Not applicableEnded earlyNCT03871751What this trial is testingHome-based SSP on Individuals With PWSWho this might be right forPrader-Willi Syndrome Indiana University 10
Not applicableUnknownNCT05548751What this trial is testingPhysiotherapy Assessment Based on the ICF Model in The Lesch-Nyhan Syndrome: Case ReportWho this might be right forLesch-Nyhan Syndrome Uskudar University 1
Not applicableUnknownNCT03766386What this trial is testingThe Natural History of Danon DiseaseWho this might be right forDanon Disease University of California, San Diego 200
Large-scale testing (Phase 3)WithdrawnNCT05387798What this trial is testingExtension Study of RAD011 (Cannabidiol Oral Solution) in Patients With Prader-Willi SyndromeWho this might be right forPrader-Willi Syndrome Radius Pharmaceuticals, Inc.
Not applicableStudy completedNCT01822184What this trial is testingObservational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)Who this might be right forMucopolysaccharidosis (MPS)Hunter Syndrome Shire 100
Not applicableWithdrawnNCT02298647What this trial is testingBiomarker for Gangliosidosis: BioGM1/BioGM2 (BioGM1/GM2)Who this might be right forHepato-splenomegalyDysostosis MultiplexSeizures+1 more CENTOGENE GmbH Rostock
Early research (Phase 1)Study completedNCT01065558What this trial is testingSafety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan DiseaseWho this might be right forLesch-Nyhan Disease Psyadon Pharma 5
Not applicableStudy completedNCT03161171What this trial is testingParental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-IIIWho this might be right forMucopolysaccharidosis Type IMucopolysaccharidosis Type IIMucopolysaccharidosis Type III+2 more University Hospital Heidelberg 34
Post-approval studies (Phase 4)WithdrawnNCT04066088What this trial is testingDose Clinical Trial of Guanfacine Extended Release for the Reduction of Aggression and Self-injuries Behavior Associated With Prader-Willi SyndromeWho this might be right forPrader-Willi Syndrome NYU Langone Health
Not applicableActive Not RecruitingNCT03655223What this trial is testingEarly Check: Expanded Screening in NewbornsWho this might be right forSpinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more RTI International 30,000
Post-approval studies (Phase 4)Study completedNCT00607386What this trial is testingSafety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase TherapyWho this might be right forHunter SyndromeMucopolysaccharidosis IIMPS II Shire 28
Large-scale testing (Phase 3)Ended earlyNCT05098509What this trial is testingAssess RAD011 (Cannabidiol Oral Solution) for the Treatment of Participants With Prader-Willi SyndromeWho this might be right forPrader-Willi Syndrome Radius Pharmaceuticals, Inc. 4
Post-approval studies (Phase 4)Study completedNCT03031626What this trial is testingOxygen Versus Medical Air for Treatment of CSA in Prader Will SyndromeWho this might be right forSleep Apnea, CentralPrader-Willi Syndrome The Hospital for Sick Children 9
Not applicableStudy completedNCT04554355What this trial is testingEffects of a PA Intervention for Fatness and Fitness in Adolescents With Intellectual DisabilityWho this might be right forObesityHealth-related Physical FitnessIntellectual Disability Hong Kong Baptist University 86
Not applicableLooking for participantsNCT06740162What this trial is testingPhysical Activity and Community EmPOWERment ProjectWho this might be right forIntellectual DisabilityNeurodevelopmental DisordersAutism Spectrum Disorder+10 more University of North Carolina, Chapel Hill 376
Not applicableStudy completedNCT02529085What this trial is testingPWS European Blood Bank for Infants and Controls From 0 to 48 MonthsWho this might be right forPrader-Willi Syndrome University Hospital, Toulouse 215
Large-scale testing (Phase 3)Study completedNCT00007020What this trial is testingCompassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic AcidWho this might be right forInfantile Refsum's DiseaseZellweger SyndromeAdrenoleukodystrophy+2 more Mirum Pharmaceuticals, Inc. 85
Not applicableUnknownNCT06156124What this trial is testingThe Role of Family and Individual Factors in Going Through Adolescence - Perspective of a Healthy Child.Who this might be right forDisability, IntellectualDisability PsychologicalDisability, Developmental+7 more University of Gdansk 320
Not applicableStudy completedNCT03718416What this trial is testingNatural History Study of Serious Medical Events in PWSWho this might be right forPrader-Willi Syndrome Foundation for Prader-Willi Research 700
Not applicableStudy completedNCT04526379What this trial is testingStudy of Emotion and Cognition Abilities of Children With PWS and Proposition of an Innovative RemediationWho this might be right forPrader-Willi Syndrome University Hospital, Toulouse 50