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Myotonic Dystrophy Type 1 (DM1) clinical trials at University of California Health

1 in progress, 0 open to eligible people

Showing trials for
  • Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients

    Sorry, not currently recruiting here

    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.

    at UCLA

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