A Study of Pitolisant in Patients With Prader-Willi Syndrome

NCT06366464 | Recruiting Phase 3 FDA Drug

• 1 other identifier: HBS-101-CL-312
• Study Type: interventional
• Target: 134
• Locations: 13 countries
• Sites: 54

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

Conditions

Prader-Willi Syndrome

Keywords

pitolisant; excessive daytime sleepiness; irritable and disruptive behaviors; Prader-Willi syndrome

Outcome Measures

Primary Outcomes (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.

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

Secondary Outcomes (8)

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

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

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

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

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

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

• 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

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

• 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)

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

Study Arms (3)

Double-Blind Treatment Period Pitolisant

EXPERIMENTAL

Pitolisant tablets administered once daily in the morning upon wakening

Drug: Pitolisant tablet

Double-Blind Treatment Period Placebo

PLACEBO COMPARATOR

Matching placebo administered tablets once daily in the morning upon wakening

Other: Placebo tablet

Open-Label Extension Period Pitolisant

OTHER

Pitolisant tablets administered once daily in the morning upon wakening

Drug: Pitolisant tablet

Interventions

Pitolisant tablet DRUG

Pitolisant tablet

Also known as: pitolisant

Double-Blind Treatment Period Pitolisant; Open-Label Extension Period Pitolisant

Placebo tablet OTHER

Placebo tablet

Also known as: placebo

Double-Blind Treatment Period Placebo

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Harmony Biosciences Management, Inc.: lead

Study Sites (54)

Santa Monica Clinical Trials

Los Angeles, California, 90025, United States

RECRUITING

Center of Excellence in Diabetes and Endocrinology

Sacramento, California, 95821-2123, United States

RECRUITING

Rady Children's Hospital - Scan Diego

San Diego, California, 92123, United States

RECRUITING

Colorado Children's Hospital

Aurora, Colorado, 80045, United States

RECRUITING

Nemours Children's Hospital

Wilmington, Delaware, 19803-3607, United States

RECRUITING

Atlanta Diabetes Associates

Atlanta, Georgia, 30318, United States

WITHDRAWN

Emory University School of Medicine

Atlanta, Georgia, 30322, United States

RECRUITING

Rare Disease Research

Atlanta, Georgia, 30329, United States

WITHDRAWN

Ann And Robert H Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

RECRUITING

Riley Children's Hospital

Indianapolis, Indiana, 46202, United States

RECRUITING

Johns Hopkins Hospital

Baltimore, Maryland, 21224, United States

RECRUITING

Mayo Clinic-PPDS

Rochester, Minnesota, 55905, United States

RECRUITING

Maimonides Medical Center

Brooklyn, New York, 11219, United States

RECRUITING

Montefiore Medical Center

The Bronx, New York, 10467, United States

RECRUITING

Science 37 (at-home option)

Morrisville, North Carolina, 27560, United States

RECRUITING

CTI Clinical Research Center

Cincinnati, Ohio, 45212, United States

WITHDRAWN

Center for Human Genetics

Cleveland, Ohio, 441016, United States

RECRUITING

Texas Children's Hospital

Houston, Texas, 77030, United States

RECRUITING

Road Runner Research

San Antonio, Texas, 78249-3539, United States

RECRUITING

Texas Valley Clinical Research

Weslaco, Texas, 78596, United States

TERMINATED

Childrens Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States

RECRUITING

Queensland Children's Hospital

Brisbane, Queensland, Australia

RECRUITING

Royal Prince Alfred Hospital

Camperdown, Australia

RECRUITING

Sydney Children's Hospital

Randwick, Australia

RECRUITING

Children's Hospital at Westmead

Westmead, Australia

RECRUITING

UZ Brussels

Jette, Belgium

RECRUITING

AMNDX Inc.

Thornhill, Ontario, Canada

RECRUITING

Jodha Tishon Inc.

Toronto, Ontario, Canada

RECRUITING

Aarhus University Hospital

Aarhus, Denmark

RECRUITING

Rigshospitalet

Copenhagen, Denmark

RECRUITING

CHU d'Angers

Angers, France

RECRUITING

CHU de Toulouse-Hôpital Des Enfants

Toulouse, France

RECRUITING

University Hospital Essen

Essen, Germany

RECRUITING

Azienda Ospedaliero Universitaria A Meyer

Florence, Italy

RECRUITING

Istituto G Gaslini Ospedale Pediatrico IRCCS - INCIPIT - PIN

Genova, Italy

RECRUITING

Ospedale San Raffaele S.r.l. - PPDS

Milan, Italy

RECRUITING

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

Padua, Italy

RECRUITING

Ospedale Pediatrico Bambino Gesù IRCCS

Roma, Italy

RECRUITING

IRCCS Materno Infantile Burlo Garofolo - INCIPIT - PIN

Trieste, Italy

RECRUITING

Samodzielny Publiczny Szpital Kliniczny

Szczecin, Poland

RECRUITING

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

Wroclaw, Poland

RECRUITING

Institutul National de Endocrinologie C. I. Parhon

Bucharest, Romania

RECRUITING

Institutul National de Endocrinologie C. I. Parhon

Bucharest, Romania

RECRUITING

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

Bucharest, Romania

RECRUITING

Louis Turcanu Emergency Clinical Hospital for Children

Timișoara, Romania

RECRUITING

Corporacio Sanitaria Parc Tauli, Sabadell

Barcelona, Spain

RECRUITING

Hospital Sant Joan de Deu

Barcelona, Spain

RECRUITING

Hospital Universitario 12 de Octubre

Madrid, Spain

RECRUITING

Universitario Virgen de la Victoria

Málaga, Spain

RECRUITING

Karolinska Universitetssjukhuset Solna

Solna, Sweden

RECRUITING

Fulbourn Hospital

Cambridge, England, United Kingdom

RECRUITING

Hull Royal Infirmary

Hull, England, United Kingdom

RECRUITING

Ninewells Hospital - PPDS

Dundee, Scotland, United Kingdom

RECRUITING

Royal Hospital for Children and Young People

Edinburgh, Scotland, United Kingdom

RECRUITING

MeSH Terms

Conditions

Prader-Willi Syndrome; Disorders of Excessive Somnolence; Problem Behavior

Interventions

pitolisant

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders; Mental Disorders; Behavioral Symptoms; Behavior; Child Behavior

Central Study Contacts

Ann Adee

CONTACT

773-383-6258; clinicaltrials@harmonybiosciences.com

Linnea Ryan

CONTACT

clinicaltrials@harmonybiosciences.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 9, 2023
• First Posted: April 16, 2024
• Study Start: May 28, 2024
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): July 1, 2027
• Last Updated: March 17, 2026

Record last verified: 2026-03

Locations

BE (1); US (21); DK (2); IT (6); FR (2); PL (2); AU (4); CA (2); DE (1); RO (4); SE (1); GB (4); ES (4)