2 completed, 1 terminated. 3 Phase 1, 3 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 3 | Completed 2, Terminated 1 |
| Phase 2 | 3 | Completed 2, Terminated 1 |
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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 |
|---|---|---|---|---|---|---|
| A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis LYS-GM101 - GM1 Gangliosidosis | Phase 1 / Phase 2 | Terminated | LYSOGENE | 5 | 2021-05-11 | 2023-06-09 |
| Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy SAF-301 - Mucopolysaccharidosis Type III A, Sanfilippo Disease Type A | Phase 1 / Phase 2 | Completed | LYSOGENE | 4 | 2013-05-01 | 2017-06-20 |
| Intracerebral Gene Therapy for Sanfilippo Type A Syndrome SAF-301 - Mucopolysaccharidosis Type III A, Sanfilippo Disease Type A | Phase 1 / Phase 2 | Completed | LYSOGENE | 4 | 2011-08-01 | 2014-05-06 |
Genetic Diseases, Inborn (2) · Metabolic Diseases (2) · Mucopolysaccharidosis III (2) · Neurodegenerative Diseases (2) · Gangliosidosis, GM1 (1) · Lysosomal Storage Diseases (1) · Nervous System Diseases (1)
France (3) · United Kingdom (1) · United States (1)