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Histamine 3 Receptor Antagonist

Pitolisant for Prader-Willi Syndrome

Phase 2
Waitlist Available
Research Sponsored by Harmony Biosciences, LLC
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
In the opinion of the Investigator, patient/parent(s)/legal guardian(s) is capable of understanding and complying with the requirements of the protocol and administration of oral study drug.
If using cannabidiol and/or tetrahydrocannabinol, patient must be on a stable dose for 28 days prior to randomization and agree to continue the stable dose for the duration of the Double-Blind Treatment Phase of the study (dose adjustments will be permitted in the OLE Phase).
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to week 11
Awards & highlights

Study Summary

This trial will study whether pitolisant is safe and effective in treating excessive daytime sleepiness in patients with Prader Willi syndrome.

Who is the study for?
This trial is for individuals aged 6 to 65 with Prader-Willi Syndrome who experience excessive daytime sleepiness. Participants must have a confirmed diagnosis, get enough nightly sleep, and if on certain medications or treatments, they need to be on stable doses. Females of childbearing age must use nonhormonal contraception and not be pregnant.Check my eligibility
What is being tested?
The study tests the safety and effectiveness of pitolisant oral tablets against placebo in reducing excessive daytime sleepiness in Prader-Willi Syndrome patients. It includes an initial phase where participants are randomly given either the medication or a placebo, followed by an open label extension where all receive pitolisant.See study design
What are the potential side effects?
While specific side effects for pitolisant are not listed here, common ones may include headache, insomnia, nausea, anxiety, irritability or dizziness. Side effects can vary from person to person.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I understand and can follow the study's requirements and take oral medication as directed.
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I have been on a stable dose of CBD/THC for 28 days and agree to keep it the same during the study.
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I've been on a stable dose of my behavior management medication for at least 28 days.
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My Prader-Willi Syndrome (PWS) diagnosis is confirmed by genetic tests or medical records.
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I am a woman who can have children, not pregnant, and will avoid pregnancy during the study.
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I have recorded my sleep, getting enough hours each night according to my age group.
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I have been on a stable dose of my hormone treatments or other medications for 3 months.
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I've been on a stable dose of medication for sleepiness for at least 28 days.
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I have been on a stable dose of oxytocin or carbetocin for the last 28 days and agree to keep it the same for the study.
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I have a reliable caregiver who can help with my study needs.
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I am between 6 and 65 years old.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to week 11
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to week 11 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Secondary outcome measures
Attention
Behavior
Behavioral and Cognitive Rigidity
+5 more
Other outcome measures
Ghrelin levels
Hyperphagia

Trial Design

4Treatment groups
Experimental Treatment
Active Control
Placebo Group
Group I: Open-Label PitolisantExperimental Treatment1 Intervention
Age-based dosing (prior to implementation of amendment 6) with maximum once daily doses of 17.8 mg for pediatric patients (6 to <12 years), 26.7 mg for adolescent patients (12 to <18 years), or 35.6 mg for adult patients (18 to 65 years). Weight-based dosing (after implementation of amendment 6) with maximum once daily doses of 17.8 mg for patients ≤40 kg, 35.6 mg for patients >40 to ≤80 kg, and 44.5 mg for patients >80 kg.
Group II: Double-Blind Treatment Phase Lower Dose PitolisantActive Control1 Intervention
Pediatric patients (6 to less than 12 years of age): Week 1: 4.45 mg pitolisant administered once daily in the morning; Week 2: 8.9 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 8.9 mg pitolisant administered once daily in the morning. Adolescent patients (12 to less than 18 years of age): Week 1: 4.45 mg pitolisant administered once daily in the morning; Week 2: 8.9 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 13.35 mg pitolisant administered once daily in the morning. Adult patients (18 to 65 years of age): Week 1: 4.45 mg pitolisant administered once daily in the morning; Week 2: 8.9 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 17.8 mg pitolisant administered once daily in the morning.
Group III: Double-Blind Treatment Phase Higher Dose PitolisantActive Control1 Intervention
Pediatric patients (6 to less than 12 years of age): Week 1: 4.45 mg pitolisant administered once daily in the morning; Week 2: 8.9 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 17.8 mg pitolisant administered once daily in the morning. Adolescent patients (12 to less than 18 years of age): Week 1: 8.9 mg pitolisant administered once daily in the morning; Week 2: 17.8 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 26.7 mg pitolisant administered once daily in the morning. Adult patients (18 to 65 years of age): Week 1: 8.9 mg pitolisant administered once daily in the morning; Week 2: 17.8 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 35.6 mg pitolisant administered once daily in the morning.
Group IV: Double-Blind Treatment Phase PlaceboPlacebo Group1 Intervention
Pediatric patients (6 to less than 12 years of age): Week 1: Matching placebo tablets; Week 2: Matching placebo tablets; Weeks 3 through 11: Matching placebo tablets Adolescent patients (12 to less than 18 years of age): Week 1: Matching placebo tablets; Week 2: Matching placebo tablets; Weeks 3 through 11: Matching placebo tablets Adult patients (18 to 65 years of age): Week 1: Matching placebo tablets; Week 2: Matching placebo tablets; Weeks 3 through 11: Matching placebo tablets

Find a Location

Who is running the clinical trial?

Harmony Biosciences, LLCLead Sponsor
6 Previous Clinical Trials
1,719 Total Patients Enrolled

Media Library

Pitolisant (Histamine 3 Receptor Antagonist) Clinical Trial Eligibility Overview. Trial Name: NCT04257929 — Phase 2
Prader-Willi Syndrome Research Study Groups: Double-Blind Treatment Phase Lower Dose Pitolisant, Double-Blind Treatment Phase Higher Dose Pitolisant, Double-Blind Treatment Phase Placebo, Open-Label Pitolisant
Prader-Willi Syndrome Clinical Trial 2023: Pitolisant Highlights & Side Effects. Trial Name: NCT04257929 — Phase 2
Pitolisant (Histamine 3 Receptor Antagonist) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04257929 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
~15 spots leftby Mar 2025