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DM1 clinical trials

View clinical trials related to DM1.

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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: NCT02084407 Completed - DM1 Clinical Trials

Induction of Pluripotent Stem Cells From Human Fibroblasts of DM1 Patients

Start date: November 2013
Phase: N/A
Study type: Interventional

The objective of the study is the modelisation of human cardiomyocyts from pluripotent stem cells in order to study the cardiac alterations induced by CTG expansions.