Myotonic dystrophy 1
Clinical trials for Myotonic dystrophy 1 explained in plain language.
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Experimental drug AOC 1001 tested for Long-Term safety in rare muscle disease
Disease control CompletedThis study is a follow-up to an earlier trial, testing the long-term safety and effects of a drug called AOC 1001 in adults with myotonic dystrophy type 1 (DM1), a genetic muscle disease. 37 participants who completed the first study received multiple doses of AOC 1001 by IV infu…
Matched conditions: MYOTONIC DYSTROPHY 1
Phase 2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Sep 13, 2026 00:00 UTC
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Hope for muscle strength: new drug targets myotonic dystrophy
Disease control CompletedThis phase 3 trial tests an experimental drug called AOC 1001 in 159 adults with myotonic dystrophy type 1, a genetic condition that causes muscle weakness and stiffness. Participants receive either the drug or a placebo by intravenous infusion. The main goal is to see if the dru…
Matched conditions: MYOTONIC DYSTROPHY 1
Phase 3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Sep 03, 2026 00:00 UTC
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New drug AOC 1001 tested for rare muscle disease
Disease control CompletedThis study tested a new drug called AOC 1001 in 39 adults with myotonic dystrophy type 1, a genetic muscle disorder. Participants received either the drug or a placebo by IV infusion. The goal was to check safety and how the drug moves through the body. Results will help decide i…
Matched conditions: MYOTONIC DYSTROPHY 1
Phase 1/2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Jun 27, 2026 11:00 UTC
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New drug PGN-EDODM1 tested for muscle disease
Disease control CompletedThis early-stage trial tested a single dose of the drug PGN-EDODM1 in 24 adults with myotonic dystrophy type 1, a genetic muscle disorder. The main goal was to check safety and how the body handles the drug. The study is complete, but results are not yet available.
Matched conditions: MYOTONIC DYSTROPHY 1
Phase 1 • Sponsor: PepGen Inc • Aim: Disease control
Last updated Jun 27, 2026 09:04 UTC
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Experimental drug tideglusib tested for rare muscle disease
Disease control CompletedThis phase 2 study tested the safety and effectiveness of tideglusib, an experimental drug, in 16 adolescents and adults with congenital or juvenile-onset myotonic dystrophy type 1. Participants received either 400 mg or 1000 mg of tideglusib daily. The study measured side effect…
Matched conditions: MYOTONIC DYSTROPHY 1
Phase 2 • Sponsor: AMO Pharma Limited • Aim: Disease control
Last updated Jun 26, 2026 16:48 UTC
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Can a wakefulness drug combat the crushing fatigue of myotonic dystrophy?
Symptom relief CompletedThis phase 2 trial tests whether pitolisant, a wake-promoting drug, can reduce excessive daytime sleepiness in adults aged 18 to 65 with myotonic dystrophy type 1. Participants receive either pitolisant or a placebo for a period, and researchers measure changes in sleepiness, fat…
Matched conditions: MYOTONIC DYSTROPHY 1
Phase 2 • Sponsor: Harmony Biosciences Management, Inc. • Aim: Symptom relief
Last updated Sep 06, 2026 00:00 UTC
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Hidden danger: muscle disease linked to deadly clots
Knowledge-focused CompletedThis study investigates why people with myotonic dystrophy type 1 are 10 times more likely to develop dangerous blood clots in the legs or lungs. Researchers will compare blood samples from 130 participants—including patients with the disease, those with a history of clots, and h…
Matched conditions: MYOTONIC DYSTROPHY 1
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Jun 26, 2026 15:53 UTC