A Study of Pitolisant in Patients With Prader-Willi Syndrome
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Pitolisant tablet, Placebo tablet.
- Who it may be relevant to
- Registry conditions: Prader-Willi Syndrome. Basic parameters: from 6 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Australia, Belgium, Canada, Denmark +8
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
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
Overview
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.
Interventions
- Drug Pitolisant tablet
Pitolisant tablet - Other Placebo tablet
Placebo tablet
Primary outcome measures
- 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)]
Secondary outcome measures (8)
- Change in severity of irritable and disruptive behaviors as measured by the Aberrant Behavior Checklist-Community, Second Edition (ABC-C) Irritability domain [Time frame: 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) [Time frame: 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) [Time frame: 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 [Time frame: 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) [Time frame: Baseline and end of the Double Blind Treatment Period (Day 77)]
- Change in severity of EDS as measured by the Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD [parent/caregiver version]) total score [Time frame: Baseline and end of the Double Blind Treatment Period (Day 77)]
- 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 [Time frame: Baseline and end of the Double Blind Treatment Period (Day 77)]
- Percentage of patients reporting TEAEs [Time frame: Baseline up to Day 441]
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
United States · 21 centers
- Santa Monica Clinical Trials — Los Angeles
- Center of Excellence in Diabetes and Endocrinology — Sacramento
- Rady Children's Hospital - Scan Diego — San Diego
- Colorado Children's Hospital — Aurora
- Nemours Children's Hospital — Wilmington
- Atlanta Diabetes Associates — Atlanta
- Emory University School of Medicine — Atlanta
- Rare Disease Research — Atlanta
- … and 13 more centers
Italy · 6 centers
- Azienda Ospedaliero Universitaria A Meyer — Florence
- Istituto G Gaslini Ospedale Pediatrico IRCCS - INCIPIT - PIN — Genova
- Ospedale San Raffaele S.r.l. - PPDS — Milan
- Azienda Ospedale Università Padova - Dipartimento Salute della Donna e del Bambino - INCIP — Padova
- Ospedale Pediatrico Bambino Gesù IRCCS — Roma
- IRCCS Materno Infantile Burlo Garofolo - INCIPIT - PIN — Trieste
Australia · 4 centers
- Queensland Children's Hospital — Brisbane
- Royal Prince Alfred Hospital — Camperdown
- Sydney Children's Hospital — Randwick
- Children's Hospital at Westmead — Westmead
Romania · 4 centers
- Institutul National de Endocrinologie C. I. Parhon — Bucharest
- Institutul National de Endocrinologie C. I. Parhon — Bucharest
- National Clinical Center for Children Neurorehabilitation "Dr. Nicolae Robanescu" — Bucharest
- Louis Turcanu Emergency Clinical Hospital for Children — Timișoara
Spain · 4 centers
- Corporacio Sanitaria Parc Tauli, Sabadell — Barcelona
- Hospital Sant Joan de Deu — Barcelona
- Hospital Universitario 12 de Octubre — Madrid
- Universitario Virgen de la Victoria — Málaga
United Kingdom · 4 centers
- Fulbourn Hospital — Cambridge
- Hull Royal Infirmary — Hull
- Ninewells Hospital - PPDS — Dundee
- Royal Hospital for Children and Young People — Edinburgh
Canada · 2 centers
- AMNDX Inc. — Thornhill
- Jodha Tishon Inc. — Toronto
Denmark · 2 centers
- Aarhus University Hospital — Aarhus
- Rigshospitalet — Copenhagen
France · 2 centers
- CHU d'Angers — Angers
- CHU de Toulouse-Hôpital Des Enfants — Toulouse
Poland · 2 centers
- Samodzielny Publiczny Szpital Kliniczny — Szczecin
- Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu-Chalubinskiego — Wroclaw
Belgium · 1 center
- UZ Brussels — Jette
Germany · 1 center
- University Hospital Essen — Essen
Sweden · 1 center
- Karolinska Universitetssjukhuset Solna — Solna
Identifiers
NCT: NCT06366464 · HBS-101-CL-312