Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy

PHASE2/PHASE3RecruitingINTERVENTIONAL
Enrollment

76

Participants

Timeline

Start Date

August 23, 2021

Primary Completion Date

December 31, 2026

Study Completion Date

December 31, 2026

Conditions
Congenital Myotonic Dystrophy
Interventions
DRUG

Tideglusib

Tideglusib dosing will be weight-adjusted at 400 mg, 600 mg, or 1000 mg dose levels, or weight banded fixed doses of 400 mg, 600 mg, 800 mg or 1000 mg, with each subject starting at a weight-adjusted 400 mg dose level for 2 weeks, then up titrating to a weight-adjusted 600 mg dose level for the next 2 weeks.

Trial Locations (14)

1010

COMPLETED

New Zealand Clinical Research (NZCR), Auckland

2031

RECRUITING

The Bright Alliance, Randwick

14642

RECRUITING

University of Rochester - Medical Center, Rochester

15213

RECRUITING

University of Pittsburgh Medical Center, Pittsburgh

23219

COMPLETED

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

23507

WITHDRAWN

Children's Hospital of The King's Daughters, Norfolk

52242

RECRUITING

University of Iowa Hospitals and Clinics, Iowa City

60611

RECRUITING

Lurie's Children's Hospital, Chicago

72202

RECRUITING

Arkansas Children's Hospital, Little Rock

84132

RECRUITING

University of Utah Clinical Neurosciences Center, Salt Lake City

90095

ENROLLING_BY_INVITATION

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

94304

ENROLLING_BY_INVITATION

Stanford University, Palo Alto

N6A 4G5

ENROLLING_BY_INVITATION

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

K1H 8L1

RECRUITING

Children's Hospital of Eastern Ontario, Ottawa

Sponsors

Lead Sponsor

All Listed Sponsors
lead

AMO Pharma Limited

INDUSTRY

NCT05004129 - Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy | Biotech Hunter | Biotech Hunter