1 active not recruiting, 1 completed. 2 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 2 | Active not recruiting 1, Completed 1 |
Follow Zellweger Syndrome: new trials by email Literature Review of Zellweger Syndrome trials Research Report on Zellweger Syndrome
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 |
|---|---|---|---|---|---|---|
| Hydroxychloroquine Administration for Reduction of Pexophagy Hydroxychloroquine, Placebo - Zellweger Syndrome, Peroxisome Biogenesis Disorders | Phase 2 | Completed | The Hospital for Sick Children | 3 | 2019-01-11 | 2020-12-17 |
| 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 | 2026-11-03 |
Alpha-Methylacyl-CoA Racemase Deficiency (1) · Aspartylglucosaminuria (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) · Peroxisomal ACYL-COA oxidase deficiency (1) · Peroxisomal Disorders (1) · Peroxisome biogenesis disorders (1)
Abnormalities, Multiple (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) · Kidney Diseases (2) · Liver Diseases (2) · Male Urogenital Diseases (2) · Metabolic Diseases (2)
Hydroxychloroquine (1) · Regimen B (1) · Stem Cell Transplantation (1)
Masonic Cancer Center, University of Minnesota (1) · The Hospital for Sick Children (1)
Canada (1) · United States (1)
Neal Sondheimer, MD (1) · Paul Orchard, M.D. (1)