4 suspended. 4 Phase 1, 4 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 4 | Suspended 4 |
| Phase 2 | 4 | Suspended 4 |
Follow Metabolism, Inborn Errors: new trials by email Literature Review of Metabolism, Inborn Errors trials Research Report on Metabolism, Inborn Errors
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 |
| Effect of Acetaminophen and N-Acetylcysteine on Liver Metabolism on Homocystinuria Acetaminophen, N-acetylcysteine - CBS Deficiency | Phase 1 / Phase 2 | Suspended | Hospital de Clinicas de Porto Alegre | 10 | 2022-02-11 | 2021-06-30 |
| RGX-111 Gene Therapy in Patients With MPS I RGX-111 - Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome | Phase 1 / Phase 2 | Suspended | REGENXBIO Inc. | 21 | 2019-04-03 | 2026-03-12 |
| Clinical Trial of Ambroxol in Patients With Type I Gaucher Disease Ambroxol - Type I Gaucher Disease | Phase 1 / Phase 2 | Suspended | Exsar Corporation | 20 | 2012-12-01 | 2013-02-15 |
Gaucher Disease (1) · Homocystinuria (1) · Leukodystrophy, Globoid Cell (1) · Lysosomal Storage Diseases (1) · Mucopolysaccharidosis I (1) · Rare Diseases (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Genetic Diseases, Inborn (4) · Metabolic Diseases (4) · Nutritional and Metabolic Diseases (4) · Brain Diseases (3) · Brain Diseases, Metabolic (3) · Brain Diseases, Metabolic, Inborn (3) · Central Nervous System Diseases (3) · Nervous System Diseases (3) · Connective Tissue Diseases (2) · Lipid Metabolism Disorders (2) · Lipid Metabolism, Inborn Errors (2) · Lipidoses (2) · Lysosomal Storage Diseases (2)
Acetaminophen (1) · Acetylcysteine (1) · Ambroxol (1)
Exsar Corporation (1) · Gemma Biotherapeutics (1) · Hospital de Clinicas de Porto Alegre (1) · REGENXBIO Inc. (1)
Brazil (3) · United States (3) · Canada (1) · Israel (1) · Netherlands (1) · United Kingdom (1)
Ida VD Schwartz, PhD (1) · May Orfali, MD (1) · Robert Johnston (1)