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Changes Announced to Make Participation in Pitolisant Study Easier

Harmony Biosciences recently announced three changes to their Clinical Trial of Pitolisant that reduce the requirements of participating in the study.

Harmony Biosciences recently announced changes to their Phase 2 Clinical Trial of Pitolisant that reduce the requirements of participating in the study.

Changes-announced-to-make-participation-in-Pitolisant-study-easier-Research-featureThree important changes have been made to the trial to make participation easier:

  1. No overnight or daytime sleep studies are needed

  2. A remote screening visit may be allowed

  3. More flexibility of remote visits during both the Double-Blind study and the optional Open-Label extension phase

In addition to evaluating the safety of Pitolisant and its effectiveness in treating excessive daytime sleepiness, the trial will measure how Pitolisant affects learning, attention, memory, and hunger.

Sixty volunteers, ages 6-65 with PWS, are needed for the study. Volunteers will be randomly split into three groups: 1 group will be on a low dose, 1 group will be on a high dose, and 1 group will receive a placebo for the duration of the study. At the conclusion of the study, all participants have the option of continuing in the Open-Label extension and receiving Pitolisant.

Participation in the trial lasts approximately 4 months and includes 5 visits (2 of which may be completed in your home).

Harmony recently presented information about the trial in this short 30-minute webinar:


Individuals interested in participating in the study are encouraged to contact one of the 15 sites located in the US. Click here and scroll mid-way down the page for contact information for each site.

Learn more about PWS Clinical Trials

FPWR Enewsletter

Topics: Research

Susan Hedstrom


Susan Hedstrom is the Executive Director for the Foundation for Prader-Willi Research. Passionate about finding treatments for PWS, Susan joined FPWR in 2009 shortly after her son, Jayden, was diagnosed with Prader-Willi Syndrome. Rather than accepting PWS as it has been defined, Susan has chosen to work with a team of pro-active and tireless individuals to accelerate PWS research in order to change the future of PWS. Inspired by her first FPWR conference and the team of researchers that were working to find answers for the syndrome, she joined the FPWR team in 2010 and led the development of the One SMALL Step walk program. Under Susan’s leadership, over $15 million has been raised for PWS related research.