A Study of Pitolisant in Patients with Prader-Willi Syndrome
- Conditions
- Prader-Willi Syndrome
- Interventions
- Other: Placebo tablet
- Registration Number
- NCT06366464
- Lead Sponsor
- Harmony Biosciences Management, Inc.
- 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
- 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 134
- 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.
- 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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Double-Blind Treatment Period Pitolisant Pitolisant tablet Pitolisant tablets administered once daily in the morning upon wakening Double-Blind Treatment Period Placebo Placebo tablet Matching placebo administered tablets once daily in the morning upon wakening Open-Label Extension Period Pitolisant Pitolisant tablet Pitolisant tablets administered once daily in the morning upon wakening
- Primary Outcome Measures
Name Time Method Change in severity of EDS as measured by Patient-Reported Outcomes Measurement Information System Bank v1.0 - Sleep-Related Impairment (PROMIS-SRI) T-score Baseline and end of the Double Blind Treatment Period (Day 77) The PROMIS-SRI item bank consists of 13 items with a 5-point rating scale.
- Secondary Outcome Measures
Name Time Method 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) The ABC-C is a 58-item questionnaire, divided into 5 subscales (Irritability, Social Withdrawal, Stereotypic Behavior, Hyperactive/Noncompliance, and Inappropriate Speech).
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) The CaGI-S for EDS is a 1-item, 5-point rating scale.
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) The CGI-S for EDS is a 1-item, 5-point rating scale.
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) The CaGI-S for Irritable and/or Disruptive Behaviors is a 1-item, 5-point rating scale.
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) 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.Change in severity of EDS as measured by the Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD [parent/caregiver version]) total score Baseline and end of the Double Blind Treatment Period (Day 77) The ESS-CHAD (parent/caregiver version) is an 8-item, 4-point rating scale.
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 Baseline and end of the Double Blind Treatment Period (Day 77) The ABC-C is a 58-item questionnaire, divided into 5 subscales (Irritability, Social Withdrawal, Stereotypic Behavior, Hyperactive/Noncompliance, and Inappropriate Speech).
Percentage of patients reporting TEAEs Baseline up to Day 441 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.
Trial Locations
- Locations (22)
Santa Monica Clinical Trials
🇺🇸Los Angeles, California, United States
Center of Excellence in Diabetes and Endocrinology
🇺🇸Sacramento, California, United States
Colorado Children's Hospital
🇺🇸Aurora, Colorado, United States
Nemours Children's Hospital
🇺🇸Wilmington, Delaware, United States
Atlanta Diabetes Associates
🇺🇸Atlanta, Georgia, United States
Rady Children's Hospital - Scan Diego
🇺🇸San Diego, California, United States
Tri-Valley Sleep Center
🇺🇸San Ramon, California, United States
Emory University School of Medicine
🇺🇸Atlanta, Georgia, United States
Rare Disease Research
🇺🇸Atlanta, Georgia, United States
Ann And Robert H Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
Riley Children's Hospital
🇺🇸Indianapolis, Indiana, United States
Johns Hopkins Hospital
🇺🇸Baltimore, Maryland, United States
Mayo Clinic-PPDS
🇺🇸Rochester, Minnesota, United States
Montefiore Medical Center
🇺🇸Bronx, New York, United States
Maimonides Medical Center
🇺🇸Brooklyn, New York, United States
CTI Clinical Research Center
🇺🇸Cincinnati, Ohio, United States
Center for Human Genetics
🇺🇸Cleveland, Ohio, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States
Road Runner Research
🇺🇸San Antonio, Texas, United States
Texas Valley Clinical Research
🇺🇸Weslaco, Texas, United States
Childrens Hospital of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
Queensland Children's Hospital
🇦🇺Brisbane, Queensland, Australia