Prader-Willi Syndrome

Browse recruiting and upcoming clinical trials for Prader-Willi syndrome.

Patient-friendly, AI-assisted summaries based on official ClinicalTrials.gov ↗ records. Last updated: Oct 10, 2026

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ClinicalTrials.gov is the complete and most up-to-date source. Trials may be missing, and trial information may have changed since our last check. Always verify important information in the official registry.

18 trials + 1 related

Showing 18 trials, plus 1 related

Who can join AIPeople aged 13–50 with genetically confirmed Prader‑Willi syndrome (PWS) in Nutritional Phase 3.
What is being studied AITests safety, tolerability, blood levels, and symptom effects of oral CSTI-500 in people with PWS.
Study typeTreatment (drug) · Phase 2
CountriesUnited States
Recruiting
Who can join AIPeople aged 18–26 with Prader‑Willi syndrome, hypothalamic obesity, or general (non‑syndromic) obesity who have BMI in the obesity range.
What is being studied AITests weekly tirzepatide's effects on weight, body fat, appetite, and metabolic markers over 48 weeks.
Study typeTreatment (drug) · Phase 4
CountriesUnited States
Who can join AIAdults with intellectual disability (including autism spectrum disorder, Down syndrome, Fragile X, Williams, Prader‑Willi, and other related neurodevelopmental syndromes).
What is being studied AITests a 16-week inclusive exercise (PACE) program versus waitlist to increase physical activity and related outcomes.
Study typeTreatment (other)
CountriesUnited States
Who can join AIPeople aged 6 years and older with genetically confirmed Prader‑Willi syndrome who have excessive daytime sleepiness.
What is being studied AICompares pitolisant with placebo for treating excessive daytime sleepiness in people with Prader-Willi syndrome.
Study typeTreatment (drug) · Phase 3
CountriesAustralia, Belgium, Canada +10
Who can join AIPeople with Prader‑Willi syndrome aged 7 years and older who completed the End‑of‑Treatment visit in a qualifying parent pitolisant study.
What is being studied AITests long-term safety of pitolisant in people with Prader-Willi syndrome.
Study typeTreatment (drug) · Phase 3
CountriesUnited States
Enrolling by invitation
Who can join AIInfants (15 months or younger) with a confirmed diagnosis of one of several rare neurogenetic conditions and their parent/primary caregiver (English-speaking household).
What is being studied AITests a parent-focused early intervention (PIXI) for infants diagnosed with rare neurogenetic disorders.
Study typeTreatment (other)
CountriesUnited States
Who can join AIChildren aged 5 to 7 years with genetically confirmed Prader-Willi syndrome.
What is being studied AICompares brain activity and smell tests in 5–7-year-old children with PWS who were early OT-exposed or not.
Study typeOther
CountriesFrance
Who can join AIChildren and people with imprinting disorders (examples: Silver‑Russell, Beckwith‑Wiedemann, Angelman, Prader‑Willi, and others).
What is being studied AICollects clinical, genetic, metabolic and morphometric data to describe natural history and metabolic profiles.
Study typeObservational
CountriesFrance
Who can join AIAdults (18+) with complex rare genetic syndromes listed in the study, e.g., Prader‑Willi, Turner, Noonan, Rett and others.
What is being studied AICollects and analyzes adult medical records to describe health problems, labs, symptoms, and medication use.
Study typeObservational
CountriesNetherlands
Who can join AIPeople diagnosed with Prader-Willi syndrome.
What is being studied AICollects medical, genetic, follow-up, care, and quality-of-life data for people with Prader-Willi syndrome.
Study typeObservational
CountriesFrance
Who can join AIPeople of any age with a clinical or molecular diagnosis of one or more of the listed rare diseases.
What is being studied AICollects and organizes clinical data to create a single institutional registry for many rare diseases.
Study typeRegistry
CountriesArgentina

Trials that may include Prader-Willi syndrome (1)

ClinicalTrials.gov returned these when we searched for this condition, but we could not confirm from the registry records that they are intended for people with it. Check each trial’s eligibility criteria, and ask the study team if you are unsure.