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

PHASE2CompletedINTERVENTIONAL
Enrollment

30

Participants

Timeline

Start Date

June 28, 2021

Primary Completion Date

October 6, 2023

Study Completion Date

October 31, 2024

Conditions
Myotonic Dystrophy 1Excessive Daytime Sleepiness
Interventions
DRUG

Pitolisant Oral Tablet

"Pitolisant 4.45 mg tablets: white, round, plain, biconvex film-coated tablet, 3.7 mm in diameter. Each tablet contains 5 mg of pitolisant hydrochloride equivalent to 4.45 mg of pitolisant.~Pitolisant 17.8 mg tablets: white, round, plain, biconvex film-coated tablet, 7.5 mm in diameter. Each tablet contains 20 mg of pitolisant hydrochloride equivalent to 17.8 mg of pitolisant."

DRUG

Placebo oral tablet

Matching placebo tablets will be provided for each strength of active pitolisant film-coated tablets.

Trial Locations (13)

14642

University of Rochester Medical Center, Rochester

19104

University of Pennsylvania, Philadelphia

21205

Kennedy Krieger Institute Center for Genetic Muscle Disorders, Baltimore

27157

Wake Forest, Winston-Salem

27599

University of North Carolina, Chapel Hill

30329

Rare Disease Research, Atlanta

33612

University of South Florida, Tampa

46202

Indiana University, Indianapolis

55455

University of Minnesota, Minneapolis

80045

University of Colorado School of Medicine, Aurora

92697

UCI Center for Clinical Research, Irvine

Unknown

The Ottawa Hospital, Ottawa

Hôpital de Chicoutimi, Chicoutimi

All Listed Sponsors
lead

Harmony Biosciences Management, Inc.

INDUSTRY