ClinicalTrials.gov record NCT06366464
A Study of Pitolisant in Patients With Prader-Willi Syndrome
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
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This Phase 3 randomized, double-blind, placebo-controlled study tests pitolisant in people with Prader-Willi syndrome who have excessive daytime sleepiness. Participants age 6 and up are randomized to pitolisant or matching placebo once daily during a 77-day double-blind period; an optional longer open-label extension follows. The study measures sleepiness, behavior (including irritability and hyperphagia), and safety.
What it is studying. The study tests pitolisant versus placebo for effects on excessive daytime sleepiness in people with Prader-Willi syndrome. It also assesses effects on irritable and disruptive behaviors, hyperphagia, other behavioral problems (social withdrawal, stereotypic behavior, hyperactivity/noncompliance, inappropriate speech), and collects safety data.
What the researchers hope to learn. To evaluate the efficacy and safety of pitolisant for excessive daytime sleepiness in Prader-Willi syndrome and to measure its impact on irritability/disruptive behaviors, hyperphagia, other behavioral domains, and treatment-emergent adverse events.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- Pitolisant tablet
- Other
- Placebo tablet
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
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
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
The study will consist of an up to 45-day Screening/Baseline Period, a Double-Blind Treatment Period, and an optional Open-Label Extension Period. After completion of all Baseline assessments, patients who meet all eligibility criteria will be randomized 1:1 to receive once daily pitolisant or matching placebo. During the Double-Blind Treatment Period, in-person visits will be at Day 29, Day 57, and Day 77. Patients who do not elect to enter the Open-Label Extension Period will have follow-up visits 15 days and 30 days after the final dose of study drug. During the optional Open-Label Extension Period, in-person visits will be at Day 113, Day 260, and Day 441. Patients will have follow-up visits 15 days and 30 days after the final dose of pitolisant.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
After up to a 45‑day screening/baseline period, eligible participants are randomized 1:1 to take either pitolisant or matching placebo once daily in the morning. During the double‑blind period there are in‑person visits at Day 29, Day 57, and Day 77; participants who do not enter the optional open‑label extension have follow‑up visits 15 and 30 days after their final dose. Those entering the open‑label extension have in‑person visits at Day 113, Day 260, and Day 441, with follow‑ups 15 and 30 days after the final dose. Caregivers will complete questionnaires about sleepiness, behaviors, and eating (hyperphagia), and the study team will record side effects and other safety information through the study period.
How the study is structured. Phase 3, randomized 1:1, double-blind (quadruple-masked), placebo-controlled, parallel-group drug trial. After screening, eligible participants are assigned to pitolisant tablets or matching placebo taken once each morning. The double-blind period includes in-person visits at Day 29, Day 57, and Day 77; an optional open-label extension has visits at Day 113, Day 260, and Day 441. Follow-up visits occur 15 and 30 days after the final dose.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
- The listed sites are in Australia, Belgium, Canada, Denmark, France, Germany, Italy, Poland, Romania, Spain, Sweden, United Kingdom, United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on Prader-Willi syndrome
Important dates
- Study start
- May 28, 2024
- Primary completion
- April 2027 (estimated)
- Study completion
- April 2028 (estimated)
- Target enrollment
- 134 participants (estimated)
- Last updated on registry
- Aug 25, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Harmony Biosciences Management, Inc.
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Ann Adee 📞 773-383-6258 ✉ clinicaltrials@harmonybiosciences.com
- Linnea Ryan ✉ clinicaltrials@harmonybiosciences.com
Participating sites (57)
- Santa Monica Clinical TrialsRecruiting
Los Angeles, California, 90025 · United States
- Samantha Montenez · 310-586-0843 · e. smontanez@smclinicaltrials.com
- Daniel Norman, MD
- Center of Excellence in Diabetes and EndocrinologyRecruiting
Sacramento, California, 95821-2123 · United States
- Natalie Marlen · 279-210-9814 · marlenn@sutterhealth.org
- Gnanagurudasan Prakasam, MD
- Rady Children's Hospital - Scan DiegoRecruiting
San Diego, California, 92123 · United States
- Megan Warner · mwarner2@rchsd.org
- Rakesh Bhattacharjee, MD
- Colorado Children's HospitalRecruiting
Aurora, Colorado, 80045 · United States
- Nathan Holly · 920-264-4742 · nathan.holly@childrenscolorado.org
- Shawn McCandless, MD
- Nemours Children's HospitalRecruiting
Wilmington, Delaware, 19803-3607 · United States
- Kimberly Renner · 302-651-6400 · kimberly.renner@nemours.org
- Aaron Chidekel, MD
- Atlanta Diabetes AssociatesWithdrawn
Atlanta, Georgia, 30318 · United States
- Emory University School of MedicineRecruiting
Atlanta, Georgia, 30322 · United States
- Gwendolyn Gunn · 404-778-8552 · gwendolyn.gisiner.gunn@emory.edu
- Michael Gambello, MD, PhD
- Rare Disease ResearchWithdrawn
Atlanta, Georgia, 30329 · United States
- Ann And Robert H Lurie Children's Hospital of ChicagoRecruiting
Chicago, Illinois, 60611 · United States
- Sarayu Ratnam, PhD · 312-227-6617 · sratnam@luriechildrens.org
- Priya Khanna, MD
- Riley Children's HospitalRecruiting
Indianapolis, Indiana, 46202 · United States
- Julia Meyer · 317-278-7118 · meyerj@iupui.edu
- Hasnaa Jalou, MD
Terms used on this page · explanations by MedicalUpdates.ai
- randomized
- Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
- double-blind
- Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
- placebo-controlled
- A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.
- primary objective
- The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.
- legal guardian
- A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
- study drug
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- 5 half-lives
- The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
- prior to Screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- Baseline
- Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
- Open-Label Extension
- A continuation study in which participants who completed an earlier study can keep receiving the treatment, usually with everyone knowing what they are given.
- eligibility criteria
- The rules the study team uses to decide who may take part. They are usually split into inclusion criteria and exclusion criteria.
- 1:1
- How many participants go to each group. In a 2:1 ratio, two are assigned to the first group for every one assigned to the second.
- matching placebo
- A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
- follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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