Completed PHASE2 INTERVENTIONAL 3-arm NCT04886518

Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1

A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1, Followed by an Open-Label Extension

Sponsor: Harmony Biosciences Management, Inc.

Conditions Excessive Daytime Sleepiness Myotonic Dystrophy 1

Interventions Pitolisant Oral Tablet Placebo oral tablet

Updated 18 times since 2021 Last updated: Apr 7, 2025 Started: Jun 28, 2021 Primary completion: Oct 6, 2023 Completion: Oct 31, 2024

This information is for research purposes only and is not medical advice. Consult a healthcare provider before making any medical decision.

A PHASE2 clinical study on Excessive Daytime Sleepiness and Myotonic Dystrophy 1, this trial is completed. The trial is conducted by Harmony Biosciences Management, Inc. and has accumulated 18 data snapshots since 2021. Longitudinal tracking of this trial contributes to a broader understanding of treatment development timelines.

Status Flow

Change History

18 versions recorded

May 2025 — Present [monthly]

Completed PHASE2

Status: Active Not Recruiting → Completed
Mar 2025 — May 2025 [monthly]

Active Not Recruiting PHASE2
Sep 2024 — Mar 2025 [monthly]

Active Not Recruiting PHASE2
Jul 2024 — Sep 2024 [monthly]

Active Not Recruiting PHASE2
Mar 2024 — Jul 2024 [monthly]

Active Not Recruiting PHASE2

▶ Show 13 earlier versions

Sep 2023 — Mar 2024 [monthly]

Active Not Recruiting PHASE2

Status: Recruiting → Active Not Recruiting
Aug 2023 — Sep 2023 [monthly]

Recruiting PHASE2
Apr 2023 — Aug 2023 [monthly]

Recruiting PHASE2
Feb 2023 — Apr 2023 [monthly]

Recruiting PHASE2
Sep 2022 — Feb 2023 [monthly]

Recruiting PHASE2
Jun 2022 — Sep 2022 [monthly]

Recruiting PHASE2
May 2022 — Jun 2022 [monthly]

Recruiting PHASE2
Apr 2022 — May 2022 [monthly]

Recruiting PHASE2
Mar 2022 — Apr 2022 [monthly]

Recruiting PHASE2
Jan 2022 — Mar 2022 [monthly]

Recruiting PHASE2
Nov 2021 — Jan 2022 [monthly]

Recruiting PHASE2
Sep 2021 — Nov 2021 [monthly]

Recruiting PHASE2

Status: Not Yet Recruiting → Recruiting
Jun 2021 — Sep 2021 [monthly]

Not Yet Recruiting PHASE2

First recorded

Eligibility Summary

No eligibility information available.

Contact Information

Sponsor contact:

Harmony Biosciences Management, Inc.

Data source: Harmony Biosciences Management, Inc.

For direct contact, visit the study record on ClinicalTrials.gov .

Study Locations

Atlanta, United States , Aurora, United States , Baltimore, United States , Chapel Hill, United States , Chicoutimi, Canada , Indianapolis, United States , Irvine, United States , Minneapolis, United States , Ottawa, Canada , Philadelphia, United States and 3 more locations