1 suspended. 1 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Suspended 1 |
| Phase 2 | 1 | Suspended 1 |
Follow Leukodystrophy, Globoid Cell: new trials by email Literature Review of Leukodystrophy, Globoid Cell trials Research Report on Leukodystrophy, Globoid Cell
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 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 |
Lysosomal Storage Diseases (1) · Rare Diseases (1)
Brain Diseases (1) · Brain Diseases, Metabolic (1) · Brain Diseases, Metabolic, Inborn (1) · Central Nervous System Diseases (1) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (1) · Demyelinating Diseases (1) · Disease Attributes (1) · Genetic Diseases, Inborn (1) · Hereditary Central Nervous System Demyelinating Diseases (1) · Leukoencephalopathies (1) · Lipid Metabolism Disorders (1) · Lipid Metabolism, Inborn Errors (1) · Lipidoses (1) · Lysosomal Storage Diseases, Nervous System (1) · Metabolic Diseases (1)
Brazil (1) · Canada (1) · Israel (1) · Netherlands (1) · United Kingdom (1) · United States (1)
May Orfali, MD (1)