Clinical trial NCT06366464: A Study of Pitolisant in Patients With Prader-Willi Syndrome
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Prader-Willi Syndrome
Keywords: pitolisant
excessive daytime sleepiness
irritable and disruptive behaviors
Prader-Willi syndrome
Lead sponsor: Harmony Biosciences Management, Inc.
Sponsor class: INDUSTRY
First posted: 2024-04-16
Enrollment: 134
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
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
timeFrame: Baseline and end of the Double Blind Treatment Period (Day 77)
Secondary outcomes:
measure: Change in severity of irritable and disruptive behaviors as measured by the Aberrant Behavior Checklist-Community, Second Edition (ABC-C) Irritability domain
timeFrame: Baseline and end of the Double Blind Treatment Period (Day 77)
measure: Change in overall severity of EDS as measured by the Caregiver Global Impression of Severity for Excessive Daytime Sleepiness (CaGI-S for EDS)
timeFrame: Baseline and end of the Double Blind Treatment Period (Day 77)
measure: Change in overall severity of EDS as measured by the Clinical Global Impression of Severity for Excessive Daytime Sleepiness (CGI-S for EDS)
timeFrame: Baseline and end of the Double Blind Treatment Period (Day 77)
measure: 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
timeFrame: Baseline and end of the Double Blind Treatment Period (Day 77)
measure: 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)
timeFrame: Baseline and end of the Double Blind Treatment Period (Day 77)
measure: Change in severity of EDS as measured by the Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD [parent/caregiver version]) total score
timeFrame: Baseline and end of the Double Blind Treatment Period (Day 77)
measure: 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
timeFrame: Baseline and end of the Double Blind Treatment Period (Day 77)
measure: Percentage of patients reporting TEAEs
timeFrame: Baseline up to Day 441
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Santa Monica Clinical Trials
state: California
city: Sacramento
country: United States
facility: Center of Excellence in Diabetes and Endocrinology
state: California
city: San Diego
country: United States
facility: Rady Children's Hospital - Scan Diego
state: California
city: San Ramon
country: United States
facility: Tri-Valley Sleep Center
state: California
city: Aurora
country: United States
facility: Colorado Children's Hospital
state: Colorado
city: Wilmington
country: United States
facility: Nemours Children's Hospital
state: Delaware
city: Atlanta
country: United States
facility: Atlanta Diabetes Associates
state: Georgia
city: Atlanta
country: United States
facility: Emory University School of Medicine
state: Georgia
city: Atlanta
country: United States
facility: Rare Disease Research
state: Georgia
city: Chicago
country: United States
facility: Ann And Robert H Lurie Children's Hospital of Chicago
state: Illinois
city: Indianapolis
country: United States
facility: Riley Children's Hospital
state: Indiana
city: Baltimore
country: United States
facility: Johns Hopkins Hospital
state: Maryland
city: Rochester
country: United States
facility: Mayo Clinic-PPDS
state: Minnesota
city: Bronx
country: United States
facility: Montefiore Medical Center
state: New York
city: Brooklyn
country: United States
facility: Maimonides Medical Center
state: New York
city: Morrisville
country: United States
facility: Science 37 (at-home option)
state: North Carolina
city: Cincinnati
country: United States
facility: CTI Clinical Research Center
state: Ohio
city: Cleveland
country: United States
facility: Center for Human Genetics
state: Ohio
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: San Antonio
country: United States
facility: Road Runner Research
state: Texas
city: Weslaco
country: United States
facility: Texas Valley Clinical Research
state: Texas
city: Milwaukee
country: United States
facility: Childrens Hospital of Wisconsin
state: Wisconsin
city: Brisbane
country: Australia
facility: Queensland Children's Hospital
state: Queensland
city: Camperdown
country: Australia
facility: Royal Prince Alfred Hospital
city: Randwick
country: Australia
facility: Sydney Children's Hospital
city: Westmead
country: Australia
facility: Children's Hospital at Westmead
city: Jette
country: Belgium
facility: UZ Brussels
city: Aarhus
country: Denmark
facility: Aarhus University Hospital
city: Toulouse
country: France
facility: CHU de Toulouse-Hôpital Des Enfants
city: Firenze
country: Italy
facility: Azienda Ospedaliero Universitaria A Meyer
city: Szczecin
country: Poland
facility: Samodzielny Publiczny Szpital Kliniczny
city: Barcelona
country: Spain
facility: Hospital Sant Joan de Deu
city: Madrid
country: Spain
facility: Hospital Universitario 12 de Octubre
city: Solna
country: Sweden
facility: Karolinska Universitetssjukhuset Solna
Study officials:
References: