RecruitingNCT07700225
Neuromuscular
Myotonic Dystrophy
Also known as Steinert disease, DM1, dystrophia myotonica
Myotonic Dystrophy type 1 (DM1) is the most common adult-onset muscular dystrophy, caused by a CTG trinucleotide repeat expansion in the DMPK gene on chromosome 19q13.
ORPHA:273 ↗Gene DMPKPrevalence 1 in 8,000Onset Variable; childhood to adulthood depending on subtypeAutosomal dominant trinucleotide repeat expansion
42
studies recruiting now
as of 7 Sept 2026
142
studies registered in total
as of 7 Sept 2026
12
countries with a recruiting site
as of 7 Sept 2026
13 Jul 2026
most recent study posted
among recruiting studies
Recruiting trials
RecruitingPhase 3NCT07486934
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
RecruitingPhase 3NCT07587242
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
RecruitingNot applicableNCT07580365
VirtualPark_Pediatric
RecruitingNCT06747884
Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 42 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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About Myotonic Dystrophy
Myotonic Dystrophy type 1 (DM1) is the most common adult-onset muscular dystrophy, caused by a CTG trinucleotide repeat expansion in the DMPK gene on chromosome 19q13.3. It is a multisystem disorder affecting skeletal muscle, cardiac muscle, the lens of the eye, and the endocrine and central nervous systems. Disease severity correlates with repeat length, and anticipation — worsening across generations — is a hallmark feature.
Common clinical features
Myotonia (delayed muscle relaxation after contraction)Progressive distal muscle weakness and wastingCardiac conduction defects and arrhythmiasPosterior subcapsular cataractsExcessive daytime sleepiness and cognitive involvementFrontal balding and testicular atrophy in malesSwallowing difficulties and gastrointestinal dysmotility
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
3 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Phase 3Mexiletine
Phase 2Mecasermin Rinfabate
Phase 2Tideglusib (Zentylor nypta)
Before you apply
Things trial teams commonly ask about for Myotonic Dystrophy. Not eligibility rules; those are set by each study.
- Trials often require genetic confirmation of CTG repeat length (typically >50 repeats for DM1); have your genetic report ready
- Cardiac eligibility screens are common — bring a recent ECG and echocardiogram as conduction defects may be exclusion criteria
- Functional outcome measures such as grip strength myometry and the MIRS scale are standard; baseline assessments strengthen eligibility documentation
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).