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

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 Myotonic Dystrophy Type 1 ages 18 to 65 years. The secondary objectives of this study are to assess the impact of pitolisant on fatigue, cognitive function and the burden of disease along with assessing the long-term safety and effectiveness of pitolisant in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years.

Timeline

Start

2021-06-28

Primary completion

2023-10-06

Completion

2024-10-31

Publications

• D'Ambrosio ES, Chuang K, David WS, Amato AA, Gonzalez-Perez P. Frequency and type of cancers in myotonic dystrophy: A retrospective cross-sectional study. Muscle Nerve. 2023 Aug;68(2):142-148. doi: 10.1002/mus.27801. Epub 2023 Mar 27.

Drugs

Indications