NCT06366464 A Study of Pitolisant in Patients With Prader-Willi Syndrome
| NCT ID | NCT06366464 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Harmony Biosciences Management, Inc. |
| Condition | Prader-Willi Syndrome |
| Study Type | INTERVENTIONAL |
| Enrollment | 134 participants |
| Start Date | 2024-05-28 |
| Primary Completion | 2027-04 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 3 trials are large pivotal studies comparing the treatment to current standard of care or placebo. Your participation directly contributes to the evidence needed for regulatory approval.
This trial targets 134 participants in total. It began in 2024-05-28 with a primary completion date of 2027-04.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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
Eligibility Criteria
Inclusion Criteria: * Genetically confirmed diagnosis of PWS * Excessive daytime sleepiness * Has a consistent parent/caregiver (preferably the same person throughout the study) who is willing and able to complete the required study assessments. * In the opinion of the Investigator, the patient/parent(s)/caregiver(s)/legal guardian(s) are capable of understanding and complying with the requirements of the protocol and administration of oral study drug. 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
Contact & Investigator
Medical Director
STUDY DIRECTOR
Harmony Biosciences Inc
Frequently Asked Questions
Who can join the NCT06366464 clinical trial?
This trial is open to participants of all sexes, aged 6 Years or older, studying Prader-Willi Syndrome. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06366464 trial and what does that mean for participants?
Phase 3 trials are large-scale studies comparing the new treatment to existing standards of care or a placebo. They provide the evidence needed for regulatory approval. This trial targets 134 participants.
Is NCT06366464 currently recruiting?
Yes, NCT06366464 is actively recruiting participants. Contact the research team at clinicaltrials@harmonybiosciences.com for enrollment information.
Where is the NCT06366464 trial being conducted?
This trial is being conducted at Los Angeles, United States, Sacramento, United States, San Diego, United States, Aurora, United States and 11 additional locations.
Who is sponsoring the NCT06366464 clinical trial?
NCT06366464 is sponsored by Harmony Biosciences Management, Inc.. The principal investigator is Medical Director at Harmony Biosciences Inc. The trial plans to enroll 134 participants.