Not applicableEnded earlyNCT04976231What this trial is testingMPS II ImmunophenotypingWho this might be right forMPS IIMucopolysaccharidosis IIHunter Syndrome Duke University 6
Not applicableApproved For MarketingNCT04074512What this trial is testingCopper Histidinate Treatment for Menkes DiseaseWho this might be right forMenkes Disease Sentynl Therapeutics, Inc.
Not applicableStudy completedNCT04597645What this trial is testingPrader-Willi Syndrome Body CompositionWho this might be right forPrader Labhart Willi SyndromeBody Weight ChangesSarcopenia Taipei Tzu Chi Hospital, Buddhist Tzu Chi Medical Foundation 20
Not applicableUnknownNCT04768803What this trial is testingGhrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, and/or Overweight, and/or ObesityWho this might be right forAngelman SyndromeSmith-Magenis SyndromeX Fragile Syndrome+2 more University Hospital, Toulouse 300
Not applicableStudy completedNCT06336577What this trial is testingInfectious and Non-infectious Lower Respiratory Diseases in Children With Down Syndrome Followed in Pediatric Pulmonology Consultations in Ile-de-FranceWho this might be right forDown Syndrome Centre Hospitalier Sud Francilien 108
Large-scale testing (Phase 3)Study completedNCT02412787What this trial is testingStudy of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094Who this might be right forHunter Syndrome Shire 56
Testing effectiveness (Phase 2)Study completedNCT05153434What this trial is testingOral ARD-101 in Patients With Prader-Willi SyndromeWho this might be right forPrader-Willi Syndrome Aardvark Therapeutics, Inc. 19
Not applicableStudy completedNCT04687007What this trial is testingSMART-ALD - A New Lifestyle Intervention to Improve Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD)Who this might be right forX-linked Adrenoleukodystrophy Leipzig University Medical Center 68