Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

PHASE2/PHASE3CompletedINTERVENTIONAL
Enrollment

56

Participants

Timeline

Start Date

March 3, 2021

Primary Completion Date

April 4, 2023

Study Completion Date

April 4, 2023

Conditions
Congenital Myotonic Dystrophy
Interventions
DRUG

Tideglusib

Tideglusib for oral suspension, weight-adjusted at 400mg, 600mg or 1000 mg dose levels, once daily

DRUG

Placebo

Matching placebo formulation

Trial Locations (14)

1010

New Zealand Clinical Research (NZCR), Auckland

2031

The Bright Alliance, Randwick

14642

University of Rochester Medical Center, Rochester

15213

University of Pittsburgh Medical Center, Pittsburgh

23219

Virginia Commonwealth University - Department of Neurology. Muscular Dystrophy Translational Research Program., Richmond

52242

University of Iowa Hospitals and Clinics, Iowa City

60611

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago

72202

Arkansas Children's Hospital, Little Rock

84112

University of Utah Hospital, Salt Lake City

90095

University of California, Los Angeles (UCLA), Los Angeles

94304

Stanford University, Palo Alto

N6A4G5

Children's Hospital London Health Sciences Centre (LHSC), London

K1H 8L1

Children's Hospital of Eastern Ontario, Ottawa

NE2 4HH

Newcastle University, Newcastle upon Tyne

Sponsors

Lead Sponsor

All Listed Sponsors
lead

AMO Pharma Limited

INDUSTRY

NCT03692312 - Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy | Biotech Hunter | Biotech Hunter