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National clinical trial(s)
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
AUSTRIA
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
BELGIUM
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
BELGIUM
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
CANADA
Québec
ADDRESS: NOT PROVIDED - CA
A Phase 3, Randomized, Double-Blind Trial of Two Formulations of Setmelanotide (Daily and Weekly) With a Crossover to Open-Label Once Weekly Setmelanotide in Patients With Specific Gene Defects in the Melanocortin-4 Receptor Pathway Who Are Currently on a Stable Dose of the Once Daily Formulation -CA
Institution: Information not provided - CA
FRANCE
BOURGOGNE-FRANCHE-COMTE
DIJON
SESAM: A Phase II Double-blind Multi-center, Placebo-controlled Trial, to Assess the Efficacy and Safety of Alpelisib (BYL719) in Pediatric and Adult Patients With Megalencephaly-CApillary Malformation Polymicrogyria Syndrome (MCAP) -FR
CHU Dijon Bourgogne - Hôpital François Mitterrand
Centre Hospitalier Universitaire Dijon
FRANCE
HAUTS-DE-FRANCE
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
FRANCE
HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
RISE: 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. - Maralixibat Infant Safety Evaluation -FR
Institution: Information not provided - FR
FRANCE
HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
A Phase 3, Randomized, Double-Blind Trial of Two Formulations of Setmelanotide (Daily and Weekly) With a Crossover to Open-Label Once Weekly Setmelanotide in Patients With Specific Gene Defects in the Melanocortin-4 Receptor Pathway Who Are Currently on a Stable Dose of the Once Daily Formulation -FR
Institution: Information not provided - FR
FRANCE
HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
A Randomized, Double-blind, Placebo-controlled, Multi-center, 2-part, Phase 2 Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome -FR
Institution: Information not provided - FR
FRANCE
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
FRANCE
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
FRANCE
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
FRANCE
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
FRANCE
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
FRANCE
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
FRANCE
OCCITANIE
TOULOUSE
Long-term Interventional Follow-up Study up to 4 Years of Age of Children With Prader-Willi Syndrome Included in the OTBB3 Clinical Trial and Comparison With an Untreated Cohort of Children With Prader-Willi Syndrome -FR
CHU de Toulouse - Hôpital des Enfants
Centre de référence du syndrome de Prader-Willi
GERMANY
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
GERMANY
Bayern
MÜNCHEN
SynCoRAS: Improvement of Synaptic Plasticity and Cognitive Function in RAS Pathway Disorders (GeNeRARe study)
TUM Fakultät für Medizin
GERMANY
Bayern
MÜNCHEN
SynCoRAS: Improvement of Synaptic Plasticity and Cognitive Function in RAS Pathway Disorders (GeNeRARe study)
kbo-Kinderzentrum München gemeinnützige GmbH
kbo-Kinderzentrum München
GERMANY
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
GERMANY
Berlin
ADDRESS: NOT PROVIDED - DE
A Phase 3, Randomized, Double-Blind Trial of Two Formulations of Setmelanotide (Daily and Weekly) With a Crossover to Open-Label Once Weekly Setmelanotide in Patients With Specific Gene Defects in the Melanocortin-4 Receptor Pathway Who Are Currently on a Stable Dose of the Once Daily Formulation -DE
Institution: Information not provided - DE
ITALY
EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
Open-label study to evaluate the efficacy and tolerability of orally administered rosiglitazone in patients with progressive bone Fibrodysplasia (FOP)
Institution: Information not provided - IT
ITALY
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 - IT
Institution: Information not provided - IT
ITALY
EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
A Randomized, Double-blind, Placebo-controlled, Multi-center, 2-part, Phase 2 Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome - IT
Institution: Information not provided - IT
ITALY
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
JAPAN
JAPAN
ADDRESS : NOT PROVIDED - JP
Multicenter randomized double-blind comparison test followed by open-label continuous administration test of NPC-12T for Fibrodysplasia Ossificans Progressiva
Institution: Information not provided - JP
NETHERLANDS
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
NETHERLANDS
Zuid-Holland
ROTTERDAM
N- acetylcysteine treatment for skin picking in children and young adults with PWS: a randomized, controlled, cross-over trial
Stichting Kind en Groei
SPAIN
Comunidad Valenciana
VALENCIA
PIVOINE: Rollover Study; Multicentre, Phase III, Open-label Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged >=14 Years with Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed Study PVO-1A-301 or PVO-1A-202/PVO-1A-204 [...] -ES
Hospital Universitario y Politécnico La Fe
SPAIN
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
SPAIN
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
SPAIN
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
SPAIN
Madrid
ADDRESS: NOT PROVIDED - ES
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) -ES
Institution: Information not provided - ES
SPAIN
Madrid
ADDRESS: NOT PROVIDED - ES
A Randomized, Double-blind, Placebo-controlled, Multi-center, 2-part, Phase 2 Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome -ES
Institution: Information not provided - ES
SPAIN
Madrid
ADDRESS: NOT PROVIDED - ES
Progress: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva -ES
Institution: Information not provided - ES
SPAIN
Madrid
MADRID
A Phase 3 Multi-Center, One-Year, Open-Label study of Setmelanotide in Pediatric Patients Aged 2 to <6 years of age with Rare Genetic Causes of Obesity -ES
Hospital Infantil Universitario Niño Jesús
Servicio de Endocrinología, Crecimiento y Metabolismo
SPAIN
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
SPAIN
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
SWEDEN
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
UNITED KINGDOM
Greater London
ADDRESS: NOT PROVIDED - GB
A Phase 3, Randomized, Double-Blind Trial of Two Formulations of Setmelanotide (Daily and Weekly) With a Crossover to Open-Label Once Weekly Setmelanotide in Patients With Specific Gene Defects in the Melanocortin-4 Receptor Pathway Who Are Currently on a Stable Dose of the Once Daily Formulation -CA
Institution: Information not provided - GB
UNITED KINGDOM
Greater London
ADDRESS: NOT PROVIDED - GB
A Phase 3, Randomized, Double-Blind Trial of Two Formulations of Setmelanotide (Daily and Weekly) With a Crossover to Open-Label Once Weekly Setmelanotide in Patients With Specific Gene Defects in the Melanocortin-4 Receptor Pathway Who Are Currently on a Stable Dose of the Once Daily Formulation -GB
Institution: Information not provided - GB
UNITED KINGDOM
Greater London
LONDON
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
UNITED KINGDOM
Merseyside
LIVERPOOL
Effects of exenatide on appetite and ghrelin levels in patients with Prader-Willi Syndrome
Fazakerley Hospital
University Hospital Aintree - Diabetes and Endocrinology
UNITED KINGDOM
West Midlands
BIRMINGHAM
Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity
University Hospital Birmingham
Marshfield Clinic
UNITED STATES
Ohio
CINCINNATI
Quercetin in Children With Fanconi Anemia; a Pilot Study (Phase 1) - US
Cincinnati Children's Hospital Medical Center
Department of Pediatry
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Phase 2 Study: Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Children and Adolescents With Prader-Willi Syndrome
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Evaluating the Neurophysiologic and Clinical Effects of Single-Dose Acamprosate, Lovastatin, Minocycline and Placebo in Fragile X Syndrome
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Effects of AFQ056 on Language Learning in Young Children With Fragile X Syndrome (FXS)
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
A Multicenter Extension Study to Evaluate the Long-Term Safety and Durability of the Therapeutic Effect of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome
Institution: Information not provided - US
SPAIN
Madrid
ADDRESS: NOT PROVIDED - ES
A phase II, randomized, double-blind, placebo-controlled, parallel group study to evaluate the safety, efficacy, and pharmacodynamics of 52 weeks of treatment with basmisanil in children with dup15q syndrome -ES
Institution: Information not provided - ES
Multinational clinical trial(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
HUNGARY
Dél-Dunántúl
ADDRESS: NOT PROVIDED - HU
A Randomized, Double-blind, Placebo-controlled, Multi-center, 2-part, Phase 2 Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome.
Institution: Information not provided - HU
NETHERLANDS
Utrecht
ADDRESS: NOT PROVIDED - NL
STOPFOP: Saracatinib Trial TO Prevent FOP
Institution: Information not provided - NL
NETHERLANDS
Utrecht
ADDRESS: NOT PROVIDED - NL
Global Growth Hormone Study in Adults With Prader-Willi Syndroom
Institution: Information not provided - NL
SWITZERLAND
Suisse Romande
ADDRESS: NOT PROVIDED - CH
Sequential, Two-period Study to Assess the Pharmacokinetics, Safety & Tolerability of Single and Multiple Oral Doses of AFQ056 in Patients With FXS (Fragile X Syndrome) Aged 5-11 Years (Cohort 1) and 3-4 Years (Cohort 2) (Phase I) (Coordination)
Institution: Information not provided - CH
SWITZERLAND
Suisse Romande
ADDRESS: NOT PROVIDED - CH
An Open-label Study to Evaluate the Long-term Safety and Tolerability of AFQ056 in Adolescent Patients With Fragile X Syndrome (Phase II-III) (Coordination)
Institution: Information not provided - CH
SWITZERLAND
Suisse Romande
ADDRESS: NOT PROVIDED - CH
A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy and Safety of AFQ056 in Adolescent Patients With Fragile X Syndrome (Phase II-III) (Coordination)
Institution: Information not provided - CH
SWITZERLAND
Suisse Romande
ADDRESS: NOT PROVIDED - CH
An Open-label Study to Evaluate the Long-term Safety, Tolerability and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome (Phase II) (Coordination)
Institution: Information not provided - CH
SWITZERLAND
Suisse Romande
ADDRESS: NOT PROVIDED - CH
A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate AFQ056 in Adult Patients With Fragile X Syndrome (Phase II) (Coordination)
Institution: Information not provided - CH
SWITZERLAND
Suisse Romande
ADDRESS: NOT PROVIDED - CH
A Randomized, Double-blind, 12-week, Parallel Group, Placebo-controlled Study of Efficacy and Safety of RO4917523 in Patients With Fragile X Syndrome (Phase II) (Coordination)
Institution: Information not provided - CH
UNITED KINGDOM
Greater London
LONDON
ZEPHYR: A Phase 2b/3 Study to Evaluate the Safety, Tolerability, and Effects of Livoletide (AZP-531), an Unacylated Ghrelin Analog, on Food-related Behaviors in Patients With Prader-Willi Syndrome
Millendo Therapeutics Ltd
UNITED STATES
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.
UNITED STATES
Texas
HOUSTON
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.
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
RISE: 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. - Maralixibat Infant Safety Evaluation.
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Phase III Clinical Trial for the Treatment of Myeloid Leukemia in Children with Down Syndrome 2018 - ML-DS 2018
Institution: Information not provided - US
UNITED STATES
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
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US