Not applicableStudy completedNCT05024110What this trial is testingEffect of Exercise Intensity on Vitamin DWho this might be right forDown SyndromeVitamin D Deficiency Taif University 44
Not applicableLooking for participantsNCT06213090What this trial is testingPatterns of Neurodevelopmental DisordersWho this might be right forNeurodevelopmental DisordersAutism Spectrum DisorderPediatric Autoimmune Neuropsychiatric Disorder Associated With Streptococcal Infection+5 more Richard Frye 1,000
Not applicableStudy completedNCT03548779What this trial is testingNorth Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2Who this might be right forEpilepsy; SeizureNeuromuscular DiseasesBrain Malformation+14 more University of North Carolina, Chapel Hill 548
Early research (Phase 1)Active Not RecruitingNCT05422482What this trial is testingEvaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS ⅡWho this might be right forMucopolysaccharidosis IIHunter Syndrome GC Biopharma Corp 12
Not applicableLooking for participantsNCT06224413What this trial is testingParticipants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene AutotemcelWho this might be right forCerebral Adrenoleukodystrophy (CALD) Genetix Biotherapeutics Inc. 120
Not applicableUnknownNCT02728544What this trial is testingPost Exercise Irisin Levels in PWS PatientsWho this might be right forPrader Willi Syndrome Shaare Zedek Medical Center 32
Not applicableStudy completedNCT01971957What this trial is testingSjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical MarkersWho this might be right forSjogren-Larsson Syndrome (SLS) University of Nebraska 20
Testing effectiveness (Phase 2)WithdrawnNCT05146284What this trial is testingStudy to Assess PXL770 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)Who this might be right forAdrenomyeloneuropathy Poxel SA
Not applicableEnrolling By InvitationNCT03836300What this trial is testingParent and Infant Inter(X)Action Intervention (PIXI)Who this might be right forFragile X SyndromeAngelman SyndromePrader-Willi Syndrome+11 more RTI International 120
Not applicableStudy completedNCT02381457What this trial is testingSNP-based Microdeletion and Aneuploidy RegisTry (SMART)Who this might be right for22q11 Deletion SyndromeDiGeorge SyndromeTrisomy 21+8 more Natera, Inc. 20,960
Not applicableLooking for participantsNCT05945576What this trial is testingIDMet (RaDiCo Cohort) (RaDiCo-IDMet)Who this might be right forSilver Russell SyndromeBeckwith-Wiedemann SyndromeTransient Neonatal Diabetes Mellitus+6 more Institut National de la Santé Et de la Recherche Médicale, France 2,000
Not applicableStudy completedNCT01523288What this trial is testingThe Intestinal Function in People With Prader-Willi SyndromeWho this might be right forPrader-Willi SyndromeConstipationIncontinence Aarhus University Hospital 21
Testing effectiveness (Phase 2)Study completedNCT02060526What this trial is testingRandomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA DiseaseWho this might be right forSanfilippo Syndrome Shire 21
Testing effectiveness (Phase 2)Study completedNCT04257929What this trial is testingEvaluate the Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome, Followed by an Open Label ExtensionWho this might be right forPrader-Willi Syndrome Harmony Biosciences Management, Inc. 65
Not applicableLooking for participantsNCT05939453What this trial is testingImpact of Bright Light Therapy on Prader-Willi SyndromeWho this might be right forPrader-Willi SyndromeExcessive Daytime SleepinessHyperphagia+3 more Maimonides Medical Center 50
Not applicableLooking for participantsNCT07122505What this trial is testingOromyofunctional Therapy: a Rehabilitation Program for OSA in Children With Down Syndrome and Prader-Willi SyndromeWho this might be right forObstructive Sleep Apnea (OSA)Orofacial Myofunctional Disorders University Ghent 60
Not applicableStudy completedNCT02464514What this trial is testingMacronutrient Regulation of Ghrelin and Peptide YYWho this might be right forObesityPrader Willi Syndrome Duke University 28
Not applicableStudy completedNCT03689621What this trial is testingProof of Concept Study of Vagus Nerve StimulationWho this might be right forTranscutaneous Vagal Nerve Stimulation (tVNS)Prader-Willi Syndrome Jessica Beresford-Webb 9
Testing effectiveness (Phase 2)Active Not RecruitingNCT05665166What this trial is testingGene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type IIWho this might be right forMucopolysaccharidosis II University of Manchester 5
Not applicableStudy completedNCT03627416What this trial is testingRepetitive Transcranial Magnetic Stimulation as Therapy in Hereditary Spastic Paraplegia and AdrenomyeloneuropathyWho this might be right forHereditary Spastic ParaplegiaAdrenomyeloneuropathy Jakub Antczak 15