An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Tideglusib for the Treatment of Congenital or Childhood Onset DM1 (REACH CDM X)

Who is this study for? Patients with Congenital Myotonic Dystrophy
What treatments are being studied? Tideglusib
Status: Recruiting
Location: See all (14) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2/Phase 3
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Maximum Age: 45
Healthy Volunteers: f
View:

⁃ Subjects who do not enter this study directly from completing the AMO-02-MD-2-003 study (i.e. subjects who did not complete AMO-02-MD-2-003, subjects who completed AMO-02-MD-2-003 but did not directly rollover or subjects who are re-entering AMO-02-MD-2-004), will not be considered eligible for the study without meeting all of the criteria below:

• Subjects under study must be individuals with a diagnosis of Congenital or Childhood Onset DM1.

• Diagnosis must be genetically confirmed

• Subjects must be male or female aged ≥6 years to ≤45 years at Screening

• Subjects must have a Clinical Global Impression - Severity (CGI-S) score of 3 or greater at Screening (V-1)

• Written, voluntary informed consent must be obtained before any study related procedures are conducted. Where a parent or legally authorized representative (LAR) provides consent, there must also be assent from the subject (as required by local regulations)

• Subject's caregiver must be willing and able to support participation for duration of study

• Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol

⁃ Subjects entering directly from completing the antecedent AMO-02-MD-2-003 study will not be considered eligible for the study without meeting all of the criteria below:

• Subjects who have completed the antecedent AMO-02-MD-2-003 study through V11

• Written, voluntary informed consent must be obtained before any study related procedures are conducted. Where a parent or LAR provides consent, there must also be assent from the subject (as required by local regulations)

• Subject's caregiver must be willing and able to support participation for duration of study

• Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol

Locations
United States
Arkansas
Arkansas Children's Hospital
RECRUITING
Little Rock
California
University of California, Los Angeles (UCLA)
ENROLLING_BY_INVITATION
Los Angeles
Stanford University
ENROLLING_BY_INVITATION
Palo Alto
Iowa
University of Iowa Hospitals and Clinics
RECRUITING
Iowa City
Illinois
Lurie's Children's Hospital
RECRUITING
Chicago
New York
University of Rochester - Medical Center
RECRUITING
Rochester
Pennsylvania
University of Pittsburgh Medical Center
RECRUITING
Pittsburgh
Utah
University of Utah Clinical Neurosciences Center
RECRUITING
Salt Lake City
Virginia
Children's Hospital of The King's Daughters
WITHDRAWN
Norfolk
Virginia Commonwealth University-Department of Neurology - Muscular Dystrophy Translational Research Program
COMPLETED
Richmond
Other Locations
Australia
The Bright Alliance
RECRUITING
Randwick
Canada
Children's Hospital London Health Sciences Centre (LHSC)
ENROLLING_BY_INVITATION
London
Children's Hospital of Eastern Ontario
RECRUITING
Ottawa
New Zealand
New Zealand Clinical Research (NZCR)
COMPLETED
Auckland
Time Frame
Start Date: 2021-08-23
Estimated Completion Date: 2026-12-31
Participants
Target number of participants: 76
Treatments
Experimental: Tideglusib
Weight adjusted or weight banded tideglusib, orally, once daily
Sponsors
Leads: AMO Pharma Limited

This content was sourced from clinicaltrials.gov

Similar Clinical Trials