1 active not recruiting, 1 completed, 1 not yet recruiting, 1 terminated. 4 Phase 2, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Active not recruiting 1, Completed 1, Not yet recruiting 1, Terminated 1 |
| Phase 1 | 1 | Active not recruiting 1 |
Follow Krabbe Disease: new trials by email Literature Review of Krabbe Disease trials Research Report on Krabbe Disease
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 |
|---|---|---|---|---|---|---|
| Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol) PLX-200 - Lysosomal Storage Disorders, Sandhoff Disease | Phase 2 | Not yet recruiting | Polaryx Therapeutics, Inc. | 24 | 2026-12-01 | 2026-07-31 |
| 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 | Duke University | 9 | 2023-02-03 | 2026-11-04 |
| Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Campath-1H, Cyclophosphamide, Busulfan - Mucopolysaccharidosis, Hurler Syndrome | Phase 2 | Completed | Masonic Cancer Center, University of Minnesota | 46 | 2009-12-01 | 2018-02-05 |
| Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism Stem Cell Transplantation, Cyclophosphamide, Campath-1H - Hurler's Syndrome, Maroteaux-Lamy Syndrome | Phase 2 | Terminated | Masonic Cancer Center, University of Minnesota | 18 | 2008-03-01 | 2017-12-28 |
Leukodystrophy, Globoid Cell (4) · Sphingolipidoses (3) · Aspartylglucosaminuria (2) · Fucosidosis (2) · Leukodystrophy, Metachromatic (2) · Lysosomal Storage Diseases (2) · Metabolism, Inborn Errors (2) · Mucopolysaccharidosis I (2) · Mucopolysaccharidosis VI (2) · Mucopolysaccharidosis VII (2) · Peroxisomal Disorders (2) · alpha-Mannosidosis (2) · Adrenoleukodystrophy (1) · Brain Diseases (1) · Brain Diseases, Metabolic (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Nutritional and Metabolic Diseases (4) · Brain Diseases (3) · Brain Diseases, Metabolic (3) · Brain Diseases, Metabolic, Inborn (3) · Central Nervous System Diseases (3) · Demyelinating Diseases (3) · Genetic Diseases, Inborn (3) · Hereditary Central Nervous System Demyelinating Diseases (3) · Leukoencephalopathies (3) · Lipid Metabolism Disorders (3) · Lipid Metabolism, Inborn Errors (3) · Lipidoses (3) · Lysosomal Storage Diseases, Nervous System (3) · Metabolic Diseases (3)
Alemtuzumab (2) · Busulfan (2) · Cyclophosphamide (2) · Stem Cell Transplantation (2) · Bone Marrow Transplantation (1) · Cyclosporine (1) · Gemfibrozil (1) · Mycophenolic Acid (1)
Masonic Cancer Center, University of Minnesota (2) · Duke University (1) · Polaryx Therapeutics, Inc. (1)
United States (3)
Paul Orchard, MD (2) · Joanne Kurtzberg, MD (1)