1 active not recruiting, 1 recruiting, 1 terminated. 1 Not applicable, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 1 | Terminated 1 |
| Phase 2 | 1 | Active not recruiting 1 |
Follow Pseudo-Zellweger syndrome: new trials by email Literature Review of Pseudo-Zellweger syndrome trials Research Report on Pseudo-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 |
|---|---|---|---|---|---|---|
| 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 | |
| Study of Bile Acids in Patients With Peroxisomal Disorders chenodeoxycholic acid, cholic acid, ursodiol - Infantile Refsum's Disease, Zellweger Syndrome | Not applicable | Terminated | University of Cincinnati | 25 | 2015-03-25 |
Zellweger Syndrome (3) · Alpha-Methylacyl-CoA Racemase Deficiency (2) · Peroxisomal ACYL-COA oxidase deficiency (2) · Peroxisomal Disorders (2) · Refsum Disease, Infantile (2) · Sphingolipidoses (2) · Adrenoleukodystrophy (1) · Aspartylglucosaminuria (1) · Chondrodysplasia Punctata, Rhizomelic (1) · Fucosidosis (1) · Hereditary Diffuse Leukoencephalopathy with Spheroids (1) · Leukodystrophy, Globoid Cell (1) · Leukodystrophy, Metachromatic (1) · Metabolism, Inborn Errors (1)
Abnormalities, Multiple (3) · Brain Diseases (3) · Brain Diseases, Metabolic (3) · Brain Diseases, Metabolic, Inborn (3) · Central Nervous System Diseases (3) · Congenital Abnormalities (3) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Digestive System Diseases (3) · Female Urogenital Diseases (3) · Female Urogenital Diseases and Pregnancy Complications (3) · Genetic Diseases, Inborn (3) · Heredodegenerative Disorders, Nervous System (3) · Kidney Diseases (3) · Liver Diseases (3) · Male Urogenital Diseases (3)
Chenodeoxycholic Acid (1) · Cholic Acid (1) · Regimen B (1) · Stem Cell Transplantation (1) · Ursodeoxycholic Acid (1)
Masonic Cancer Center, University of Minnesota (1) · McGill University Health Centre/Research Institute of the McGill University Health Centre (1) · University of Cincinnati (1)
Canada (1) · United States (1)
Kenneth Setchell (1) · Nancy E Braverman, MD, MS (1) · Paul Orchard, M.D. (1)