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NCT06366464 · ClinicalTrials.gov registry record · Phase 3
A Study of Pitolisant in Patients With Prader-Willi Syndrome
A Phase 3 study of Prader-Willi Syndrome, sponsored by Harmony Biosciences Management.
- Recruiting
- Registry status
- Phase 3
- Development phase
- 134
- Enrollment target
- 20
- Study locations
NCT06366464: Recruiting Phase 3 study of Prader-Willi Syndrome, sponsored by Harmony Biosciences Management.
NCT06366464 is a Phase 3 study of Prader-Willi Syndrome that is actively recruiting participants, run by Harmony Biosciences Management. The registered enrollment target is 134 participants, above the 63-participant average among 16 other Prader-Willi Syndrome trials with a reported enrollment target (113% higher). The trial reports 20 study locations across 12 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06366464, a Phase 3 study of Prader-Willi Syndrome, is actively recruiting participants, sponsored by Harmony Biosciences Management.
- RECRUITING
- Registry status
- Phase 3
- Development phase
- 134 participants
- Enrollment target
- 20
- Study locations
Study Summary
This is a Phase 3, randomized, double-blind, placebo-controlled, multicenter, global clinical study to assess the efficacy and safety of pitolisant in patients living with Prader-Willi syndrome. The primary objective of this study is to evaluate the efficacy of pitolisant in treating excessive daytime sleepiness (EDS) in patients ≥6 years of age with Prader-Willi syndrome. Secondary objectives include assessing the impact of pitolisant on: Irritable and disruptive behaviors Hyperphagia Other behavioral problems including social withdrawal, stereotypic behavior, hyperactivity/noncompliance, and inappropriate speech
Primary Outcome
The PROMIS-SRI item bank consists of 13 items with a 5-point rating scale.
Conditions Studied
Interventions
- DRUG Pitolisant tablet
- OTHER Placebo tablet
Study Locations (20)
California
- Santa Monica Clinical Trials - Los Angeles
- Center of Excellence in Diabetes and Endocrinology - Sacramento
- Rady Children's Hospital - Scan Diego - San Diego
Georgia
- Atlanta Diabetes Associates - Atlanta
- Emory University School of Medicine - Atlanta
- Rare Disease Research - Atlanta
Texas
- Texas Children's Hospital - Houston
- Road Runner Research - San Antonio
- Texas Valley Clinical Research - Weslaco
New York
- Maimonides Medical Center - Brooklyn
- Montefiore Medical Center - The Bronx
Ohio
- CTI Clinical Research Center - Cincinnati
- Center for Human Genetics - Cleveland
Colorado
- Colorado Children's Hospital - Aurora
Delaware
- Nemours Children's Hospital - Wilmington
Illinois
- Ann And Robert H Lurie Children's Hospital of Chicago - Chicago
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 134 participants |
| Start Date | 2024-05-28 |
| Est. Completion | 2027-07 |
| Phase | Phase 3 |
What NCT06366464 shows while recruiting
NCT06366464 is an interventional study that assigns participants to a tested intervention. The registered 134 participants enrollment target is mid-sized for trials with a published cap, above the 63-participant average among 16 other Prader-Willi Syndrome trials with a reported enrollment target (113% higher).
The record links to 1 condition, with Prader-Willi Syndrome appearing as the primary indexed condition, and to 2 interventions - of which Pitolisant tablet is the first listed.
NCT06366464 names 20 study sites across 12 states, led by California, Georgia, Texas.
Frequently Asked Questions
What is clinical trial NCT06366464 about?
NCT06366464 is a clinical study titled "A Study of Pitolisant in Patients With Prader-Willi Syndrome". This is a Phase 3, randomized, double-blind, placebo-controlled, multicenter, global clinical study to assess the efficacy and safety of pitolisant in patients living with Prader-Willi syndrome. The primary objective of this study is to evaluate the efficacy of pitolisant in treating excessive dayt...
What is the current status of trial NCT06366464?
This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 134 participants. The study started on 2024-05-28. Estimated completion is 2027-07.
What conditions does trial NCT06366464 study?
This clinical trial studies the following conditions: Prader-Willi Syndrome.
What interventions are being tested in trial NCT06366464?
The interventions under investigation include: Pitolisant tablet (DRUG), Placebo tablet (OTHER).
Who is sponsoring clinical trial NCT06366464?
This trial is sponsored by Harmony Biosciences Management, which has 10 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06366464 being conducted?
This trial has 20 study locations across California, Colorado, Delaware, Georgia, 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 NCT06366464's enrollment target sits among peer trials
134 3rd of 16 higher than 14 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
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