1 active not recruiting, 1 completed, 1 recruiting, 1 suspended. 5 Phase 1, 3 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 5 | Active not recruiting 1, Completed 1, Recruiting 1, Suspended 1, Terminated 1 |
| Phase 2 | 3 | Active not recruiting 1, Suspended 1, Terminated 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 |
|---|---|---|---|---|---|---|
| Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT FBX-101 - Krabbe Disease | Phase 1 / Phase 2 | Active not recruiting | Forge Biologics, Inc | 9 | 2023-02-03 | 2025-11-20 |
| 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 |
| UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells DUOC-01 - Adrenoleukodystrophy, Batten Disease | Phase 1 | Recruiting | Joanne Kurtzberg, MD | 40 | 2014-09-01 | 2026-09-03 |
| Human Placental-Derived Stem Cell Transplantation Human Placental Derived Stem Cell - Mucopolysaccharidosis I, Mucopolysaccharidosis VI | Phase 1 | Completed | New York Medical College | 43 | 2013-04-01 | 2022-10-25 |
| Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders hematopoietic stem cell infusion - Hurler Syndrome (MPS I), Hurler-Scheie Syndrome | Phase 1 / Phase 2 | Terminated | Talaris Therapeutics Inc. | 3 | 2011-04-01 | 2023-04-12 |
Adrenoleukodystrophy (3) · Leukodystrophy, Metachromatic (3) · Brain Diseases, Metabolic, Inborn (2) · Lysosomal Storage Diseases (2) · Metabolism, Inborn Errors (2) · Mucopolysaccharidosis I (2) · Mucopolysaccharidosis II (2) · Neuronal Ceroid-Lipofuscinoses (2) · Niemann-Pick Diseases (2) · Sandhoff Disease (2) · Tay-Sachs Disease (2) · alpha-Mannosidosis (2) · Anemia, Aplastic (1) · Anemia, Diamond-Blackfan (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5) · Nutritional and Metabolic Diseases (5) · Brain Diseases (4) · Brain Diseases, Metabolic (4) · Central Nervous System Diseases (4) · Demyelinating Diseases (4) · Genetic Diseases, Inborn (4) · Hereditary Central Nervous System Demyelinating Diseases (4) · Heredodegenerative Disorders, Nervous System (4) · Leukoencephalopathies (4) · Lipid Metabolism Disorders (4) · Lipid Metabolism, Inborn Errors (4) · Lipidoses (4) · Lysosomal Storage Diseases, Nervous System (4) · Metabolic Diseases (4)
Forge Biologics, Inc (1) · Gemma Biotherapeutics (1) · Joanne Kurtzberg, MD (1) · New York Medical College (1) · Talaris Therapeutics Inc. (1)
United States (5) · Brazil (1) · Canada (1) · Israel (1) · Netherlands (1) · United Kingdom (1)
Joanne Kurtzberg, MD (1) · May Orfali, MD (1) · Mitchell S Cairo, MD (1) · Suzanne T Ildstad, MD (1)