New drug pitolisant tested for safety in rare genetic disorder
NCT ID NCT07219485
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 3 trial is testing the safety of pitolisant in 150 people with Prader-Willi syndrome who have already taken the drug in a previous study. Participants will receive pitolisant tablets and be monitored for side effects. The goal is to see if pitolisant is safe for long-term use in this condition.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- pitolisant
- What this could lead to
- If successful, this study could confirm that pitolisant is safe for long-term use in Prader-Willi syndrome, potentially leading to a new treatment option to help manage symptoms.
- What could go wrong
- This is an open-label safety study, not a placebo-controlled trial, so results may be less definitive. Side effects are possible, and the drug may not work for everyone.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Science 37
Morrisville, North Carolina, 27560, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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