3 completed, 2 unknown, 1 active not recruiting, 1 recruiting. 2 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 2 | Completed 2 |
| Phase 2 | 1 | Completed 1 |
Follow Sarcoglycanopathies: new trials by email Literature Review of Sarcoglycanopathies trials Research Report on Sarcoglycanopathies
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 |
|---|---|---|---|---|---|---|
| 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 | |
| Long-Term Development of Muscular Dystrophy Outcome Assessments LGMD1B, LGMD1C | Recruiting | Virginia Commonwealth University | 1000 | 2023-10-18 | 2025-11-18 | |
| 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 | |
| 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 | |
| Limb Girdle Muscular Dystrophy Type 2E Recruitment Study Limb-Girdle Muscular Dystrophy, Type 2E | Unknown | Nationwide Children's Hospital | 25 | 2018-03-28 | 2023-03-06 | |
| A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 (Patidistrogene Bexoparvovec) in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D) SRP-9004 - Limb-Girdle Muscular Dystrophy, Type 2D | Phase 1 / Phase 2 | Completed | Sarepta Therapeutics, Inc. | 6 | 2015-02-01 | 2023-06-15 |
| Gene Transfer Therapy for Treating Children and Adults With Limb Girdle Muscular Dystrophy Type 2D (LGMD2D) rAAV1.tMCK.human-alpha-sarcoglycan- First cohort, Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- Second cohort - Muscular Dystrophies | Phase 1 | Completed | Nationwide Children's Hospital | 6 | 2008-03-01 | 2013-02-05 |
Muscular Dystrophies, Limb-Girdle (4) · Muscular Dystrophies (3) · Limb-girdle muscular dystrophy, type 2E (2) · Arthritis, Rheumatoid (1) · Biliary Tract Neoplasms (1) · Colorectal Neoplasms (1) · Esophageal Neoplasms (1) · Frailty (1) · Gastrointestinal Neoplasms (1) · Limb-Girdle Muscular Dystrophy, Type 1G (1) · Limb-girdle muscular dystrophy type 2A (1) · Limb-girdle muscular dystrophy type 2F (1) · Limb-girdle muscular dystrophy, type 1B (1) · Limb-girdle muscular dystrophy, type 2B (1)
Cardiomyopathies (7) · Cardiovascular Diseases (7) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (7) · Genetic Diseases, Inborn (7) · Heart Diseases (7) · Muscular Diseases (7) · Muscular Disorders, Atrophic (7) · Musculoskeletal Diseases (7) · Nervous System Diseases (7) · Neuromuscular Diseases (7) · Respiration Disorders (7) · Respiratory Tract Diseases (7) · Muscular Dystrophies (4) · Muscular Dystrophies, Limb-Girdle (3) · Arthritis (1)
Nationwide Children's Hospital (2) · Sarepta Therapeutics, Inc. (2) · Ankara Etlik City Hospital (1) · Hacettepe University (1) · Virginia Commonwealth University (1)
United States (4) · Turkey (Türkiye) (3) · Belgium (1) · Brazil (1) · Canada (1) · Germany (1) · Italy (1) · Spain (1) · Sweden (1) · United Kingdom (1)
Galip Can Uyar, MD (1) · Gonca Bumin, Prof (1) · Jerry R Mendell, M.D. (1) · Jerry R. Mendell, MD (1) · Nicholas Johnson, MD (1)
PMID 19798725, PMID 21031578, PMID 25238819, PMID 25863045, PMID 30852765, PMID 32715341
U01AR060911 (1), U54AR050733 (1)