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NCT04257929 · ClinicalTrials.gov registry record · Phase 2
A Phase 2 Study to Evaluate the Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome, Followed by an Open Label Extension
A Phase 2 study of Prader-Willi Syndrome, sponsored by Harmony Biosciences Management.
- Active
- Registry status
- Phase 2
- Development phase
- 65
- Enrollment target
- 13
- Study locations
NCT04257929: Active Phase 2 study of Prader-Willi Syndrome, sponsored by Harmony Biosciences Management.
NCT04257929 is a Phase 2 study of Prader-Willi Syndrome that is active but no longer recruiting, run by Harmony Biosciences Management. The registered enrollment target is 65 participants, roughly in line with the 67-participant average among 16 other Prader-Willi Syndrome trials with a reported enrollment target. The trial reports 13 study locations across 10 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04257929, a Phase 2 study of Prader-Willi Syndrome, is active but no longer recruiting, sponsored by Harmony Biosciences Management.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 65 participants
- Enrollment target
- 13
- Study locations
Study Summary
The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Prader Willi syndrome (PWS) ages 6 to 65 years.
Primary Outcome
Change in Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD) (parent/caregiver version) score from Baseline to Week 11 for pitolisant compared with placebo. The score of the ESS-CHAD ranges from 0 to 24. A decrease in score represents an improvement in excessive daytime sleepiness.
Conditions Studied
Interventions
- DRUG Placebo oral tablet
- DRUG Pitolisant oral tablets
Study Locations (13)
California
- Rady Children's Hospital - San Diego - San Diego
- Sleep Medicine Specialists of California - San Ramon
- Santa Monica Clinical Trials - Santa Monica
Texas
- Texas Children's Hospital/Baylor College of Medicine - Houston
- Road Runner Research - San Antonio
Colorado
- Children's Hospital Colorado - Aurora
Delaware
- Nemours Alfred I duPont Hospital for Children - Wilmington
Florida
- University of Florida College of Medicine - Gainesville
Illinois
- Ann and Robert H Lurie Children's Hospital - Chicago
Maryland
- Johns Hopkins School of Medicine - Baltimore
Nebraska
- University of Nebraska Medical Center - Omaha
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 65 participants |
| Start Date | 2020-12-09 |
| Est. Completion | 2028-09-07 |
| Phase | Phase 2 |
What the registry record for NCT04257929 still lists
NCT04257929 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 65 participants, a relatively small participant target, roughly in line with the 67-participant average among 16 other Prader-Willi Syndrome trials with a reported enrollment target.
The record links to 1 condition, with Prader-Willi Syndrome appearing as the primary indexed condition, and to 2 interventions - of which Placebo oral tablet is the first listed.
NCT04257929 lists 13 locations in 10 states (California, Texas, Colorado).
Frequently Asked Questions
What is clinical trial NCT04257929 about?
NCT04257929 is a clinical study titled "A Phase 2 Study to Evaluate the Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome, Followed by an Open Label Extension". The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Prader Willi syndrome (PWS) ages 6 to 65 years.
What is the current status of trial NCT04257929?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 65 participants. The study started on 2020-12-09. Estimated completion is 2028-09-07.
What conditions does trial NCT04257929 study?
This clinical trial studies the following conditions: Prader-Willi Syndrome.
What interventions are being tested in trial NCT04257929?
The interventions under investigation include: Placebo oral tablet (DRUG), Pitolisant oral tablets (DRUG).
Who is sponsoring clinical trial NCT04257929?
This trial is sponsored by Harmony Biosciences Management, which has 10 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04257929 being conducted?
This trial has 13 study locations across California, Colorado, Delaware, Florida, Illinois. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Prader-Willi Syndrome
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04257929's enrollment target sits among peer trials
65 6th of 16 higher than 11 of 16 other Prader-Willi Syndrome trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Prader-Willi Syndrome trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
A Study of Setmelanotide in Patients With Prader-Willi Syndrome
ACTIVE NOT RECRUITING · Phase 2
-
CBDV vs Placebo in Children and Adults up to Age 30 With Prader-Willi Syndrome (PWS)
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-
Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome
COMPLETED · Phase 2
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A Study of Oral ARD-101 in Patients With Prader-Willi Syndrome
COMPLETED · Phase 2
-
Physical Activity and Community EmPOWERment Project
RECRUITING · NA
-
A Clinical Evaluation of Non-Invasive Vagus Nerve Stimulation for Temper Outbursts in People With PWS
RECRUITING · Phase 3
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