Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
Start Date
8/23/2021
Completion Date
12/31/2026
Summary
This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who are treatment naïve.
Detailed Description
This is an open-label study of either a weight-adjusted 1000 mg fixed dose or a weight banded fixed dose of tideglusib across a 52-week treatment period with an open-ended optional extended access period. The subjects are children and adolescents with Congenital DM1 who participated in the antecedent AMO-02-MD-2-003 study or individuals with either Congenital or Childhood onset DM1 who are treatment naïve.
Eligibility Criteria
Age Range: 6 years to 45 years
Interventions
Tideglusib
Conditions
Locations
Arkansas Children's Hospital
Little Rock, Arkansas 72202
United States
University of California, Los Angeles (UCLA)
Los Angeles, California 90095
United States
Stanford University
Palo Alto, California 94304
United States
Lurie's Children's Hospital
Chicago, Illinois 60611
United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa 52242
United States
University of Rochester - Medical Center
Rochester, New York 14642
United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania 15213
United States
University of Utah Clinical Neurosciences Center
Salt Lake City, Utah 84132
United States
Children's Hospital of The King's Daughters
Norfolk, Virginia 23507
United States
Virginia Commonwealth University-Department of Neurology - Muscular Dystrophy Translational Research Program
Richmond, Virginia 23219
United States
The Bright Alliance
Randwick, New South Wales 2031
Australia
Children's Hospital London Health Sciences Centre (LHSC)
London, Ontario N6A 4G5
Canada
Children's Hospital of Eastern Ontario
Ottawa, Ontario K1H 8L1
Canada
New Zealand Clinical Research (NZCR)
Auckland, 1010
New Zealand