1 active not recruiting, 1 recruiting, 1 suspended. 3 Phase 1, 3 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 3 | Active not recruiting 1, Recruiting 1, Suspended 1 |
| Phase 2 | 3 | Active not recruiting 1, Recruiting 1, Suspended 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 |
|---|---|---|---|---|---|---|
| Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1 GB221 - Spinal Muscular Atrophy Type I | Phase 1 / Phase 2 | Recruiting | Gemma Biotherapeutics | 22 | 2026-01-06 | 2026-06-29 |
| Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease PBKR03 - Leukodystrophy, Globoid Cell | Phase 1 / Phase 2 | Suspended | Gemma Biotherapeutics | 24 | 2022-02-24 | 2026-06-30 |
| Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis PBGM01 - GM1 Gangliosidosis, GM1 Gangliosidosis, Type I | Phase 1 / Phase 2 | Active not recruiting | Gemma Biotherapeutics | 26 | 2021-03-17 | 2026-06-30 |
Lysosomal Storage Diseases (2) · Rare Diseases (2) · Gangliosidosis, GM1 (1) · Leukodystrophy, Globoid Cell (1) · Spinal Muscular Atrophies of Childhood (1)
Brazil (3) · United Kingdom (2) · United States (2) · Canada (1) · Israel (1) · Netherlands (1) · Turkey (Türkiye) (1)
May Orfali, MD (3)