A Study of Pitolisant in Patients With Prader-Willi Syndrome

Prader-Willi Syndrome Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06366464
• Other study ID #: HBS-101-CL-312
• Status: Recruiting
• Phase: Phase 3
• Start date: April 30, 2024
• Est. completion date: July 2027

Study information

• Verified date: May 2024
• Source: Harmony Biosciences, LLC
• Contact: Ann Adee
• Phone: 773-383-6258
• Email: clinicaltrials[@]harmonybiosciences.com
• Is FDA regulated: No
• Study type: Interventional

Clinical 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.

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

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 information / eligibility

• Status: Recruiting
• Enrollment: 134
• Est. completion date: July 2027
• Est. primary completion date: July 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Prader-Willi Syndrome
• Syndrome

Intervention

Drug:
• Pitolisant tablet
Pitolisant tablet

Other:
• Placebo tablet
Placebo tablet

Locations

Country	Name	City	State
United States	Atlanta Diabetes Associates	Atlanta	Georgia
United States	Santa Monica Clinical Trials	Los Angeles	California
United States	Texas Valley Clinical Research	Weslaco	Texas

Sponsors (1)

Lead Sponsor	Collaborator
Harmony Biosciences, LLC

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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).	Baseline and end of the Double Blind Treatment Period (Day 77)
Secondary	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.	Baseline and end of the Double Blind Treatment Period (Day 77)
Secondary	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.	Baseline and end of the Double Blind Treatment Period (Day 77)
Secondary	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.	Baseline and end of the Double Blind Treatment Period (Day 77)
Secondary	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.	Baseline and end of the Double Blind Treatment Period (Day 77)
Secondary	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.	Baseline and end of the Double Blind Treatment Period (Day 77)
Secondary	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).	Baseline and end of the Double Blind Treatment Period (Day 77)
Secondary	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.	Baseline up to Day 441