RecruitingNot applicableNCT07711730
Neuromuscular
Limb-Girdle Muscular Dystrophy
Also known as LGMD, calpainopathy, dysferlinopathy
Limb-Girdle Muscular Dystrophies are a clinically and genetically heterogeneous group of disorders characterised by progressive weakness predominantly affecting the shoulder and pelvic girdle muscles. Over 30 genetic subtypes have been defi
ORPHA:370494 ↗Gene Multiple (CAPN3Gene DYSFGene etc)Prevalence 1 in 14,500 to 1 in 123,000 (subtype-dependent)Onset Variable; childhood to mid-adulthood depending on subtypeAutosomal recessive or dominant (subtype-dependent)
6
studies recruiting now
as of 7 Sept 2026
82
studies registered in total
as of 7 Sept 2026
2
countries with a recruiting site
as of 7 Sept 2026
17 Jul 2026
most recent study posted
among recruiting studies
Recruiting trials
RecruitingNCT05989620
Long-Term Development of Muscular Dystrophy Outcome Assessments
RecruitingNCT00313677
Clinical Trial Readiness for the Dystroglycanopathies
RecruitingNCT05902351
Natural History Study for Charcot Marie Tooth Disease
RecruitingNCT00390104
Molecular Analysis of Patients With Neuromuscular Disease
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 6 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.
Keep watching
Get an email when a new Limb-Girdle Muscular Dystrophy study opens.
One email a day at most. Unsubscribe with one click.
Used only for these alerts. Privacy.
Support
Patient organisations
Registry: Dysferlin Registry / LGMD-EURO · Join ↗. Registries connect patients to researchers and often hear about trials first.
About Limb-Girdle Muscular Dystrophy
Limb-Girdle Muscular Dystrophies are a clinically and genetically heterogeneous group of disorders characterised by progressive weakness predominantly affecting the shoulder and pelvic girdle muscles. Over 30 genetic subtypes have been defined, with LGMD R1 (calpain-3 deficiency) and LGMD R2 (dysferlin deficiency) being the most prevalent. Cardiac and respiratory involvement varies significantly by subtype.
Common clinical features
Progressive proximal shoulder and hip girdle weaknessDifficulty rising from the floor or climbing stairsMarkedly elevated serum creatine kinaseScapular wingingCardiomyopathy or arrhythmia (subtype-dependent)Respiratory muscle weakness in advanced diseaseCalf hypertrophy in some subtypes
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
4 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Phase 3Bidridistrogene Xeboparvovec
Phase 3Ribitol
Phase 3Deflazacort (Calcort)
Phase 1/2Stamulumab
Before you apply
Things trial teams commonly ask about for Limb-Girdle Muscular Dystrophy. Not eligibility rules; those are set by each study.
- Trials are highly subtype-specific; a next-generation sequencing (NGS) muscle gene panel or whole exome sequencing confirming your exact LGMD subtype is essential before searching
- Protein immunohistochemistry from a muscle biopsy (e.g. calpain-3, dysferlin staining) complements genetic data and is often required for enrolment
- North Star Ambulatory Assessment (NSAA) or Motor Function Measure (MFM) scores are standard baseline metrics — obtain these from a neuromuscular physiotherapist
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).