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New drug aims to tackle sleepiness in rare genetic disorder

NCT ID NCT06366464

Symptom relief Sponsor: Harmony Biosciences Management, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Aug 26, 2026 · Updated 3 times

Summary

This Phase 3 trial tests whether pitolisant can reduce excessive daytime sleepiness in people aged 6 and older with Prader-Willi syndrome. About 134 participants will receive either pitolisant or a placebo, and researchers will also look at effects on behavior and appetite. The study is currently recruiting.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
pitolisant
What this could lead to
If successful, pitolisant could become a new treatment to reduce daytime sleepiness and improve behavior in people with Prader-Willi syndrome.
What could go wrong
This is a Phase 3 trial, but results are not guaranteed. The drug may not work better than placebo, and side effects are possible.
Why investors are watching

Harmony Biosciences is running a Phase 3 trial of pitolisant for excessive daytime sleepiness in people with Prader-Willi syndrome, a rare genetic condition. For a small company, this readout matters because pitolisant is likely a key product in its pipeline, and a positive result could expand its use beyond current approved indications. The trial also measures effects on behaviors like irritability and hyperphagia, which could broaden the drug's appeal if successful.

If it works: A positive result could lead to a new regulatory approval for pitolisant in Prader-Willi syndrome, giving Harmony a second revenue source. It might also strengthen the company's position in rare disease treatments and attract more attention from larger partners or acquirers.

If it fails: A failed or delayed trial could set back the company's growth plans and hurt its stock, since small companies often depend on a few key products. Trials fail often, so investors should expect that pitolisant might not meet its goals in this patient group.

AI-written from the trial record. Speculative, and not investment advice.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 134 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2024

Expected to finish

Apr 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

6 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

Disorders of Excessive Somnolence Prader-Willi syndrome Problem Behavior

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Prader-Willi syndrome

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    52 sites in 13 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

  • Contact

    Email: •••••@•••••

Locations

  • AMNDX Inc.

    RECRUITING

    Thornhill, Ontario, Canada

  • Aarhus University Hospital

    RECRUITING

    Aarhus, Denmark

  • Ann And Robert H Lurie Children's Hospital of Chicago

    RECRUITING

    Chicago, Illinois, 60611, United States

  • Atlanta Diabetes Associates

    WITHDRAWN

    Atlanta, Georgia, 30318, United States

  • Azienda Ospedale Università Padova - Dipartimento Salute della Donna e del Bambino - INCIPIT - PIN

    RECRUITING

    Padova, Italy

  • Azienda Ospedaliero Universitaria A Meyer

    RECRUITING

    Florence, Italy

  • CHU d'Angers

    RECRUITING

    Angers, France

  • CHU de Toulouse-Hôpital Des Enfants

    RECRUITING

    Toulouse, France

  • CTI Clinical Research Center

    WITHDRAWN

    Cincinnati, Ohio, 45212, United States

  • Center for Human Genetics

    RECRUITING

    Cleveland, Ohio, 441016, United States

  • Center of Excellence in Diabetes and Endocrinology

    RECRUITING

    Sacramento, California, 95821-2123, United States

  • Children's Hospital at Westmead

    RECRUITING

    Westmead, Australia

  • Childrens Hospital of Wisconsin

    RECRUITING

    Milwaukee, Wisconsin, 53226, United States

  • Colorado Children's Hospital

    RECRUITING

    Aurora, Colorado, 80045, United States

  • Corporacio Sanitaria Parc Tauli, Sabadell

    RECRUITING

    Barcelona, Spain

  • Emory University School of Medicine

    RECRUITING

    Atlanta, Georgia, 30322, United States

  • Fulbourn Hospital

    RECRUITING

    Cambridge, England, United Kingdom

  • Hospital Sant Joan de Deu

    RECRUITING

    Barcelona, Spain

  • Hospital Universitario 12 de Octubre

    RECRUITING

    Madrid, Spain

  • Hull Royal Infirmary

    RECRUITING

    Hull, England, United Kingdom

  • IRCCS Materno Infantile Burlo Garofolo - INCIPIT - PIN

    RECRUITING

    Trieste, Italy

  • Institutul National de Endocrinologie C. I. Parhon

    RECRUITING

    Bucharest, Romania

  • Institutul National de Endocrinologie C. I. Parhon

    RECRUITING

    Bucharest, Romania

  • Istituto G Gaslini Ospedale Pediatrico IRCCS - INCIPIT - PIN

    RECRUITING

    Genova, Italy

  • Jodha Tishon Inc.

    WITHDRAWN

    Toronto, Ontario, Canada

  • Johns Hopkins Hospital

    RECRUITING

    Baltimore, Maryland, 21224, United States

  • Johns Hopkins University Hospital

    RECRUITING

    Baltimore, Maryland, 21205, United States

  • Karolinska Universitetssjukhuset Solna

    RECRUITING

    Solna, Sweden

  • Louis Turcanu Emergency Clinical Hospital for Children

    RECRUITING

    Timișoara, Romania

  • Maimonides Medical Center

    RECRUITING

    Brooklyn, New York, 11219, United States

  • Mayo Clinic-PPDS

    RECRUITING

    Rochester, Minnesota, 55905, United States

  • Montefiore Medical Center

    RECRUITING

    The Bronx, New York, 10467, United States

  • National Clinical Center for Children Neurorehabilitation "Dr. Nicolae Robanescu"

    RECRUITING

    Bucharest, Romania

  • Nemours Children's Hospital

    RECRUITING

    Wilmington, Delaware, 19803-3607, United States

  • Ninewells Hospital - PPDS

    RECRUITING

    Dundee, Scotland, United Kingdom

  • Ospedale Pediatrico Bambino Gesù IRCCS

    RECRUITING

    Roma, Italy

  • Ospedale San Raffaele S.r.l. - PPDS

    RECRUITING

    Milan, Italy

  • Queensland Children's Hospital

    RECRUITING

    Brisbane, Queensland, Australia

  • Rady Children's Hospital - Scan Diego

    RECRUITING

    San Diego, California, 92123, United States

  • Rare Disease Research

    WITHDRAWN

    Atlanta, Georgia, 30329, United States

  • Rigshospitalet

    RECRUITING

    Copenhagen, Denmark

  • Riley Children's Hospital

    RECRUITING

    Indianapolis, Indiana, 46202, United States

  • Road Runner Research

    RECRUITING

    San Antonio, Texas, 78249-3539, United States

  • Royal Hospital for Children and Young People

    RECRUITING

    Edinburgh, Scotland, United Kingdom

  • Royal Prince Alfred Hospital

    RECRUITING

    Camperdown, Australia

  • Samodzielny Publiczny Szpital Kliniczny

    RECRUITING

    Szczecin, Poland

  • Santa Monica Clinical Trials

    RECRUITING

    Los Angeles, California, 90025, United States

  • Science 37

    RECRUITING

    Morrisville, North Carolina, 27560, United States

  • Science 37 (at-home option)

    RECRUITING

    Morrisville, North Carolina, 27560, United States

  • Sydney Children's Hospital

    RECRUITING

    Randwick, Australia

  • Texas Children's Hospital

    RECRUITING

    Houston, Texas, 77030, United States

  • Texas Valley Clinical Research

    TERMINATED

    Weslaco, Texas, 78596, United States

  • UZ Brussels

    RECRUITING

    Jette, Belgium

  • Universitario Virgen de la Victoria

    RECRUITING

    Málaga, Spain

  • University Hospital Essen

    RECRUITING

    Essen, Germany

  • University of Florence

    RECRUITING

    Florence, Tuscany, 50139, Italy

  • Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu-Chalubinskiego 2-2a

    RECRUITING

    Wroclaw, Poland

More trials for these conditions

Other studies related to the condition(s) this trial covers.