2 unknown, 1 terminated. 2 Not applicable, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 2 | Unknown 2 |
| Phase 2 | 1 | Terminated 1 |
Follow Niemann-Pick Disease, Type B: new trials by email Literature Review of Niemann-Pick Disease, Type B trials Research Report on Niemann-Pick Disease, Type B
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 |
|---|---|---|---|---|---|---|
| Magnetic Resonance Spectroscopy in Acid Sphingomyelinase Deficiency MR Spectroscopy (MRS), MR Elastography (MRE) - Asmd, Visceral Type | Not applicable | Unknown | Eline C. B. Eskes | 34 | 2023-05-05 | 2023-06-15 |
| Biomarkers Of Lung InVolvement In ASMD eNose, Volatile exhaled breath sample, Condensate sample - Asmd, Visceral Type | Not applicable | Unknown | Eline C. B. Eskes | 68 | 2022-12-05 | 2023-06-22 |
| 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 |
Aspartylglucosaminuria (1) · Fucosidosis (1) · Leukodystrophy, Globoid Cell (1) · Leukodystrophy, Metachromatic (1) · Metabolism, Inborn Errors (1) · Mucopolysaccharidosis I (1) · Mucopolysaccharidosis VI (1) · Mucopolysaccharidosis VII (1) · Niemann-Pick Disease, Type C (1) · Peroxisomal Disorders (1) · Sphingolipidoses (1) · Wolman Disease (1) · alpha-Mannosidosis (1)
Brain Diseases (3) · Brain Diseases, Metabolic (3) · Brain Diseases, Metabolic, Inborn (3) · Central Nervous System Diseases (3) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Genetic Diseases, Inborn (3) · Hemic and Lymphatic Diseases (3) · Histiocytosis (3) · Histiocytosis, Non-Langerhans-Cell (3) · Lipid Metabolism Disorders (3) · Lipid Metabolism, Inborn Errors (3) · Lipidoses (3) · Lymphatic Diseases (3) · Lysosomal Storage Diseases (3) · Lysosomal Storage Diseases, Nervous System (3)
Alemtuzumab (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Cyclophosphamide (1) · Stem Cell Transplantation (1)
Eline C. B. Eskes (2) · Masonic Cancer Center, University of Minnesota (1)
Netherlands (2) · United States (1)
Carla EM Hollak, MD, PhD, prof (1) · Carla EM Hollak, prof dr (1) · Paul Orchard, MD (1)