Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
Status:
Recruiting
Trial end date:
2023-09-01
Target enrollment:
Participant gender:
Summary
AOC 1001-CS1 is a randomized, double-blind, placebo-controlled, Phase 1/2 study to evaluate
the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple-doses
of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients
(MARINA).
Part A is a single dose design with 1 cohort (dose level). In Part A, the patient duration is
6 months as the treatment period is 1 day followed by a 6 month follow-up period.
Part B is a multiple-ascending dose design with 3 cohorts (dose levels). In Part B, the
patient duration is 6 months as the treatment period is 3 months followed by a 3 month
follow-up period.