A Study of Pitolisant in Patients With Prader-Willi Syndrome (NCT06366464)
A Phase 3, Randomized, Double-Blind, Placebo-controlled, Efficacy and Safety Study of Pitolisant Followed by an Open-Label Extension in Patients With Prader-Willi Syndrome
🩺 Plain-English Summary (8th-Grade Level)
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
Who can join: To join this study, participants generally need to meet the listed inclusion criteria (4 items) and avoid the listed exclusions (4 items).
Location: Santa Monica Clinical Trials — Los Angeles, California
Age range: 6 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistCheck every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).
Usually NOT eligible if any of the following apply:
- Exclusion Criteria:
- Has a diagnosis of sleep apnea (OSA, CSA) that is not adequately controlled
- Has a diagnosis of hypersomnia due to another sleep/medical disorder
- Participation in an interventional research study involving another investigational medication, device, or behavioral treatment within 30 days or 5 half-lives (whichever is longer) of the investigational medication prior to Screening
🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.
📍 All Participating Trial Locations (57 Sites)
Showing the first 25 of 57 sites.
- Santa Monica Clinical Trials — Los Angeles, California
- Center of Excellence in Diabetes and Endocrinology — Sacramento, California
- Rady Children's Hospital - Scan Diego — San Diego, California
- Colorado Children's Hospital — Aurora, Colorado
- Nemours Children's Hospital — Wilmington, Delaware
- Atlanta Diabetes Associates — Atlanta, Georgia
- Emory University School of Medicine — Atlanta, Georgia
- Rare Disease Research — Atlanta, Georgia
- Ann And Robert H Lurie Children's Hospital of Chicago — Chicago, Illinois
- Riley Children's Hospital — Indianapolis, Indiana
- Johns Hopkins University Hospital — Baltimore, Maryland
- Johns Hopkins Hospital — Baltimore, Maryland
- Mayo Clinic-PPDS — Rochester, Minnesota
- Maimonides Medical Center — Brooklyn, New York
- Montefiore Medical Center — The Bronx, New York
- Science 37 (at-home option) — Morrisville, North Carolina
- Science 37 — Morrisville, North Carolina
- CTI Clinical Research Center — Cincinnati, Ohio
- Center for Human Genetics — Cleveland, Ohio
- Texas Children's Hospital — Houston, Texas
- Road Runner Research — San Antonio, Texas
- Texas Valley Clinical Research — Weslaco, Texas
- Childrens Hospital of Wisconsin — Milwaukee, Wisconsin
- Queensland Children's Hospital — Brisbane, Queensland
- Royal Prince Alfred Hospital — Camperdown,
📞 Contact the Study Coordinator
Phone: (773) 383-6258
Email: clinicaltrials@harmonybiosciences.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Prader-Willi Syndrome
- Tirzepatide in PWS, HO and GNSO — Saint Paul, Minnesota
- A Study of Setmelanotide in Patients With Prader-Willi Syndrome — Gainesville, Florida
- Regulating Together for Prader-Willi Syndrome: A Group Behavioral Therapy for Emotion Dysregulation — Kansas City, Missouri
- Impact of Bright Light Therapy on Prader-Willi Syndrome — Brooklyn, New York
- A Clinical Evaluation of Non-Invasive Vagus Nerve Stimulation for Temper Outbursts in People With PWS — Palo Alto, California
Explore by condition: All Prader-Willi Syndrome clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.