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ClinicalTrials.gov 7 recruiting now early-phase tilt small index

Prader-Willi Syndrome: early-phase trial registry

7 of 17 indexed Prader-Willi Syndrome trials are currently recruiting, with more weight in Phase 1-2.

Corpus placement: #1,004 of 28,707 conditions by registered trial count.

17 US clinical trials · 7 currently recruiting · #1,004 of 28,707 conditions by registered trial count

Prader-Willi Syndrome: 17 US clinical trials tracked, 7 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.

Enrollment posture brief

Registry condition prader-willi-syndrome

OPEN 7 | RECR 41% | LATE 5 | EARLY 8 | RANK #1004 | SPON 2

Nearest open-share peer: Erectile Dysfunction (41% recruiting · 34 trials)

Acute Myeloid Leukemia, in R70.6%Abdominal Aortic Aneurysm52.9%Prader-Willi Syndrome (this)41%Acute Lymphoblastic Leukemia23.5%Actinic Keratosis11.8%
Open-enrollment share among volume-matched conditions near Prader-Willi Syndrome

Prader-Willi Syndrome lists 7 open slots (41% of 17 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →

Key findings

What ClinicalTrials.gov does not surface for Prader-Willi Syndrome on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.

  1. Prader-Willi Syndrome is recruiting below its size-band peers.

    7 of 17 indexed trials (41.2%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.

  2. Sponsorship of Prader-Willi Syndrome trials is spread across many organizations.

    Harmony Biosciences Management accounts for 2 of 17 trials (11.8%). 14 distinct sponsors appear on at least one study for this condition label.

  3. Most Prader-Willi Syndrome trials plan to enrol fewer than 100 people.

    Median target enrolment is 30 among the 17 trials that report a genuine target (sentinel values excluded). 13 of those (76.5%) plan for fewer than 100 participants.

  4. Prader-Willi Syndrome research is mostly interventional.

    17 interventional and 0 observational studies make up this index (100% interventional). Study type is sponsor-reported on ClinicalTrials.gov.

Phase 13Phase 25Phase 34Phase 41

Open and 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

Named interventions

Most-linked intervention names on Prader-Willi Syndrome records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.

DCCR Drug
2 trials
Placebo Drug
2 trials
somatropin Drug
1 trials
ARD-101 Drug
1 trials
Nutropin AQ Drug
1 trials
CBDV Compound Drug
1 trials
Pitolisant oral tablets Drug
1 trials
Exenatide Drug
1 trials

US sites by state

Facility locations filed on Prader-Willi Syndrome trials. A trial with sites in several states counts in each.

California 7 trials
New York 6 trials
Texas 5 trials
Tennessee 5 trials
Ohio 4 trials
Minnesota 4 trials
Maryland 4 trials
Colorado 4 trials

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

What to do with this Prader-Willi Syndrome page

17 registered trials, 7 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.

Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Prader-Willi Syndrome condition card totals 17 registered trials; most-linked intervention DCCR; top US site state California. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.