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Nationale klinische studie(s)
BELGIË
ARRONDISSEMENT BRUSSELS-CAPITAL
ADDRESS: 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
BELGIË
ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS
RISE/MRX-801: Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome - BE
Cliniques universitaires Saint-Luc - UCLouvain
Service de gastro-entérologie et hépatologie pédiatrique
CANADA
Québec
ADDRESS: NOT PROVIDED - CA
MOVE Trial: A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP) -GB
Institution: Information not provided - CA
DUITSLAND
Bayern
GARMISCH-PARTENKIRCHEN
STOPFOP: Saracatinib Trial TO Prevent FOP. A 6-month double blind randomized controlled trial of AZD0530 versus placebo, followed by a 12 month open label extension phase -DE
Klinikum Garmisch-Partenkirchen GmbH
Abteilung für Kinder- & Jugendmedizin
DUITSLAND
Berlin
ADDRESS: NOT PROVIDED - DE
MOVE TRIAL: A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP) -DE-
Institution: Information not provided - DE
FRANKRIJK
AUVERGNE-RHONE-ALPES
ADDRESS: 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
FRANKRIJK
ILE-DE-FRANCE
PARIS
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
FRANKRIJK
ILE-DE-FRANCE
PARIS
A Phase 2 Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of a RARy-Specific Agonist (Palovarotene) in the Treatment of Preosseous Flare-ups in Subjects With Fibrodysplasia Ossificans Progressiva (FOP) (Phase II) - FR
Hôpital Necker-Enfants Malades
Service de Génétique Moléculaire
FRANKRIJK
ILE-DE-FRANCE
PARIS
A Phase 2, Open-Label Extension, Efficacy and Safety Study of a RARy Specific Agonist (Palovarotene) in the Treatment of Preosseous Flare-ups in Subjects With Fibrodysplasia Ossificans Progressiva (FOP) - FR
Hôpital Necker-Enfants Malades
Service de Génétique Moléculaire
FRANKRIJK
ILE-DE-FRANCE
PARIS
A Phase 2, Open-Label, Efficacy and Safety Study of an RARy Specific Agonist (Palovarotene) to Prevent Heterotopic Ossification in Subjects With Fibrodysplasia Ossificans Progressiva (FOP)
Hôpital Necker-Enfants Malades
Service de Génétique Moléculaire
FRANKRIJK
ILE-DE-FRANCE
PARIS
ACTHYF: Efficacy Assessment of Systematic Treatment With Folinic Acid and Thyroid Hormone on Psychomotor Development of Down Syndrome Young Children - FR
Institut Jérôme Lejeune
FRANKRIJK
OCCITANIE
TOULOUSE
OXYJEUNE : Effects of Intranasal Administrations of Oxytocin on Behavioural Troubles, Hyperphagia and Social Skills in Children With Prader-Willi Syndrome Aged From 3 to 12 Years (Phase III)
CHU de Toulouse - Hôpital des Enfants
Centre de référence du syndrome de Prader-Willi
ITALIË
EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
Follow-up of phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR alfa-beta+ T cells in pediatric patients affected by hematological disorders
Institution: Information not provided - IT
ITALIË
LAZIO
ROMA
A Phase 1/2 Study of ARQ 092 in Patients with Overgrowth Diseases and/or Vascular Anomalies with Genetic Alterations of the PI3K/AKT Pathway
IRCCS Ospedale Pediatrico Bambino Gesù - SEDE GIANICOLO
Ambulatorio Polispecialistico per le Malattie Rare
ITALIË
SICILIA
MESSINA
Randomized, Placebo-controlled, Cross-over, Double-blind Study of a Metabolic Support Therapy With Q10 Ubiquinol and a Multivitamin B and E Complex in Two Cohorts of Patients With Idiopathic and Syndromic Autism (Phelan-McDermid Syndrome)
Università degli Studi di Messina
NEDERLAND
Zuid-Holland
ROTTERDAM
Dutch Growth Hormone Cohort study in children with Prader-Willi syndrome: Long-term effects on growth, body composition, activity level and psychosocial development
Stichting Kind en Groei
NEDERLAND
Zuid-Holland
ROTTERDAM
Behandeling met N-acetylcysteïne voor skin picking gedrag in kinderen en jongvolwassenen met PWS: een gerandomiseerde, gecontroleerde, cross-over studie
Stichting Kind en Groei
OOSTENRIJK
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
OOSTENRIJK
WIEN
ADDRESS: NOT PROVIDED - AT
Phase III Clinical Trial for CPX-351 in Myeloid Leukemia in Children with Down Syndrome 2018 - AT
Institution: Information not provided - AT
SPANJE
Comunidad Valenciana
VALENCIA
MOVE Trial: A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP) - ES
Hospital Universitario y Politécnico La Fe
Unidad de Reumatología Pediátrica
SPANJE
Madrid
ADDRESS: NOT PROVIDED - ES
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
SPANJE
Madrid
ADDRESS: NOT PROVIDED - ES
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
Institution: Information not provided - ES
SPANJE
Madrid
ADDRESS: NOT PROVIDED - ES
FALKON: A Phase 2, two-part, placebo-controlled, parallel-group, double-blind study to assess the efficacy and safety of 2 dosage regimens of oral IPN60130 for the treatment of fibrodysplasia ossificans progressiva in male and female participants 5 years of age and older - ES
Institution: Information not provided - ES
SPANJE
Madrid
ADDRESS: NOT PROVIDED - ES
Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of autologous CD34+ cells transduced with a lentiviral vector carrying the FANCA gene (orphan drug) in patients with Fanconi Anaemia Subtype A - ES
Institution: Information not provided - ES
SPANJE
Madrid
MADRID
Setmelanotide (RM-493): Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity - ES
Hospital Infantil Universitario Niño Jesús
Servicio de Endocrinología, Crecimiento y Metabolismo
SPANJE
Madrid
MADRID
An open clinical trial, phase II to evaluate the efficacy and safety of the use of eltrombopag in children and adolescents with Fanconi Anemia - ES
Hospital Infantil Universitario Niño Jesús
Servicio de Hematología y Hemoterapia
SPANJE
Madrid
MADRID
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 - ES
Hospital Universitario La Paz
USA
Ohio
CINCINNATI
Quercetin in Children With Fanconi Anemia; a Pilot Study (Phase 1/2) - US
Cincinnati Children's Hospital Medical Center
Department of Pediatry
VERENIGD KONINKRIJK
Greater London
LONDON
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-GB
GOSH NHS Foundatin Trust
Great Ormond Street Hospital
VERENIGD KONINKRIJK
Merseyside
LIVERPOOL
Effects of exenatide on appetite and ghrelin levels in patients with Prader-Willi Syndrome
Fazakerley Hospital
University Hospital Aintree - Diabetes and Endocrinology
VERENIGD KONINKRIJK
West Midlands
BIRMINGHAM
Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity
University Hospital Birmingham
Marshfield Clinic
ZWEDEN
Region Stockholm
ADDRESS: NOT PROVIDED - SE
MOVE Trial: A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP) - SE
Institution: Information not provided - SE
ZWITSERLAND
Suisse Romande
GENÈVE
Effects of Modulation of the Dopaminergic System Using Methylphenidate on Memory and Executive Processes in Individuals With 22q11.2 Deletion Syndrome
UNIGE Université de Genève
Service Médico-Pédagogique Département de Psychiatrie
ZWITSERLAND
Suisse Romande
GENÈVE
Effects of Modulation of the Dopaminergic System Using Risperidone on Memory and Executive Processes in Individuals With 22q11.2 Deletion Syndrome
UNIGE Université de Genève
Service Médico-Pédagogique Département de Psychiatrie
Multinationale klinische studie(s)
CANADA
Québec
ADDRESS: NOT PROVIDED - CA
MOVE Trial: A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP)
Institution: Information not provided - CA
SPANJE
Madrid
MADRID
EUROFANCOLEN: Phase I/II Gene Therapy Trial of Fanconi anemia patients with a new Orphan Drug consisting of a lentiviral vector carrying the FANCA gene: A Coordinated International Action
CIEMAT - Centro de Investigaciones Energéticas Medioambientales y Tecnológicas
Terapias innovadoras en el sistema hematopoyético
USA
California
FOSTER CITY
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.
USA
Texas
HOUSTON
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.
USA
Washington
ADDRESS: NOT PROVIDED - US
EPIK-P2: A Phase II Double-blind Study With an Upfront, 16-week Randomized, Placebo-controlled Period, to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (PROS)
Institution: Information not provided - US
VERENIGD KONINKRIJK
Greater London
LONDON