in Neurofibromatosis Type 1 - FR CHU de Nantes CHU Nantes

Safety of Abatacept in patients with interstitial lung disease and common variable immunodeficiency (CVID) and related disease Institution: Information not provided - DE

NF1-EXCEL: The Effect of Lamotrigine on Cognitive Deficits Associated With Neurofibromatosis Type 1: a Phase II Randomized Controlled Multi-centre Trial - NL Erasmus MC - Erasmus Medisch Centrum Afdeling Kinderneurologie

NF1-EXCEL: The Effect of Lamotrigine on Cognitive Deficits Associated With Neurofibromatosis Type 1: a Phase II Randomized Controlled Multi-centre Trial - NL Erasmus MC - Erasmus Medisch Centrum Afdeling Neurowetenschappen

NF1-EXCEL: The Effect of Lamotrigine on Cognitive Deficits Associated With Neurofibromatosis Type 1: a Phase II Randomized Controlled Multi-centre Trial - BE Erasmus MC - Erasmus Medisch Centrum

Sorafenib in NF2: Investigation of the intra-tumoural concentration and activity of sorafenib in cutaneous schwannomas I T T C Building Peninsula Clinical Research Facility

A Clinical Trial Of The Intra-Tumoural Concentration And Activity Of Nilotinib In Intra-Cutaneous Schwannomas (Phase IV) Plymouth Derriford Hospital Plymouth Hospitals NHS Trust Headquarters

A Paediatric Phase I/II Study Of Intermittent Dosing Of The Mek-1 Inhibitor Selumetinib In Children With Neurofibromatosis Type-1 And Inoperable Plexiform Neurofibroma And/Or Progressive Optic Pathway Glioma GOSH NHS Foundatin Trust Great Ormond Street Hospital

Double-Blind, Randomized, Vehicle-Controlled Proof of Concept Clinical Trial of Patidegib Gel 2%, 4%, and Vehicle to Decrease the Number of Surgically Eligible Basal Cell Carcinomas in Gorlin Syndrome Patients Institution: Information not provided - UK

A Phase 3, Multicenter, Open-Label Extension Study of Patidegib Topical Gel, 2% in Subjects with Gorlin Syndrome (Basal Cell Nevus Syndrome) - ES Institution: Information not provided - ES

CREATE (EORTC-90101): Cross-tumoral Phase 2 clinical trial exploring Crizotinib (PF-02341066) in patients with advanced tumor induced by causal alterations of ALK and/or MET - BE Institution: Information not provided - BE

MERGE - MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study - BE Institution: Information not provided - BE

EXIST-LT: Long-term follow-up study to monitor the growth and development of pediatric patients previously treated with everolimus in study CRAD001M2301- BE Universitair Ziekenhuis Brussel Pediatric neurology / Neurologie Kinderen

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

Phase III study to evaluate GW786034 (Pazopanib) In Metastatic Renal Cell Carcinoma Haematology and Oncology Clinic - Tartu University Hospital Haematology and Oncology Clinic

EXIST-2: A Randomized, Double-blind, Placebo-controlled Study of RAD0001 in the Treatment of Angiomyolipoma in Patients With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) - ES (Phase III) Institution: Information not provided - FR

MERGE - MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study - FR Institution: Information not provided - FR

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

FancoMob : Pilot Study Assessing the Feasibility of CD34+ Cells Mobilization and Collection After Treatment With G-CSF and Plerixafor in Patients With Fanconi Anemia for Subsequent Treatment by Gene Therapy (Phase I-II) Hôpital Necker-Enfants Malades Service d'Hématologie adulte

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

Effectiveness and safety of mTOR-inhibitor in patients with tuberous sclerosis complex-KD Universitätsklinikum Heidelberg

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

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

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

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

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

Studio di fase 1/2 su ARQ 092 in pazienti affetti da malattie da iperaccrescimento e/o anomalie vascolari con alterazioni genetiche del pathway PI3K/AKT IRCCS Ospedale Pediatrico Bambino Gesù - SEDE GIANICOLO Ambulatorio Polispecialistico per le Malattie Rare

Studio clinico multicentrico, di fase 2, open label, di estensione per valutare la sicurezza e la tollerabilita' di FBS0701 (SSP-004184) in bambini, adolescenti ed adulti con sovraccarico di ferro trasfusione-dipendente Ospedali Galliera S.S.D. Ematologia

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

RATE trial: Randomised open label cross-over study into the efficacy of rapamycin in children with Tuberous Sclerosis Complex with intractable epilepsy (phase II/III trial) Erasmus MC - Erasmus Medisch Centrum Afdeling Kinderneurologie

RAPIT trial: Efficacy of RAD001/everolimus in Autism and NeuroPsychological deficits in children with TSC (phase II/III trial) Erasmus MC - Erasmus Medisch Centrum Afdeling Kinderneurologie

An Open-Label Phase 2 Study to Evaluate PT2977 for the Treatment of Von Hippel Lindau Disease-Associated Renal Cell Carcinoma-GB Addenbrooke's Hospital Addenbrookes Hospital

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

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

CREATE (EORTC 90101): Cross-tumoral Phase 2 clinical trial exploring Crizotinib (PF-02341066) in patients with advanced tumours induced by causal alterations of either ALK or MET - UK UCLH NHS Foundation Trust - University College Hospital The London Sarcoma Service

FANCOLEN-II: A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemia Subtype A - ES Hospital Universitari Vall d'Hebron

FANCOLEN-1: Clinical Trial Phase I/II to evaluate the safety and efficacy of the infusion of autologous CD34+ cells transduced with a lentiviral vector carrying the FANCA gene (orphan drug) for patients with Fanconi Anemia Subtype A Institution: Information not provided - ES

MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study (Phase II) - ES Hospital Universitario La Paz

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

Quercetin in Children With Fanconi Anemia; a Pilot Study (Phase 1/2) - US Cincinnati Children's Hospital Medical Center Department of Pediatry

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

Randomised, Double-blind, Placebo-controlled Study of the Efficacy, Safety and Tolerability of EPA-FFA Gastro-resistant Capsules, in Patients With Familial Adenomatous Polyposis (FAP) - FR Hospices Civils de Lyon

Randomised, Double-blind, Placebo-controlled Study of the Efficacy, Safety and Tolerability of EPA-FFA Gastro-resistant Capsules, in Patients With Familial Adenomatous Polyposis (FAP) - FR CHU Paris Seine-Saint-Denis - Hôpital Avicenne

Randomised, Double-blind, Placebo-controlled Study of the Efficacy, Safety and Tolerability of EPA-FFA Gastro-resistant Capsules, in Patients With Familial Adenomatous Polyposis (FAP) - FR CHU Paris Est - Hôpital Saint-Antoine

A Double-Blind, Randomized, Phase III Trial of the Safety and Efficacy of CPP-1X/Sulindac Compared With CPP-1X, Sulindac as Single Agents in Patients with Familial Adenomatous Polyposis (FAP) - UK St Mary's Hospital Centre for Genomic Medicine

A Double-Blind, Randomized, Phase III Trial of the Safety and Efficacy of CPP-1X/Sulindac Compared With CPP-1X, Sulindac as Single Agents in Patients with Familial Adenomatous Polyposis (FAP) - UK Newcastle University Institute of Genetic Medicine

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES Hospital Universitario Virgen del Rocío

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES Hospital Clínico Universitario "Lozano Blesa" de Zaragoza

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES Hospital Comarcal de Inca

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES Hospital Universitario de Canarias

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES Hospital Universitario Río Hortega

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES Hospital Clínic de Barcelona

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES Corporación Sanitaria Parc Taulí. Hospital de Sabadell Hospital de Sabadell

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES Hospital General Universitario de Alicante

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES Complexo Hospitalario Universitario de Ourense

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES Hospital Provincial de Pontevedra

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES Hospital Álvaro Cunqueiro

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES Hospital Universitario La Paz

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES Hospital Universitario de Móstoles

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES Clínica Universidad de Navarra

Sperimentazioni cliniche internazionali

A randomized, double-blinded, regimen controlled, phase II, multicenter study to assess the efficacy and safety of two different Vismodegib regimens in patients with multiple Basal Cell Carcinoma Institution: Information not provided - CH

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

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

MERGE - MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study Mirum Pharmaceuticals, Inc.

CaspaCide TCR &alpha; &beta; haplo HSCT: Follow-up of Phase 1/2 Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR &alpha; &beta;+T Cells in Pediatric Patients Affected by Hematological Disorders Bellicum Pharmaceuticals, Inc.

A Phase 2, Open-label, Multicentre, Extension Safety and Tolerability Study for Transfusionally Iron Overloaded Children, Adolescents and Adults Using SSP-004184 (SPD602) Institution: Information not provided - US

EXIST-2: Randomized, Double-blind, Placebo-controlled Study of RAD0001 in the Treatment of Angiomyolipoma in Patients With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) Novartis International AG

Randomised, Double-blind, Placebo-controlled Study of the Efficacy, Safety and Tolerability of EPA-FFA Gastro-resistant Capsules, in Patients With Familial Adenomatous Polyposis (FAP) Institution: Information not provided - DE

Phase III Trial of the Safety and Efficacy of Eflornithine Combined With Sulindac Compared to Eflornithine, Sulindac as Single Agents in Patients With Familial Adenomatous Polyposis Institution: Information not provided - US

NF1-EXCEL: The Effect of Lamotrigine on Cognitive Deficits Associated With Neurofibromatosis Type 1: a Phase II Randomized Controlled Multi-centre Trial - BE
UZ Leuven - Campus Gasthuisberg

Department of Human Genetics

Neuropsychological Impairment and Quality of Life in Neurofibromatosis Type 1 - FR
CHU de Nantes

CHU Nantes

Safety of Abatacept in patients with interstitial lung disease and common variable immunodeficiency (CVID) and related disease
Institution: Information not provided - DE

NF1-EXCEL: The Effect of Lamotrigine on Cognitive Deficits Associated With Neurofibromatosis Type 1: a Phase II Randomized Controlled Multi-centre Trial - NL
Erasmus MC - Erasmus Medisch Centrum

Afdeling Kinderneurologie

NF1-EXCEL: The Effect of Lamotrigine on Cognitive Deficits Associated With Neurofibromatosis Type 1: a Phase II Randomized Controlled Multi-centre Trial - NL
Erasmus MC - Erasmus Medisch Centrum

Afdeling Neurowetenschappen

NF1-EXCEL: The Effect of Lamotrigine on Cognitive Deficits Associated With Neurofibromatosis Type 1: a Phase II Randomized Controlled Multi-centre Trial - BE
Erasmus MC - Erasmus Medisch Centrum

Sorafenib in NF2: Investigation of the intra-tumoural concentration and activity of sorafenib in cutaneous schwannomas
I T T C Building

Peninsula Clinical Research Facility

A Clinical Trial Of The Intra-Tumoural Concentration And Activity Of Nilotinib In Intra-Cutaneous Schwannomas (Phase IV)
Plymouth Derriford Hospital

Plymouth Hospitals NHS Trust Headquarters

A Paediatric Phase I/II Study Of Intermittent Dosing Of The Mek-1 Inhibitor Selumetinib In Children With Neurofibromatosis Type-1 And Inoperable Plexiform Neurofibroma And/Or Progressive Optic Pathway Glioma
GOSH NHS Foundatin Trust

Great Ormond Street Hospital

Double-Blind, Randomized, Vehicle-Controlled Proof of Concept Clinical Trial of Patidegib Gel 2%, 4%, and Vehicle to Decrease the Number of Surgically Eligible Basal Cell Carcinomas in Gorlin Syndrome Patients
Institution: Information not provided - UK

A Phase 3, Multicenter, Open-Label Extension Study of Patidegib Topical Gel, 2% in Subjects with Gorlin Syndrome (Basal Cell Nevus Syndrome) - ES
Institution: Information not provided - ES

CREATE (EORTC-90101): Cross-tumoral Phase 2 clinical trial exploring Crizotinib (PF-02341066) in patients with advanced tumor induced by causal alterations of ALK and/or MET - BE
Institution: Information not provided - BE

MERGE - MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study - BE
Institution: Information not provided - BE

EXIST-LT: Long-term follow-up study to monitor the growth and development of pediatric patients previously treated with everolimus in study CRAD001M2301- BE
Universitair Ziekenhuis Brussel

Pediatric neurology / Neurologie Kinderen

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

Phase III study to evaluate GW786034 (Pazopanib) In Metastatic Renal Cell Carcinoma
Haematology and Oncology Clinic - Tartu University Hospital

Haematology and Oncology Clinic

EXIST-2: A Randomized, Double-blind, Placebo-controlled Study of RAD0001 in the Treatment of Angiomyolipoma in Patients With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) - ES (Phase III)
Institution: Information not provided - FR

MERGE - MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study - FR
Institution: Information not provided - FR

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

FancoMob : Pilot Study Assessing the Feasibility of CD34+ Cells Mobilization and Collection After Treatment With G-CSF and Plerixafor in Patients With Fanconi Anemia for Subsequent Treatment by Gene Therapy (Phase I-II)
Hôpital Necker-Enfants Malades

Service d'Hématologie adulte

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

Effectiveness and safety of mTOR-inhibitor in patients with tuberous sclerosis complex-KD
Universitätsklinikum Heidelberg

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

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

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

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

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

Studio di fase 1/2 su ARQ 092 in pazienti affetti da malattie da iperaccrescimento e/o anomalie vascolari con alterazioni genetiche del pathway PI3K/AKT
IRCCS Ospedale Pediatrico Bambino Gesù - SEDE GIANICOLO

Ambulatorio Polispecialistico per le Malattie Rare

Studio clinico multicentrico, di fase 2, open label, di estensione per valutare la sicurezza e la tollerabilita' di FBS0701 (SSP-004184) in bambini, adolescenti ed adulti con sovraccarico di ferro trasfusione-dipendente
Ospedali Galliera

S.S.D. Ematologia

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

RATE trial: Randomised open label cross-over study into the efficacy of rapamycin in children with Tuberous Sclerosis Complex with intractable epilepsy (phase II/III trial)
Erasmus MC - Erasmus Medisch Centrum

Afdeling Kinderneurologie

RAPIT trial: Efficacy of RAD001/everolimus in Autism and NeuroPsychological deficits in children with TSC (phase II/III trial)
Erasmus MC - Erasmus Medisch Centrum

Afdeling Kinderneurologie

An Open-Label Phase 2 Study to Evaluate PT2977 for the Treatment of Von Hippel Lindau Disease-Associated Renal Cell Carcinoma-GB
Addenbrooke's Hospital

Addenbrookes Hospital

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

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

CREATE (EORTC 90101): Cross-tumoral Phase 2 clinical trial exploring Crizotinib (PF-02341066) in patients with advanced tumours induced by causal alterations of either ALK or MET - UK
UCLH NHS Foundation Trust - University College Hospital

The London Sarcoma Service

FANCOLEN-II: A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemia Subtype A - ES
Hospital Universitari Vall d'Hebron

FANCOLEN-1: Clinical Trial Phase I/II to evaluate the safety and efficacy of the infusion of autologous CD34+ cells transduced with a lentiviral vector carrying the FANCA gene (orphan drug) for patients with Fanconi Anemia Subtype A
Institution: Information not provided - ES

MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study (Phase II) - ES
Hospital Universitario La Paz

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

Quercetin in Children With Fanconi Anemia; a Pilot Study (Phase 1/2) - US
Cincinnati Children's Hospital Medical Center

Department of Pediatry

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

Randomised, Double-blind, Placebo-controlled Study of the Efficacy, Safety and Tolerability of EPA-FFA Gastro-resistant Capsules, in Patients With Familial Adenomatous Polyposis (FAP) - FR
Hospices Civils de Lyon

Randomised, Double-blind, Placebo-controlled Study of the Efficacy, Safety and Tolerability of EPA-FFA Gastro-resistant Capsules, in Patients With Familial Adenomatous Polyposis (FAP) - FR
CHU Paris Seine-Saint-Denis - Hôpital Avicenne

Randomised, Double-blind, Placebo-controlled Study of the Efficacy, Safety and Tolerability of EPA-FFA Gastro-resistant Capsules, in Patients With Familial Adenomatous Polyposis (FAP) - FR
CHU Paris Est - Hôpital Saint-Antoine

A Double-Blind, Randomized, Phase III Trial of the Safety and Efficacy of CPP-1X/Sulindac Compared With CPP-1X, Sulindac as Single Agents in Patients with Familial Adenomatous Polyposis (FAP) - UK
St Mary's Hospital

Centre for Genomic Medicine

A Double-Blind, Randomized, Phase III Trial of the Safety and Efficacy of CPP-1X/Sulindac Compared With CPP-1X, Sulindac as Single Agents in Patients with Familial Adenomatous Polyposis (FAP) - UK
Newcastle University

Institute of Genetic Medicine

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES
Hospital Universitario Virgen del Rocío

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES
Hospital Clínico Universitario "Lozano Blesa" de Zaragoza

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES
Hospital Comarcal de Inca

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES
Hospital Universitario de Canarias

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES
Hospital Universitario Río Hortega

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES
Hospital Clínic de Barcelona

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES
Corporación Sanitaria Parc Taulí. Hospital de Sabadell

Hospital de Sabadell

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES
Hospital General Universitario de Alicante

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES
Complexo Hospitalario Universitario de Ourense

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES
Hospital Provincial de Pontevedra

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES
Hospital Álvaro Cunqueiro

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES
Hospital Universitario La Paz

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES
Hospital Universitario de Móstoles

Randomised, double-blind, placebo-controlled study of the, efficacy, safety and tolerability of EPA-FFA gastro-resistant capsules, in patients with familial adenomatous polyposis (FAP) (Phase III) - ES
Clínica Universidad de Navarra

A randomized, double-blinded, regimen controlled, phase II, multicenter study to assess the efficacy and safety of two different Vismodegib regimens in patients with multiple Basal Cell Carcinoma
Institution: Information not provided - CH

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

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

MERGE - MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study
Mirum Pharmaceuticals, Inc.

CaspaCide TCR α β haplo HSCT: Follow-up of Phase 1/2 Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR α β+T Cells in Pediatric Patients Affected by Hematological Disorders
Bellicum Pharmaceuticals, Inc.

A Phase 2, Open-label, Multicentre, Extension Safety and Tolerability Study for Transfusionally Iron Overloaded Children, Adolescents and Adults Using SSP-004184 (SPD602)
Institution: Information not provided - US

EXIST-2: Randomized, Double-blind, Placebo-controlled Study of RAD0001 in the Treatment of Angiomyolipoma in Patients With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)
Novartis International AG

Randomised, Double-blind, Placebo-controlled Study of the Efficacy, Safety and Tolerability of EPA-FFA Gastro-resistant Capsules, in Patients With Familial Adenomatous Polyposis (FAP)
Institution: Information not provided - DE

Phase III Trial of the Safety and Efficacy of Eflornithine Combined With Sulindac Compared to Eflornithine, Sulindac as Single Agents in Patients With Familial Adenomatous Polyposis
Institution: Information not provided - US