RecruitingNot applicableNCT07607730
Neurological
Prader-Willi Syndrome
Also known as PWS, chromosome 15q11-q13 paternal deletion, hypotonia-hypomentia-hypogonadism-obesity syndrome
Prader-Willi syndrome (PWS) is caused by loss of expression of paternally inherited genes on chromosome 15q11-q13 due to paternal deletion (70%), maternal uniparental disomy (25%), or imprinting defects. Neonates present with severe hypoton
ORPHA:739 ↗Gene SNRPN locusGene MAGEL2Gene NDNGene MKRN3 (15q11-q13 region)Prevalence 1-5 per 10,000 (Orphanet)Onset Neonatal, InfantileGenetic (imprinting disorder, paternal deletion or maternal UPD)
16
studies recruiting now
as of 7 Sept 2026
152
studies registered in total
as of 7 Sept 2026
14
countries with a recruiting site
as of 7 Sept 2026
26 May 2026
most recent study posted
among recruiting studies
Recruiting trials
RecruitingPhase 3NCT06366464
A Study of Pitolisant in Patients With Prader-Willi Syndrome
RecruitingNot applicableNCT05791604
The Intervention of Obesity in Children With Prader-Willi Syndrome Using Prebiotics and Probiotics
RecruitingNot applicableNCT05939453
Impact of Bright Light Therapy on Prader-Willi Syndrome
RecruitingPhase 2NCT07348601
A Study of CSTI-500 in Patients With Prader-Willi Syndrome
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 16 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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About Prader-Willi Syndrome
Prader-Willi syndrome (PWS) is caused by loss of expression of paternally inherited genes on chromosome 15q11-q13 due to paternal deletion (70%), maternal uniparental disomy (25%), or imprinting defects. Neonates present with severe hypotonia and feeding difficulties; during childhood, insatiable hyperphagia develops leading to severe obesity. Additional features include intellectual disability, hypogonadism, short stature, and behavioral problems including obsessive-compulsive traits.
Common clinical features
Severe neonatal hypotoniaHyperphagia and obesityIntellectual disabilityHypogonadismShort statureBehavioral problemsSleep-disordered breathing
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
1 approved treatment and 16 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Approved: Somatropin (Genotropin)
Phase 3Beloranib
Phase 3Topiramate (Epitomax)
Phase 3Pitolisant (Wakix)
Phase 3Rimonabant (Acomplia)
Phase 3Carbetocin (Duratocin)
Phase 2/3Livoletide
Phase 2/3Oxytocin (Orasthin)
Phase 2Metoprolol
+ 8 more in development
Before you apply
Things trial teams commonly ask about for Prader-Willi Syndrome. Not eligibility rules; those are set by each study.
- Molecular subtype (deletion vs UPD vs imprinting defect) determines eligibility for some trials — chromosome 15 methylation analysis is required
- BMI, hyperphagia rating scale scores, and metabolic panel are standard baseline eligibility measures
- Carbetocin (intranasal oxytocin) and other hyperphagia-targeting trials typically require stable growth hormone therapy as background medication
- Sleep study (polysomnography) documenting sleep-disordered breathing may be required or exclusionary
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).