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ClinicalTrials.gov 7 recruiting now official registry

Prader-Willi Syndrome clinical trials

7 of 17 indexed Prader-Willi Syndrome trials are currently recruiting.

17 US clinical trials · 7 currently recruiting

Prader-Willi Syndrome is the subject of 17 registered US clinical trials on ClinicalTrials.gov, 7 of them currently open to new participants. 5 are in Phase 3-4 (later-stage) and 8 in Phase 1-2 (earlier-stage). The most active sponsor is Harmony Biosciences Management, running 2 of these trials.

The research picture

Prader-Willi Syndrome has 17 registered US clinical trials, 7 of them open to new participants right now, about 41% of the total.

7
recruiting participants now
41%
of trials open to enrollment
5
in Phase 3–4 (later-stage)
2
top sponsor: Harmony Biosciences Management
Phase 13Phase 25Phase 34Phase 41

Active & Recent Trials

17 total, page 1 of 1

RECRUITING NA 376 participants

Physical Activity and Community EmPOWERment Project

University of North Carolina, Chapel Hill

NCT06740162

RECRUITING Phase 1 150 participants

A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment

Rhythm Pharmaceuticals

NCT06239116

RECRUITING Phase 3 134 participants

A Study of Pitolisant in Patients With Prader-Willi Syndrome

Harmony Biosciences Management

NCT06366464

RECRUITING Phase 3 102 participants

A Clinical Evaluation of Non-Invasive Vagus Nerve Stimulation for Temper Outbursts in People With PWS

Foundation for Prader-Willi Research

NCT06144645

RECRUITING Phase 4 36 participants

Tirzepatide in PWS, HO and GNSO

Grace Kim

NCT06901245

RECRUITING NA 30 participants

Impact of Bright Light Therapy on Prader-Willi Syndrome

Maimonides Medical Center

NCT05939453

RECRUITING NA 20 participants

Cerebellar TMS and Satiety in Prader-Willi Syndrome

Brigham and Women's Hospital

NCT05938543

ACTIVE NOT RECRUITING Phase 3 83 participants

Open-Label Extension Study of DCCR in Patients With Prader-Willi Syndrome

Soleno Therapeutics

NCT05701774

ACTIVE NOT RECRUITING Phase 2 65 participants

A Phase 2 Study to Evaluate the Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome, Followed by an Open Label Extension

Harmony Biosciences Management

NCT04257929

ACTIVE NOT RECRUITING Phase 2 18 participants

A Study of Setmelanotide in Patients With Prader-Willi Syndrome

Rhythm Pharmaceuticals

NCT06772597

COMPLETED Phase 1 30 participants

Bioequivalence of Two Somatropin Products (Norditropin® Versus Genotropin®) in Healthy Adult Volunteers

Novo Nordisk A/S

NCT01401244

COMPLETED Phase 2 23 participants

Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome

Montefiore Medical Center

NCT02629991

COMPLETED Phase 2 19 participants

A Study of Oral ARD-101 in Patients With Prader-Willi Syndrome

Aardvark Therapeutics

NCT05153434

COMPLETED Phase 2 14 participants

CBDV vs Placebo in Children and Adults up to Age 30 With Prader-Willi Syndrome (PWS)

Montefiore Medical Center

NCT03848481

COMPLETED Phase 1 13 participants

Clinical Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome

Essentialis

NCT02034071

COMPLETED Phase 3 11 participants

Growth Hormone Use in Adults With Prader-Willi Syndrome

Children's Mercy Hospital Kansas City

NCT00444964

COMPLETED NA 10 participants

Effects of Exenatide on Overweight Adolescents With Prader-Willi Syndrome

Children's Hospital Los Angeles

NCT01444898

Phase Distribution

PhaseTrial count
Phase 1 3
Phase 2 5
Phase 3 4
Phase 4 1

Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.

Reading the Prader-Willi Syndrome Trial Landscape

17 indexed Prader-Willi Syndrome studies · 7 recruiting (41%).

Phase mix: 5 late (III/IV) vs 8 early (I/II).

Top sponsor: Harmony Biosciences Management (2 trials), 9 others listed. 17 shown below, recruiting first.

How to read this condition's trial landscape

That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.

Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.

All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation, eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.

Frequently Asked Questions

How many clinical trials are there for Prader-Willi Syndrome?

PlainTrial tracks 17 US clinical trials for Prader-Willi Syndrome, of which 7 are currently recruiting participants. Data sourced from ClinicalTrials.gov.

How do I find a recruiting trial for Prader-Willi Syndrome?

Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.

Is this data current?

Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.

Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.

Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2026 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.

Verify with NIH →

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial for this condition, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.