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Myotonic Muscular Dystrophy clinical trials

View clinical trials related to Myotonic Muscular Dystrophy.

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NCT ID: NCT05027269 Completed - Clinical trials for Myotonic Dystrophy 1

Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients

MARINA
Start date: October 28, 2021
Phase: Phase 1/Phase 2
Study type: Interventional

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 2 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.

NCT ID: NCT00004769 Completed - Clinical trials for Myotonic Muscular Dystrophy

Study of Muscle Wasting and Altered Metabolism in Patients With Myotonic Dystrophy

Start date: December 1993
Phase: N/A
Study type: Observational

OBJECTIVES: I. Examine the interrelationships between muscle wasting (phenotype), the degree of myotonic dystrophy (DM) gene expression (genotype) in patients with DM. II. Characterize the insulin resistance in these patients. III. Assess the glucose uptake in the leg and forearm tissues of these patients. IV. Determine the stability of the DM gene lesion in muscles over a 5-10 year period.