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NCT05004129 · ClinicalTrials.gov registry record · Phase 2
Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
A Phase 2 study of Congenital Myotonic Dystrophy, sponsored by AMO Pharma Limited.
- Recruiting
- Registry status
- Phase 2
- Development phase
- 76
- Enrollment target
- 14
- Study locations
NCT05004129: Recruiting Phase 2 study of Congenital Myotonic Dystrophy, sponsored by AMO Pharma Limited.
NCT05004129 is a Phase 2 study of Congenital Myotonic Dystrophy that is actively recruiting participants, run by AMO Pharma Limited. The registered enrollment target is 76 participants, below the 1,688-participant average among 4 other Congenital Myotonic Dystrophy trials with a reported enrollment target (95% lower). The trial reports 14 study locations across 11 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05004129, a Phase 2 study of Congenital Myotonic Dystrophy, is actively recruiting participants, sponsored by AMO Pharma Limited.
- RECRUITING
- Registry status
- Phase 2
- Development phase
- 76 participants
- Enrollment target
- 14
- Study locations
Study 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.
Primary Outcome
The incidence of AEs, including SAEs, and abnormal findings in objective assessments (e.g. laboratory values, ECGs and vital signs) from Screening to Enrolment (where applicable), from Enrolment to End of Treatment (52 Weeks), and End of Treatment to the End of Follow-up period.
Conditions Studied
Interventions
- DRUG Tideglusib
Study Locations (14)
California
- University of California, Los Angeles (UCLA) - Los Angeles
- Stanford University - Palo Alto
Virginia
- Children's Hospital of The King's Daughters - Norfolk
- Virginia Commonwealth University-Department of Neurology - Muscular Dystrophy Translational Research Program - Richmond
Ontario
- Children's Hospital London Health Sciences Centre (LHSC) - London
- Children's Hospital of Eastern Ontario - Ottawa
Arkansas
- Arkansas Children's Hospital - Little Rock
Illinois
- Lurie's Children's Hospital - Chicago
Iowa
- University of Iowa Hospitals and Clinics - Iowa City
New York
- University of Rochester - Medical Center - Rochester
Pennsylvania
- University of Pittsburgh Medical Center - Pittsburgh
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 76 participants |
| Start Date | 2021-08-23 |
| Est. Completion | 2026-12-31 |
| Phase | Phase 2 |
What NCT05004129 shows while recruiting
NCT05004129 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 76 participants, a relatively small participant target, below the 1,688-participant average among 4 other Congenital Myotonic Dystrophy trials with a reported enrollment target (95% lower).
The record links to 1 condition, with Congenital Myotonic Dystrophy appearing as the primary indexed condition, and to 1 intervention - of which Tideglusib is the first listed.
NCT05004129 lists 14 locations in 11 states (California, Virginia, Ontario).
Frequently Asked Questions
What is clinical trial NCT05004129 about?
NCT05004129 is a clinical study titled "Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy". 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.
What is the current status of trial NCT05004129?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 76 participants. The study started on 2021-08-23. Estimated completion is 2026-12-31.
What conditions does trial NCT05004129 study?
This clinical trial studies the following conditions: Congenital Myotonic Dystrophy.
What interventions are being tested in trial NCT05004129?
The interventions under investigation include: Tideglusib (DRUG).
Who is sponsoring clinical trial NCT05004129?
This trial is sponsored by AMO Pharma Limited, which has 2 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05004129 being conducted?
This trial has 14 study locations across Arkansas, California, Illinois, Iowa, New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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