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: Review the eligibility criteria below.
Location: Santa Monica Clinical Trials — Los Angeles, California
Age range: 6 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 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
- A Study of Setmelanotide in Patients With Prader-Willi Syndrome — Gainesville, Florida
- A Clinical Evaluation of Non-Invasive Vagus Nerve Stimulation for Temper Outbursts in People With PWS — Palo Alto, California
- Open-Label Extension Study of DCCR in Patients With Prader-Willi Syndrome — Orange, California
- Tirzepatide in PWS, HO and GNSO — Saint Paul, Minnesota
- Regulating Together for Prader-Willi Syndrome: A Group Behavioral Therapy for Emotion Dysregulation — Kansas City, Missouri
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.