HomeTrialNCT04257929
NCT04257929Phase 2ACTIVE NOT RECRUITING

A Phase 2 Study to Evaluate the Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome, Followed by an Open Label Extension

Study Summary

The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Prader Willi syndrome (PWS) ages 6 to 65 years.

View on ClinicalTrials.gov ↗
Study Details
Enrollment65 participants
Primary Completion
Lead SponsorHarmony Biosciences Management, Inc.
Data Retrieved2026-04-11 04:15:56.573671+00:00
What This Trial Means for Biotech Investors
Trial Phase Context

Phase 2 trials begin testing efficacy in a larger patient group (100–300). This is often where biotech binary events occur — positive Phase 2 data can significantly advance a company's pipeline narrative, while failures can be terminal for a program.

Status & Expected Data

Enrollment is complete. The trial is in its follow-up or data collection phase — a readout may occur on or near the primary completion date.

Enrolled: 65 participants
Sponsor Catalyst Profile

Harmony Biosciences (HRMY) is the sponsoring company for this trial. BiotechSign currently grades this company B (77/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

Not financial advice. BiotechSign data is sourced from ClinicalTrials.gov, SEC EDGAR, and other public databases for research and informational purposes only. It does not constitute investment recommendations. Always do your own due diligence before making investment decisions.