| NCT Number | Study Title (abbreviated) | LGMD Subtype | Intervention/Drug | Phase | Status | Sponsor |
|---|---|---|---|---|---|---|
| NCT00494195 | Gene Transfer Therapy for LGMD2D | LGMD2D / LGMDR3 | AAV gene transfer (alpha-sarcoglycan) | Phase 1 | Completed | Nationwide Children's Hospital (pqac-00000018) |
| NCT01344798 | AAV1-gamma-sarcoglycan Gene Therapy for LGMD2C | LGMD2C / LGMDR5 | AAV1-gamma-sarcoglycan | Phase 1 | Completed | Genethon (pqac-00000020) |
| NCT01976091 | Safety Study of SRP-9004 for LGMD2D | LGMD2D / LGMDR3 | SRP-9004 (patidistrogene bexoparvovec) | Phase 1/2 | Completed | Sarepta Therapeutics, Inc. (pqac-00000018) |
| NCT05876780 | Single-Dose SRP-9003 Study for LGMD2E/R4 | LGMD2E / LGMDR4 | SRP-9003 (bidridistrogene xeboparvovec; SGCB gene transfer) | Phase 1 | Active, not recruiting | Sarepta Therapeutics, Inc. (pqac-00000018, pqac-00000019) |
| NCT06246513 | Bidridistrogene Xeboparvovec Trial for LGMD2E/R4 | LGMD2E / LGMDR4 | SRP-9003 / bidridistrogene xeboparvovec | Phase 3 | Active, not recruiting | Sarepta Therapeutics, Inc. (pqac-00000019) |
| NCT05906251 | SRP-6004 Gene Transfer Study for LGMD2B/R2 | LGMD2B / LGMDR2 | SRP-6004 (DYSF gene transfer) | Phase 1 | Terminated | Sarepta Therapeutics, Inc. (pqac-00000018) |
| NCT05588401 | GenPHSats-bASKet Gene-edited Muscle Stem Cells | LGMD (basket study) | Autologous gene-edited muscle stem cells | Phase 1/2 | Unknown | Charite University, Berlin, Germany (pqac-00000018) |
| NCT05973630 | ATA-200 for LGMD2C/R5 | LGMD2C / LGMDR5 | ATA-200 (AAV8 gamma-sarcoglycan gene therapy) | Phase 1b | Recruiting/ongoing | Asklepios BioPharmaceutical, Inc. / Atamyo Therapeutics (pqac-00000021) |


*Table: This table summarizes key current and recent gene therapy trials for autosomal recessive LGMD subtypes, including sarcoglycanopathies and dysferlinopathy. It is useful for quickly comparing study phase, status, intervention, and sponsor across the most relevant programs.*