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ClinicalTrials.gov 7 recruiting now official registry

Myotonic Dystrophy clinical trials

7 of 10 indexed Myotonic Dystrophy trials are currently recruiting.

10 US clinical trials · 7 currently recruiting

Myotonic Dystrophy is the subject of 10 registered US clinical trials on ClinicalTrials.gov, 7 of them currently open to new participants. 1 are in Phase 3-4 (later-stage) and 3 in Phase 1-2 (earlier-stage). The most active sponsor is Avidity Biosciences, running 2 of these trials.

The research picture

Myotonic Dystrophy has 10 registered US clinical trials, 7 of them open to new participants right now, about 70% of the total.

7
recruiting participants now
70%
of trials open to enrollment
1
in Phase 3–4 (later-stage)
2
top sponsor: Avidity Biosciences
Phase 12Phase 21Phase 31

Active & Recent Trials

10 total, page 1 of 1

RECRUITING 3,500 participants

Myotonic Dystrophy Family Registry

Myotonic Dystrophy Foundation

NCT02398786

RECRUITING 3,000 participants

Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry

University of Rochester

NCT00082108

RECRUITING 465 participants

Biomarker Development for Muscular Dystrophies

Massachusetts General Hospital

NCT05019625

RECRUITING 215 participants

Extracellular RNA Biomarkers of Myotonic Dystrophy

Massachusetts General Hospital

NCT05020002

RECRUITING 200 participants

Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension

Virginia Commonwealth University

NCT06747884

RECRUITING 75 participants

Development of Quantitative Muscle Imaging as a Biomarker of Disease Endpoints in Myotonic Dystrophy

Wake Forest University Health Sciences

NCT07362875

RECRUITING Phase 1 32 participants

A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 50 Years of Age With Non-congenital Myotonic Dystrophy Type 1

Sanofi

NCT06844214

ACTIVE NOT RECRUITING Phase 3 159 participants

Global Study of Del-desiran for the Treatment of DM1

Avidity Biosciences

NCT06411288

COMPLETED Phase 2 37 participants

Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients

Avidity Biosciences

NCT05479981

COMPLETED Phase 1 17 participants

Effects of SomatoKine (Iplex)Recombinant Human Insulin-like Growth Factor-1/Recombinant Human Insulin-like Growth Factor-binding Protein-3 (rhIGF-I/rhIGFBP-3) in Myotonic Dystrophy Type 1 (DM1)

University of Rochester

NCT00233519

Phase Distribution

PhaseTrial count
Phase 1 2
Phase 2 1
Phase 3 1

Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.

Reading the Myotonic Dystrophy Trial Landscape

10 indexed Myotonic Dystrophy studies · 7 recruiting (70%).

Phase mix: 1 late (III/IV) vs 3 early (I/II).

Top sponsor: Avidity Biosciences (2 trials), 6 others listed. 10 shown below, recruiting first.

How to read this condition's trial landscape

That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.

Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.

All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation, eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.

Frequently Asked Questions

How many clinical trials are there for Myotonic Dystrophy?

PlainTrial tracks 10 US clinical trials for Myotonic Dystrophy, of which 7 are currently recruiting participants. Data sourced from ClinicalTrials.gov.

How do I find a recruiting trial for Myotonic Dystrophy?

Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.

Is this data current?

Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.

Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.

Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2026 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.

Verify with NIH →

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial for this condition, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.