Muscular Dystrophies, Limb-Girdle clinical trials
The registry lists 47 studies for Muscular Dystrophies, Limb-Girdle across more than 10 countries. 10 are recruiting or about to start. 34 of them are mainly about Muscular Dystrophies, Limb-Girdle (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-10.
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Who runs them
Sarepta Therapeutics, Inc. (9) · Rigshospitalet, Denmark (4) · Cooperative International Neuromuscular Research Group (4) · Virginia Commonwealth University (3) · Neurogen Brain and Spine Institute (2) · aTyr Pharma, Inc. (2) · Assistance Publique - Hôpitaux de Paris (2) · ML Bio Solutions, Inc. (2)
Who develops what
Save this setEvery programme with a company-led study here in the last two years, whatever its size: 2 programmes from 1 company, 2 with this as their studies’ main aim; 0 with a live phase 3 study; 0 primary completions in the next 12 months.
Comparator & background therapies
What other companies’ phase 2+ studies give as the comparator or the background here (its leading trial sponsor and partners left out): a sign of what new drugs are measured against, not by itself the standard of care, its prescriptions or its market.
| Drug | Leading trial sponsor · rights | Companies, last 5 years |
|---|---|---|
GlucocorticoidRecent, limitedEvidence
| InflaRx GmbH33% of its industry studies | 11 study |
Adrenal Cortex HormonesRecent, limitedEvidence
| — | 11 study |
Programmes
“Meets, by arms” reads each study’s arms: vs its arm against the therapy’s; + X vs a combination against it; added to it plus the therapy against the therapy; studied with given together only. The next primary completion is the next phase-3 study’s when there is one.
The 2 programmes — sign in to see the whole list, free: filter by phase and aim, sort by late development, direct comparisons or the next primary completion.
Primary completion in the next 12 months (0)
Live phase 2 and 3 studies of these programmes. Primary completion is when the main measure’s data collection ends, as the registry states it (most are estimates); results come later, if they are announced.
None in the next 12 months.
Read from ClinicalTrials.gov, industry-led studies. Main aim: this is the study’s judged main condition; named, not the main aim: the sponsor names it, the study is about something else; registry mapping only: only the registry’s mapping put it here. Leading trial sponsor is the company leading the most industry studies of a drug, a measure of trial activity, not of rights. US and EU rights, and Approved: from the regulators’ records (FDA’s Drugs@FDA via openFDA; EMA’s medicines data) — the holder of a marketed or authorised original, generics and other companies’ versions; not rights elsewhere, nor approval for this condition. Phase 4 trial recorded, where neither lists it: a phase 4 study exists somewhere. Not read yet: labels by indication, exclusivity, licences, line of therapy, academic comparators, sales.
What is being tested here
Drugs under test in Muscular Dystrophies, Limb-Girdle studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| ATYR-1940 | 0 | 0 | 2 |
| Ribitol | 0 | 0 | 1 |
| Prednisone | 0 | 0 | 1 |
| Coenzyme Q10 | 0 | 1 | 1 |
| Lisinopril | 0 | 1 | 1 |
| Deflazacort | 0 | 1 | 1 |
| Stem Cell | 0 | 0 | 1 |
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 6
- Nov 2026Study of BBP-418 in Patients With LGMD2I · ML Bio Solutions, Inc.
- Dec 2026Telecare Psychosocial and Cognitive Intervention for Children and Adolescents With Limb-Girdle Muscular Dystrophy · University of Deusto
- Dec 2026Molecular Analysis of Patients With Neuromuscular Disease · Boston Children's Hospital
- May 2027The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period. · LMU Klinikum
- May 2027Evolution of the Functional and Muscular State of Patients With Muscular Dystrophy 2A Belts · Assistance Publique - Hôpitaux de Paris
- Nov 2027BBM-L101 Gene Therapy in Participants With LGMD2D/R3 · Peking University First Hospital
Who these studies take
Of the 7 Muscular Dystrophies, Limb-Girdle studies recruiting now, 3 (43%) set no upper age limit and 3 (43%) stop at 75 or younger. 5 (71%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 1 (14%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 5 (71%) | 5 (71%) |
| 18 | 6 (86%) | 6 (86%) |
| 30 | 6 (86%) | 6 (86%) |
| 40 | 6 (86%) | 6 (86%) |
| 50 | 6 (86%) | 6 (86%) |
| 65 | 5 (71%) | 5 (71%) |
| 75 | 4 (57%) | 4 (57%) |
| 85 | 4 (57%) | 4 (57%) |
Across all 47 studies, recruiting or not: 45% stop at 75 or younger and 40% at 65; 64% take children; 0% are for women only and 0% for men only; 4% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 47 of 47. search them.
- BBM-L101 Gene Therapy in Participants With LGMD2D/R3Not yet recruitingNCT07874516Phase 11 country
- Telecare Psychosocial and Cognitive Intervention for Children and Adolescents With Limb-Girdle Muscular DystrophyRecruitingNCT07711730Not applicable1 country
- Long-Term Development of Muscular Dystrophy Outcome AssessmentsRecruitingNCT059896201 country
- The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.Enrolling by invitationNCT07072676Not applicable1 country
- The Role of Muscle Ultrasound in Assessment of Sample of Patients With Limb-girdle Muscular DystrophyNot yet recruitingNCT06399770
- Rehabilitation in Muscular Dystrophies From the Hospital Facility to the Home: Pilot Project [RIMUDI]RecruitingNCT06378203Not applicable1 country
- Global FKRP RegistryRecruitingNCT040015951 country
- Motor Parameters in Patients With Limb Girdle Muscular DystrophyRecruitingNCT047720271 country
- Molecular Analysis of Patients With Neuromuscular DiseaseRecruitingNCT003901041 country
- Congenital Muscle Disease Study of Patient and Family Reported Medical InformationRecruitingNCT014034021 country
- A Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related)TerminatedNCT05906251Phase 11 country
- Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Participants in the United StatesTerminatedNCT06747273Phase 11 country
- A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)Active, not recruitingNCT05230459Phase 1/21 country
- 3 Year Follow up on ANO5 PatientsActive, not recruitingNCT052066171 country
- MRI-phenotyping of Patients With Pathogenic Anoctamin 5 VariantsActive, not recruitingNCT051027991 country
- Study of BBP-418 in Patients With LGMD2IActive, not recruitingNCT04800874Phase 21 country
- LGMD R1 Natural History StudyActive, not recruitingNCT056180801 country
- A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4Active, not recruitingNCT06246513Phase 36 countries
- Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD)CompletedNCT039812892 countries
- A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)Active, not recruitingNCT05876780Phase 11 country
- Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)TerminatedNCT03652259Phase 1/21 country
- Evolution of the Functional and Muscular State of Patients With Muscular Dystrophy 2A BeltsActive, not recruitingNCT063905661 country
- A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical PracticeActive, not recruitingNCT0447592610 countries
- A Study of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Pediatric and Adult ParticipantsWithdrawnNCT06952686Phase 31 country
- Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD)CompletedNCT02836418Phase 1/23 countries
- Limb-Girdle Video AssessmentTerminatedNCT055287571 country
- Evaluate Safety and Biological Activity of ATYR1940 in Participants With Limb Girdle Muscular Dystrophy 2B (LGMD2B) and Facioscapulohumeral Muscular Dystrophy (FSHD)CompletedNCT02579239Phase 1/23 countries
- Weekly Steroids in Muscular DystrophyCompletedNCT04054375Phase 21 country
- A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 (Patidistrogene Bexoparvovec) in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D)CompletedNCT01976091Phase 1/2
- Biomarker Development in LGMD2iCompletedNCT042026272 countries
- Limb Girdle Muscular Dystrophy Type 2E Recruitment StudyStatus unknownNCT034923461 country
- Schulze Muscular Dystrophy Ability Clinical StudyStatus unknownNCT05409079Not applicable1 country
- Limb-Girdle Muscular Dystrophy Type 2I in NorwayStatus unknownNCT039306281 country
- A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)TerminatedNCT03783923Phase 38 countries
- rAAVrh74.MHCK7.DYSF.DV for Treatment of DysferlinopathiesCompletedNCT02710500Phase 11 country
- Acceptance and Commitment Therapy for Muscle DiseaseCompletedNCT02810028Not applicable1 country
- Cell Therapy in Limb Girdle Muscular DystrophyWithdrawnNCT02245711Phase 11 country
- Stem Cell Therapy in Limb Girdle Muscular DystrophyWithdrawnNCT02050776Phase 11 country
- Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular DystrophiesCompletedNCT01126697Phase 2/33 countries
- Genetic Characterization of Individuals With Limb Girdle Muscular DystrophyCompletedNCT004579121 country
- MRI on Persons With Mutations in POMT2 Gene (LGMD2N)CompletedNCT027593021 country
- MRI and Muscle Involvement in Patients With Mutations in GMPPBCompletedNCT026353211 country
- Safety Study of Transvenous Limb Perfusion in Human Muscular DystrophyCompletedNCT00873782Phase 11 country
- Evaluation of Limb-Girdle Muscular DystrophyCompletedNCT008933341 country
- Cardiac Outcome Measures in Children With Muscular DystrophyCompletedNCT010664551 country
- Cardiac Magnetic Resonance in Children With Muscular DystrophyCompletedNCT010810801 country
- Study Evaluating MYO-029 in Adult Muscular DystrophyCompletedNCT00104078Phase 1/21 country