1 active not recruiting, 1 recruiting. 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 1 | Active not recruiting 1 |
Follow Alpha-Methylacyl-CoA Racemase Deficiency: new trials by email Literature Review of Alpha-Methylacyl-CoA Racemase Deficiency trials Research Report on Alpha-Methylacyl-CoA Racemase Deficiency
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 |
|---|---|---|---|---|---|---|
| MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen - Mucopolysaccharidosis Disorders, Hurler Syndrome | Phase 2 | Active not recruiting | Masonic Cancer Center, University of Minnesota | 149 | 2014-07-10 | 2026-01-07 |
| Longitudinal Natural History Study of Patients With Peroxisome Biogenesis Disorders (PBD) Peroxisome Biogenesis Disorder, Zellweger Spectrum Disorder | Recruiting | McGill University Health Centre/Research Institute of the McGill University Health Centre | 244 | 2012-01-01 | 2026-10-13 |
Peroxisomal ACYL-COA oxidase deficiency (2) · Pseudo-Zellweger syndrome (2) · Zellweger Syndrome (2) · Aspartylglucosaminuria (1) · Chondrodysplasia Punctata, Rhizomelic (1) · Fucosidosis (1) · Hereditary Diffuse Leukoencephalopathy with Spheroids (1) · Leukodystrophy, Globoid Cell (1) · Leukodystrophy, Metachromatic (1) · Mucopolysaccharidosis I (1) · Mucopolysaccharidosis II (1) · Mucopolysaccharidosis VI (1) · Mucopolysaccharidosis VII (1) · Osteopetrosis (1)
Abnormalities, Multiple (2) · Bone Diseases (2) · Bone Diseases, Developmental (2) · Brain Diseases (2) · Brain Diseases, Metabolic (2) · Brain Diseases, Metabolic, Inborn (2) · Central Nervous System Diseases (2) · Congenital Abnormalities (2) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Digestive System Diseases (2) · Female Urogenital Diseases (2) · Female Urogenital Diseases and Pregnancy Complications (2) · Genetic Diseases, Inborn (2) · Heredodegenerative Disorders, Nervous System (2) · Kidney Diseases (2)
Regimen B (1) · Stem Cell Transplantation (1)
Masonic Cancer Center, University of Minnesota (1) · McGill University Health Centre/Research Institute of the McGill University Health Centre (1)
Canada (1) · United States (1)
Nancy E Braverman, MD, MS (1) · Paul Orchard, M.D. (1)