A Phase 3 Randomized, Double-Blind, Placebo- Controlled, Global Study To Evaluate The Efficacy And Safety Of Intravenous Aoc 1001 For The Treatment Of Myotonic Dystrophy Type 1
Posted Date: Nov 7, 2024
- Investigator: Hani Kushlaf
- Specialties: Neurology, Neuromuscular Disorders
- Type of Study: Drug
Participants will be randomized (1:1 ratio) to receive an intravenous infusion of either AOC 1001 or saline (placebo) at the clinical study site every 8 weeks for a total of 7 doses. The final dose will occur at Week 48, followed by a final assessment at Week 54. To evaluate the efficacy of AOC 1001 on hand opening time. To evaluate the efficacy of AOC 1001 on measures of mobility, muscle strength, muscle function, and patient-reported outcomes
Criteria:
Participants Must Be 16 To 65 Years Of Age (Inclusive) At The Time Of Informed Consent And Have A Clinical And Genetic Diagnosis Of Dm1 And The Ability To Walk Independently For At Least 10 Meters.
Keywords:
Myotonic Dystrophy Type 1, Dm1
For More Information:
Ashley Smith
513-558-4874
ashley.fisher@uc.edu