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Prader-Willi SyndromePhase 3NCT06366464

A Study of Pitolisant in Patients With Prader-Willi Syndrome

A Phase 3 study testing Pitolisant tablet in people with Prader-Willi Syndrome, run by Harmony Biosciences Management, Inc. at 57 sites in 13 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 6 years and older
Conditions the study is for
Prader-Willi Syndrome
What is being tested?
Pitolisant tablet
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
134 participants
Where
57 sitesin 13 countries
Study dates
28 May 2024to Apr 2028
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 6 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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

Trial locations and contacts

57 sites in 13 countries. Contact the study team or a site near you to ask about taking part.

Ann Adee contact

773-383-6258

clinicaltrials@harmonybiosciences.com

Linnea Ryan contact

clinicaltrials@harmonybiosciences.com

57 sites in 13 countries

United States23 sites, 19 recruiting

  • Atlanta Diabetes Associates

    Atlanta, Georgia, 30318

    Withdrawn

  • Emory University School of Medicine

    Atlanta, Georgia, 30322

    Gwendolyn Gunn, 404-778-8552, gwendolyn.gisiner.gunn@emory.edu

    Michael Gambello, MD, PhD

    Recruiting

  • Rare Disease Research

    Atlanta, Georgia, 30329

    Withdrawn

  • Colorado Children's Hospital

    Aurora, Colorado, 80045

    Nathan Holly, 920-264-4742, nathan.holly@childrenscolorado.org

    Shawn McCandless, MD

    Recruiting

  • Johns Hopkins University Hospital

    Baltimore, Maryland, 21205

    Anne Scheimann, MD, MBA

    Recruiting

  • Johns Hopkins Hospital

    Baltimore, Maryland, 21224

    Ann Scheimann, 410-955-8769, ascheima@jhmi.edu,

    Ann Scheimann, MD

    Recruiting

  • Maimonides Medical Center

    Brooklyn, New York, 11219

    Hasan Mustafic, 718-283-8170, HMustafic@maimo.org,

    Edward Yi, MD

    Recruiting

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

    Chicago, Illinois, 60611

    Sarayu Ratnam, PhD, 312-227-6617, sratnam@luriechildrens.org

    Priya Khanna, MD

    Recruiting

  • CTI Clinical Research Center

    Cincinnati, Ohio, 45212

    Withdrawn

  • Center for Human Genetics

    Cleveland, Ohio, 441016

    Mehjabeen Hossain, 216-869-9202, mehjabeen.hossain@uhhospitals.org

    Anna Mitchell, MD

    Recruiting

  • Texas Children's Hospital

    Houston, Texas, 77030

    Paushpala Sen, 832-822-1343, pxsen3@texaschildrens.org

    Ifeoma Ibekwe, icibekwe@texaschildrens.org

    Recruiting

  • Riley Children's Hospital

    Indianapolis, Indiana, 46202

    Julia Meyer, 317-278-7118, meyerj@iupui.edu

    Hasnaa Jalou, MD

    Recruiting

  • Santa Monica Clinical Trials

    Los Angeles, California, 90025

    Samantha Montenez, 310-586-0843, e. smontanez@smclinicaltrials.com

    Daniel Norman, MD

    Recruiting

  • Childrens Hospital of Wisconsin

    Milwaukee, Wisconsin, 53226

    Paula Engelking, 414-266-3289, pengelki@mcw.edu

    Donald Basel, MD

    Recruiting

  • Science 37 (at-home option)

    Morrisville, North Carolina, 27560

    Dr. Sara Trepanier, 310-929-8473, HarmonyPWS@science37.com

    Dr. Sara Trepanier

    Recruiting

  • Science 37

    Morrisville, North Carolina, 27560

    Sara Trepanier, MD

    Recruiting

  • Mayo Clinic-PPDS

    Rochester, Minnesota, 55905

    Brittany Kehoe, 507-293-2316, kehoe.brittany@mayo.edu

    Queenie Tan, MD

    Recruiting

  • Center of Excellence in Diabetes and Endocrinology

    Sacramento, California, 95821-2123

    Natalie Marlen, 279-210-9814, marlenn@sutterhealth.org

    Gnanagurudasan Prakasam, MD

    Recruiting

  • Road Runner Research

    San Antonio, Texas, 78249-3539

    Nancy Lopez, 210-949-0505, nlopez@rrresearchsa.com

    Tarak Patel, MD

    Recruiting

  • Rady Children's Hospital - Scan Diego

    San Diego, California, 92123

    Megan Warner, mwarner2@rchsd.org

    Rakesh Bhattacharjee, MD

    Recruiting

  • Montefiore Medical Center

    The Bronx, New York, 10467

    Eric Hollander, MD, eholland@montefiore.org

    Eric Hollander, MD

    Recruiting

  • Texas Valley Clinical Research

    Weslaco, Texas, 78596

    Terminated

  • Nemours Children's Hospital

    Wilmington, Delaware, 19803-3607

    Kimberly Renner, 302-651-6400, kimberly.renner@nemours.org

    Aaron Chidekel, MD

    Recruiting

Other trials in United States

Italy7 sites, 7 recruiting

  • University of Florence

    Florence, Tuscany, 50139

    Stefano Stagi, MD

    Recruiting

  • Azienda Ospedaliero Universitaria A Meyer

    Florence

    Stefano Stagi, stefano.stagi@unifi.it

    Stefano Stagi

    Recruiting

  • Istituto G Gaslini Ospedale Pediatrico IRCCS - INCIPIT - PIN

    Genova

    Mohamad Maghnie, MD, mohamadmaghnie@ospedale-gaslini.ge.it

    Mohamad Maghnie, MD

    Recruiting

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

    Milan

    Roberta Pajno, MD, pajno.roberta@hsr.it

    Roberta Pajno, MD

    Recruiting

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

    Padova

    Laura Guazzarotti, MD, guazza@yahoo.com

    Laura Guazzarotti, MD

    Recruiting

  • Ospedale Pediatrico Bambino Gesù IRCCS

    Roma

    Danilo Fintini, MD, danilo.fintini@opbg.net

    Danilo Fintini, MD

    Recruiting

  • IRCCS Materno Infantile Burlo Garofolo - INCIPIT - PIN

    Trieste

    Gianluca Tornese, MD, gianluca.tornese@burlo.trieste.it

    Gianluca Tornese, MD

    Recruiting

Other trials in Italy

Australia4 sites, 4 recruiting

  • Queensland Children's Hospital

    Brisbane, Queensland

    Megan Angliss, chq_pwsresearch@health.qld.gov.au

    Dr. Vikas Goyal

    Recruiting

  • Royal Prince Alfred Hospital

    Camperdown

    Tania Markovic, tania.markovic@sydney.edu.au

    Tania Markovic

    Recruiting

  • Sydney Children's Hospital

    Randwick

    Ohn Nyunt, schn-schclinicaltrials@health.nsw.gov.au

    Ohn Nyunt

    Recruiting

  • Children's Hospital at Westmead

    Westmead

    Yoon Hi Cho, yoonhi.cho@health.nsw.gov.au

    Yoon Hi Cho

    Recruiting

Other trials in Australia

Romania4 sites, 4 recruiting

  • Institutul National de Endocrinologie C. I. Parhon

    Bucharest

    Corin Badiu, MD, badicrin@yahoo.co.uk

    Corin Badiu, MD

    Recruiting

  • Institutul National de Endocrinologie C. I. Parhon

    Bucharest

    Iuliana Gherlan, MD, iuliana.gherlan77@gmail.com

    Iuliana Gherlan

    Recruiting

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

    Bucharest

    Madalina Leanca, MD, mada_mada332@yahoo.com

    Madalina Leanca, MD

    Recruiting

  • Louis Turcanu Emergency Clinical Hospital for Children

    Timișoara

    Otilia Marginean, MD, omarginean@ymail.com

    Otilia Marginean, MD

    Recruiting

Other trials in Romania

Spain4 sites, 4 recruiting

  • Corporacio Sanitaria Parc Tauli, Sabadell

    Barcelona

    Raquel Corripio Collado, rcorripio@tauli.cat

    Raquel Corripio Collado

    Recruiting

  • Hospital Sant Joan de Deu

    Barcelona

    Oscar Sans Capdevila, oscarramon.sans@sjd.es

    Oscar Sans Capdevila

    Recruiting

  • Hospital Universitario 12 de Octubre

    Madrid

    Irene Lazaro Rodriguez, irene.lazaro@salud.madrid.org

    Irene Lazaro Rodriguez

    Recruiting

  • Universitario Virgen de la Victoria

    Málaga

    Francisco Madueno Tinahones, fjtinahones@hotmail.com

    Francisco Madueno Tinahones

    Recruiting

Other trials in Spain

United Kingdom4 sites, 4 recruiting

  • Fulbourn Hospital

    Cambridge, England

    Shahid Zaman, MD, shahid.zaman@cpft.nhs.uk

    Shahid Zaman, MD

    Recruiting

  • Ninewells Hospital - PPDS

    Dundee, Scotland

    Clare Webster, MD, clare.webster@nhs.scot

    Clare Webster, MD

    Recruiting

  • Royal Hospital for Children and Young People

    Edinburgh, Scotland

    Kathryn Cox, MD, kathryn.cox@nhslothian.scot.nhs.uk

    Kathryn Cox, MD

    Recruiting

  • Hull Royal Infirmary

    Hull, England

    Thozhukat Sathyapalan, MBBS, MD, thozhukat.sathyapalan@hyms.ac.uk

    Thozhukat Sathyapalan, MBBS, MD

    Recruiting

Other trials in United Kingdom

Canada2 sites, 1 recruiting

  • AMNDX Inc.

    Thornhill, Ontario

    Urszula Lamoureux, 416-435-6244, urszula.research@gmail.com

    Alan Lowe, MD

    Recruiting

  • Jodha Tishon Inc.

    Toronto, Ontario

    Withdrawn

Other trials in Canada

Denmark2 sites, 2 recruiting

  • Aarhus University Hospital

    Aarhus

    Cecilie Ejerskov, ceej@clin.au.dk

    Cecilie Ejerskov

    Recruiting

  • Rigshospitalet

    Copenhagen

    Stense Farholt, MD, stense.farholt@regionh.dk

    Stense Farholt, MD

    Recruiting

Other trials in Denmark

France2 sites, 2 recruiting

  • CHU d'Angers

    Angers

    Frederic Illouz, MD, frillouz@chu-angers.fr

    Frederic Illouz, MD

    Recruiting

  • CHU de Toulouse-Hôpital Des Enfants

    Toulouse

    Gwenaëlle Diene, diene.g@chu-toulouse.fr

    Gwenaëlle Diene

    Recruiting

Other trials in France

Poland2 sites, 2 recruiting

  • Samodzielny Publiczny Szpital Kliniczny

    Szczecin

    Elzbieta Petriczko, elzbietapetriczko@gmail.com

    Elzbieta Petriczko

    Recruiting

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

    Wroclaw

    Beata Wikiera, MD, wikierab@wp.pl

    Beata Wikiera, MD

    Recruiting

Other trials in Poland

Belgium1 site, 1 recruiting

  • UZ Brussels

    Jette

    Tessa Wassenberg, tessa.wassenberg@uzbrussel.be

    Tessa Wassenberg

    Recruiting

Other trials in Belgium

Germany1 site, 1 recruiting

  • University Hospital Essen

    Essen

    Cordula Kiewert, MD, cordula.kiewert@uk-essen.de

    Cordula Kiewert, MD

    Recruiting

Other trials in Germany

Sweden1 site, 1 recruiting

  • Karolinska Universitetssjukhuset Solna

    Solna

    Charlotte Höybye, charlotte.hoybye@regionstockholm.se

    Charlotte Höybye

    Recruiting

Other trials in Sweden

About this study

Official title: 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

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

Read the detailed description

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.

Being tested
Pitolisant tablet, Placebo tablet
Keywords
pitolisant, excessive daytime sleepiness, irritable and disruptive behaviors, Prader-Willi syndrome
Medical subject headings
Prader-Willi Syndrome, Disorders of Excessive Somnolence, Problem Behavior

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Pitolisant tabletDrug

    Also called pitolisant

    Pitolisant tablet

  • Placebo tabletOther

    Also called placebo

    Placebo tablet

Study groups in NCT06366464
Study groupRoleWhat they receive
Double-Blind Treatment Period PitolisantExperimentalPitolisant tablets administered once daily in the morning upon wakening
Receives: Drug: Pitolisant tablet
Double-Blind Treatment Period PlaceboPlacebo comparatorMatching placebo administered tablets once daily in the morning upon wakening
Receives: Other: Placebo tablet
Open-Label Extension Period PitolisantOtherPitolisant tablets administered once daily in the morning upon wakening
Receives: Drug: Pitolisant tablet

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change in severity of EDS as measured by Patient-Reported Outcomes Measurement Information System Bank v1.0 - Sleep-Related Impairment (PROMIS-SRI) T-score

    The PROMIS-SRI item bank consists of 13 items with a 5-point rating scale.

    Measured: Baseline and end of the Double Blind Treatment Period (Day 77)

Secondary outcomes (8)

  1. Change in severity of irritable and disruptive behaviors as measured by the Aberrant Behavior Checklist-Community, Second Edition (ABC-C) Irritability domain

    The ABC-C is a 58-item questionnaire, divided into 5 subscales (Irritability, Social Withdrawal, Stereotypic Behavior, Hyperactive/Noncompliance, and Inappropriate Speech).

    Measured: Baseline and end of the Double Blind Treatment Period (Day 77)

  2. Change in overall severity of EDS as measured by the Caregiver Global Impression of Severity for Excessive Daytime Sleepiness (CaGI-S for EDS)

    The CaGI-S for EDS is a 1-item, 5-point rating scale.

    Measured: Baseline and end of the Double Blind Treatment Period (Day 77)

  3. Change in overall severity of EDS as measured by the Clinical Global Impression of Severity for Excessive Daytime Sleepiness (CGI-S for EDS)

    The CGI-S for EDS is a 1-item, 5-point rating scale.

    Measured: Baseline and end of the Double Blind Treatment Period (Day 77)

  4. Change in overall severity of irritable and disruptive behaviors as measured by the Caregiver Global Impression of Severity (CaGI-S) for Irritable and/or Disruptive Behaviors

    The CaGI-S for Irritable and/or Disruptive Behaviors is a 1-item, 5-point rating scale.

    Measured: Baseline and end of the Double Blind Treatment Period (Day 77)

  5. Change in severity of hyperphagia as measured by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT), in conjunction with the Food Safe Zone Questionnaire (FSZQ)

    The HQ-CT is a 9-item measure of food-related preoccupations and problems.

    The FSZQ is a 20-item measure of environmental controls to manage hyperphagia.

    Measured: Baseline and end of the Double Blind Treatment Period (Day 77)

  6. Change in severity of EDS as measured by the Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD [parent/caregiver version]) total score

    The ESS-CHAD (parent/caregiver version) is an 8-item, 4-point rating scale.

    Measured: Baseline and end of the Double Blind Treatment Period (Day 77)

  7. Change in severity of other behavioral problems as measured by the Aberrant Behavior Checklist-Community, Second Edition (ABC-C) Hyperactivity/Noncompliance, Inappropriate Speech, Social Withdrawal, and Stereotypic Behavior Domains

    The ABC-C is a 58-item questionnaire, divided into 5 subscales (Irritability, Social Withdrawal, Stereotypic Behavior, Hyperactive/Noncompliance, and Inappropriate Speech).

    Measured: Baseline and end of the Double Blind Treatment Period (Day 77)

  8. Percentage of patients reporting TEAEs

    A treatment-emergent adverse events is any adverse event reported after the first dose of study drug and up to 30 days after final dose of study drug, or any worsening of a pre-existing condition reported after first dose of study drug and up to 30 days after final dose of study drug.

    Measured: Baseline up to Day 441

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Apr 2024
  2. Study start28 May 2024
  3. Primary completionApr 2027 (estimated)
  4. Study completionApr 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
134 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06366464
Sponsor's protocol ID
HBS-101-CL-312
Status last verified
Aug 2026
First submitted
9 Oct 2023
Last updated
25 Aug 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Pitolisant tablet in people with Prader-Willi Syndrome, run by Harmony Biosciences Management, Inc. at 57 sites in 13 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 25 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 57 sites in United States, Italy, Australia, Romania, Spain and 8 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Ann Adee (773-383-6258, clinicaltrials@harmonybiosciences.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change in severity of EDS as measured by Patient-Reported Outcomes Measurement Information System Bank v1.0 - Sleep-Related Impairment (PROMIS-SRI) T-score (time frame: Baseline and end of the Double Blind Treatment Period (Day 77)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06366464.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Aug 2026.

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