19 completed, 9 active not recruiting, 8 unknown, 5 terminated. 14 Phase 1, 9 Phase 2, 5 Not applicable, 4 Phase 3.
Follow Muscular Dystrophies, Limb-Girdle: new trials by email Literature Review of Muscular Dystrophies, Limb-Girdle trials Research Report on Muscular Dystrophies, Limb-Girdle
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Telecare Psychosocial and Cognitive Intervention for Children and Adolescents With Limb-Girdle Muscular Dystrophy Neuro-psychosocial teleassistance program - Limb-Girdle Muscular Dystrophy, Social Competence | Not applicable | Recruiting | University of Deusto | 30 | 2026-03-01 | 2026-07-17 |
| The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period. Assistive gait devices combined with physiotherapy - Inclusion Body Myositis, Myotonic Dystrophy 1 | Not applicable | Enrolling by invitation | LMU Klinikum | 30 | 2025-09-01 | 2025-07-18 |
| A Study of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Pediatric and Adult Participants SRP-9005, Corticosteroid - Muscular Dystrophies, Limb-Girdle | Phase 3 | Withdrawn | Sarepta Therapeutics, Inc. | 2025-06-30 | 2025-08-06 | |
| 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 States SRP-9004 - Limb Girdle Muscular Dystrophy, Limb Girdle Muscular Dystrophy Type 2D/R3 | Phase 1 | Terminated | Sarepta Therapeutics, Inc. | 4 | 2025-01-09 | 2025-09-04 |
| The Role of Muscle Ultrasound in Assessment of Sample of Patients With Limb-girdle Muscular Dystrophy Muscle ultrasound - Limb-girdle Muscular Dystrophy | Unknown | Assiut University | 50 | 2024-06-01 | 2024-05-06 | |
| Evolution of the Functional and Muscular State of Patients With Muscular Dystrophy 2A Belts calpaïnopathies - Limb Girdle Muscular Dystrophy | Active not recruiting | Assistance Publique - Hôpitaux de Paris | 25 | 2024-05-28 | 2026-01-15 | |
| LGMD R1 Natural History Study Calpain-3 Deficiency Limb Girdle Muscular Dystrophy Type 2A, Limb Girdle Muscular Dystrophy | Active not recruiting | Virginia Commonwealth University | 100 | 2024-01-31 | 2026-06-10 | |
| Longitudinal Geriatric Vulnerability, Chemotherapy Toxicity, and Functional Outcomes in Older Adults With Gastrointestinal Cancer Standard-of-Care Systemic Chemotherapy - Gastrointestinal Cancers, Frailty | Completed | Ankara Etlik City Hospital | 132 | 2024-01-15 | 2026-10-16 | |
| 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/R4 SRP-9003, Glucocorticoid - Limb-girdle Muscular Dystrophy | Phase 3 | Active not recruiting | Sarepta Therapeutics, Inc. | 17 | 2024-01-15 | 2026-03-02 |
| Rehabilitation in Muscular Dystrophies From the Hospital Facility to the Home: Pilot Project [RIMUDI] tele-rehabilitation system with virtual reality Home Kit - Virtual Reality Rehabilitation System (VRRS KHYMEIA) - Muscular Dystrophies, Limb Girdle Muscular Dystrophy | Not applicable | Unknown | IRCCS Eugenio Medea | 10 | 2023-12-15 | 2024-04-22 |
| Long-Term Development of Muscular Dystrophy Outcome Assessments LGMD1B, LGMD1C | Recruiting | Virginia Commonwealth University | 1000 | 2023-10-18 | 2025-11-18 | |
| 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) SRP-6004 - Limb Girdle Muscular Dystrophy | Phase 1 | Terminated | Sarepta Therapeutics, Inc. | 2 | 2023-05-22 | 2025-09-04 |
| A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1) AB-1003 dose level 1, AB-1003 dose level 2, Placebo - Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2 | Phase 1 / Phase 2 | Active not recruiting | AskBio Inc | 9 | 2023-05-15 | 2026-08-26 |
| 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) SRP-9003 - Limb Girdle Muscular Dystrophy | Phase 1 | Active not recruiting | Sarepta Therapeutics, Inc. | 6 | 2022-12-19 | 2026-02-19 |
| Limb-Girdle Video Assessment Limb-Girdle Muscular Dystrophy ; Subtypes 2A, 2B, and 2I | Terminated | The Emmes Company, LLC | 45 | 2022-06-20 | 2023-12-18 | |
| Schulze Muscular Dystrophy Ability Clinical Study Abilitech Assist - Muscular Dystrophies, Spinal Muscular Atrophy | Not applicable | Unknown | AbiliTech Medical Inc. | 35 | 2022-05-26 | 2022-12-05 |
| 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 Practice Limb-girdle Muscular Dystrophy | Active not recruiting | Sarepta Therapeutics, Inc. | 205 | 2021-04-22 | 2025-11-06 | |
| MRI-phenotyping of Patients With Pathogenic Anoctamin 5 Variants No intervention - Limb Girdle Muscular Dystrophy | Active not recruiting | Copenhagen University Hospital, Rigshospitalet | 200 | 2021-04-01 | 2026-08-12 | |
| Motor Parameters in Patients With Limb Girdle Muscular Dystrophy Limb Girdle Muscular Dystrophies | Unknown | Assistance Publique - Hôpitaux de Paris | 80 | 2021-04-01 | 2023-09-06 | |
| Adaptation of EDAQ Into Turkish, Examining The Reliability and Validity For People With RA Adaptation into Turkish, Examining The Reliability and Validity for People with RA - Arthritis, Rheumatoid, Activities of Daily Living | Unknown | Hacettepe University | 210 | 2021-03-30 | 2022-02-17 |
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Muscular Dystrophies (13) · Muscular Dystrophy, Duchenne (11) · Sarcoglycanopathies (7) · Muscular Dystrophy, Facioscapulohumeral (6) · Muscular Dystrophy, Limb-Girdle, Type 2I (6) · Limb-girdle muscular dystrophy type 2A (4) · Limb-girdle muscular dystrophy, type 2B (4) · Neuromuscular Diseases (4) · Limb-girdle muscular dystrophy, type 2E (3) · Walker-Warburg Syndrome (3) · Muscular Atrophy, Spinal (2) · Muscular Dystrophy, Limb-Girdle, Type 1D (2) · Muscular Dystrophy, Limb-Girdle, Type 2G (2) · Muscular Dystrophy, Limb-Girdle, Type 2M (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (49) · Genetic Diseases, Inborn (49) · Muscular Diseases (49) · Muscular Disorders, Atrophic (49) · Nervous System Diseases (49) · Musculoskeletal Diseases (48) · Neuromuscular Diseases (45) · Muscular Dystrophies (36) · Genetic Diseases, X-Linked (12) · Cardiomyopathies (7) · Cardiovascular Diseases (7) · Heart Diseases (7) · Respiration Disorders (7) · Respiratory Tract Diseases (7) · Pathological Conditions, Signs and Symptoms (5)
Prednisone (3) · Acceptance and Commitment Therapy (1) · Adrenal Cortex Hormones (1) · Glucocorticoids (1) · Lisinopril (1) · Physical Therapy Modalities (1) · Prednisolone (1) · Saline Solution (1) · Stamulumab (1) · coenzyme Q10 (1) · deflazacort (1)
Sarepta Therapeutics, Inc. (9) · Cooperative International Neuromuscular Research Group (4) · Copenhagen University Hospital, Rigshospitalet (4) · Nationwide Children's Hospital (3) · Virginia Commonwealth University (3) · Assistance Publique - Hôpitaux de Paris (2) · ML Bio Solutions, Inc. (2) · Neurogen Brain and Spine Institute (2) · aTyr Pharma, Inc. (2) · AbiliTech Medical Inc. (1) · Ankara Etlik City Hospital (1) · AskBio Inc (1) · Assiut University (1) · Boston Children's Hospital (1) · Cure CMD (1)
United States (31) · Denmark (8) · United Kingdom (5) · France (4) · Germany (4) · Italy (4) · Canada (3) · Spain (3) · Turkey (Türkiye) (3) · Belgium (2) · India (2) · Norway (2) · Sweden (2) · Brazil (1) · Japan (1)
Nicholas Johnson, MD (3) · Aleksandar Radunovic (1) · Alok K Sharma, M.S., M.Ch (1) · Amy Harper, MD (1) · Carolina Tesi-Rocha, M.D. (1) · Christopher Graham (1) · Cristobal Passalacqua, MD (1) · Galip Can Uyar, MD (1) · Gonca Bumin, Prof (1) · Gustavo Dziewczapolski, PhD (1) · Jerry R Mendell, M.D. (1) · Jerry R Mendell, MD (1) · Jerry R. Mendell, M.D. (1)
PMID 19798725, PMID 21031578, PMID 25238819, PMID 25863045, PMID 27708273, PMID 28533404, PMID 29175898, PMID 30287669, PMID 30852765, PMID 32715341, PMID 35192788, PMID 37886601, PMID 38177855, PMID 38491364, PMID 41938495
2015 (1), R01NS080929 (1), U01AR060911 (1), U54AR050733 (1), U54AR056953 (1)