Not applicableStudy completedNCT05548855What this trial is testingNatural History of Danon DiseaseWho this might be right forDanon Disease Rocket Pharmaceuticals Inc. 59
Early research (Phase 1)Study completedNCT02013258What this trial is testingOxytocin Trial in Prader-Willi SyndromeWho this might be right forPrader Willi Syndrome University of Florida 24
Not applicableStudy completedNCT02033642What this trial is testingA Family-Based Weight Loss Intervention for Youth With Intellectual DisabilityWho this might be right forObesity University of Massachusetts, Boston 31
Not applicableLooking for participantsNCT06019182What this trial is testingMEHMO Natural History and BiomarkersWho this might be right forIntellectual DisabilityEpilepsyHypogonadisms+3 more Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 150
Testing effectiveness (Phase 2)Study completedNCT00001262What this trial is testingCopper Histidine Therapy for Menkes DiseasesWho this might be right forKinky Hair Syndrome Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 60
Not applicableStudy completedNCT04463069What this trial is testingSchool-based Physical Activity Intervention for Obesity Among Adolescents With Intellectual Disability in Hong KongWho this might be right forObesity Hong Kong Baptist University 86
Large-scale testing (Phase 3)Ended earlyNCT06828861What this trial is testingARD-101 for Treatment of PWS: The Hunger Elimination or Reduction Objective TrialWho this might be right forHyperphagiaPrader-Willi Syndrome Aardvark Therapeutics, Inc. 68
Large-scale testing (Phase 3)Study completedNCT04697381What this trial is testingStudy of the Efficacy and Safety of Somatropin in Japanese Participants With PWSWho this might be right forPrader-Willi Syndrome Pfizer 33
Not applicableStudy completedNCT03582449What this trial is testingIntensive Pharmacovigilance Program for Elaprase (SHP ELA-701)Who this might be right forHunter Syndrome Shire 5
Large-scale testing (Phase 3)Active Not RecruitingNCT02616484What this trial is testingTrial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:Who this might be right forPyruvate Dehydrogenase Complex Deficiency Saol Therapeutics Inc 34
Testing effectiveness (Phase 2)Active Not RecruitingNCT04251026What this trial is testingTividenofusp Alfa (DNL310) in Pediatric Participants With Hunter SyndromeWho this might be right forMucopolysaccharidosis II Denali Therapeutics Inc. 47
Early research (Phase 1)Study completedNCT02567357What this trial is testingParents Resources for Decreasing the Incidence of Change Triggered Temper OutburstsWho this might be right forChild Behavior Queen's University, Belfast 80
Not applicableStudy completedNCT03553706What this trial is testingIndicators of Growth, Nutritional Status and Comorbide Disorders of Newborns With Down SyndromeWho this might be right forDown Syndrome,Auxological Indexes, Auxological Parametars, Intrauterine Growth Restriction University Hospital of Split 141
Testing effectiveness (Phase 2)WithdrawnNCT02226458What this trial is testingAn Exploratory Open Label Study of EPI-743 (Vincerinone TM) in Children With Autism Spectrum DisorderWho this might be right forAutism Spectrum Disorder Edison Pharmaceuticals Inc
Not applicableLooking for participantsNCT04278404What this trial is testingPharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)Who this might be right forCoronavirus Infection (COVID-19)Pulmonary Arterial HypertensionUrinary Tract Infections in Children+22 more Duke University 5,000
Not applicableStudy completedNCT00551343What this trial is testingGut Derived Hormones, Body Composition and Metabolism in Prader-Willi SyndromeWho this might be right forPrader-Willi Syndrome Garvan Institute of Medical Research 20
Testing effectiveness (Phase 2)Study completedNCT03128593What this trial is testingJR-141 in Patients With Mucopolysaccharidosis Type IIWho this might be right forMucopolysaccharidosis II JCR Pharmaceuticals Co., Ltd. 14
Not applicableStudy completedNCT01449240What this trial is testingCollection and Study of Cerebrospinal Fluid in Patients With Hunter SyndromeWho this might be right forHunter Syndrome Shire 10
Not applicableStudy completedNCT04102839What this trial is testingPWS Outcomes Assessment StudyWho this might be right forPrader-Willi Syndrome Soleno Therapeutics, Inc. 59
Large-scale testing (Phase 3)Study completedNCT02804373What this trial is testingEffects of Intranasal Administration of Oxytocin in Adults With Prader-Willi SyndromeWho this might be right forPrader-Willi Syndrome University Hospital, Toulouse 50