Evaluating pitolisant for excessive daytime sleepiness in Prader-Willi syndrome

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 3 clinical study investigates the efficacy and safety of pitolisant in treating excessive daytime sleepiness in patients aged 6 and older with Prader-Willi syndrome. The study is randomized, double-blind, and placebo-controlled, involving multiple centers globally. Participants will undergo a screening period followed by a treatment phase where they will receive either pitolisant or a placebo. Secondary objectives include assessing the drug's impact on behavioral issues such as irritability and hyperphagia.

Why it matters

Who can join: the full rules

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

Where it's running

• Santa Monica Clinical Trials
  Los Angeles, California, United States Recruiting
• Center of Excellence in Diabetes and Endocrinology
  Sacramento, California, United States Recruiting
• Rady Children's Hospital - Scan Diego
  San Diego, California, United States Recruiting
• Colorado Children's Hospital
  Aurora, Colorado, United States Recruiting
• Nemours Children's Hospital
  Wilmington, Delaware, United States Recruiting
• Emory University School of Medicine
  Atlanta, Georgia, United States Recruiting

+7 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Ann Adee
• Email: clinicaltrials@harmonybiosciences.com
• Phone: 773-383-6258

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Using vagus nerve stimulation to improve emotion and thinking in Prader-Willi Syndrome

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.