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ClinicalTrials.gov 7 recruiting now early-phase tilt small index

Myotonic Dystrophy: early-phase trial registry

7 of 10 indexed Myotonic Dystrophy trials are currently recruiting, with more weight in Phase 1-2.

Corpus placement: #1,724 of 28,707 conditions by registered trial count.

10 US clinical trials · 7 currently recruiting · #1,724 of 28,707 conditions by registered trial count

Myotonic Dystrophy: 10 US clinical trials tracked, 7 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.

Enrollment posture brief

Registry condition myotonic-dystrophy

OPEN 7 | RECR 70% | LATE 1 | EARLY 3 | RANK #1724 | SPON 2

Nearest open-share peer: Advanced Solid Tumor (70% recruiting · 190 trials)

Acute Leukemia of Ambiguous 80%Myotonic Dystrophy (this)70%Achondroplasia40%Accelerated Phase Chronic My20%Accidental Fall20%
Open-enrollment share among volume-matched conditions near Myotonic Dystrophy

Myotonic Dystrophy lists 7 open slots (70% of 10 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →

Key findings

What ClinicalTrials.gov does not surface for Myotonic Dystrophy on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.

  1. Myotonic Dystrophy is recruiting above its size-band peers.

    7 of 10 indexed trials (70%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.

  2. One sponsor concentrates Myotonic Dystrophy research.

    Avidity Biosciences accounts for 2 of 10 trials (20%). 7 distinct sponsors appear on at least one study for this condition label.

  3. Typical Myotonic Dystrophy enrolment sits around 200 participants.

    Median target enrolment is 200 among the 10 trials that report a genuine target (sentinel values excluded). 4 of those (40%) plan for fewer than 100 participants.

  4. Myotonic Dystrophy research leans observational.

    4 interventional and 6 observational studies make up this index (40% interventional). Study type is sponsor-reported on ClinicalTrials.gov.

Phase 12Phase 21Phase 31

Open and 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

Named interventions

Most-linked intervention names on Myotonic Dystrophy records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.

Placebo Drug
2 trials
SomatoKine/IPLEX Drug
1 trials
AOC 1001 Drug
1 trials
AOC 1001 (del-desiran) Drug
1 trials
Patient-entered data Other
1 trials
SAR446268 Biological
1 trials

US sites by state

Facility locations filed on Myotonic Dystrophy trials. A trial with sites in several states counts in each.

New York 5 trials
Virginia 3 trials
North Carolina 3 trials
Massachusetts 3 trials
Florida 3 trials
California 3 trials
Texas 2 trials
Pennsylvania 2 trials

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

What to do with this Myotonic Dystrophy page

10 registered trials, 7 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.

Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Myotonic Dystrophy condition card totals 10 registered trials; most-linked intervention Placebo; top US site state New York. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.