3 recruiting, 1 completed, 1 unknown. 1 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Unknown 1 |
| Phase 2 | 1 | Unknown 1 |
Follow Multiple Sulfatase Deficiency Disease: new trials by email Literature Review of Multiple Sulfatase Deficiency Disease trials Research Report on Multiple Sulfatase Deficiency 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 and Efficacy of SMART101 in Adult Patients With Hematological Malignancies After Haploidentical HSCT With Post-transplant Cyclophosphamide Allogeneic T cell progenitors, cultured ex-vivo - Hematological Malignancies | Phase 1 / Phase 2 | Unknown | Smart Immune SAS | 40 | 2023-06-06 | 2023-09-25 |
| MPS (RaDiCo Cohort) (RaDiCo-MPS) Mucopolysaccharidosis I, Mucopolysaccharidosis II | Recruiting | Institut National de la Santé Et de la Recherche Médicale, France | 1000 | 2017-12-20 | 2026-02-11 | |
| LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies Leukodystrophy, White Matter Disease | Completed | Children's Hospital of Philadelphia | 236 | 2017-01-06 | 2025-11-10 | |
| The Myelin Disorders Biorepository Project Leukodystrophy, White Matter Disease | Recruiting | Children's Hospital of Philadelphia | 12000 | 2016-12-08 | 2025-10-23 | |
| Longitudinal Study of Neurodegenerative Disorders Palliative Care, Hematopoetic Stem Cell Transplantation - MLD, Krabbe Disease | Recruiting | University of Pittsburgh | 1500 | 2012-01-11 | 2026-02-09 |
Leukodystrophy, Globoid Cell (3) · Leukoencephalopathies (3) · Pelizaeus-Merzbacher Disease (3) · Adrenoleukodystrophy (2) · Aicardi-Goutieres syndrome (2) · Alexander Disease (2) · Alexanders leukodystrophy (2) · Allan-Herndon-Dudley syndrome (2) · Attention Deficit Disorder with Hyperactivity (2) · CADASIL (2) · Canavan Disease (2) · Charcot-Marie-Tooth Disease (2) · Cockayne Syndrome (2) · Gangliosidoses (2)
Brain Diseases (5) · Brain Diseases, Metabolic (5) · Brain Diseases, Metabolic, Inborn (5) · Central Nervous System Diseases (5) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5) · Genetic Diseases, Inborn (5) · Lipid Metabolism Disorders (5) · Lipid Metabolism, Inborn Errors (5) · Lipidoses (5) · Lysosomal Storage Diseases, Nervous System (5) · Metabolic Diseases (5) · Metabolism, Inborn Errors (5) · Nervous System Diseases (5) · Nutritional and Metabolic Diseases (5) · Sphingolipidoses (5)
Palliative Care (1)
Children's Hospital of Philadelphia (2) · Institut National de la Santé Et de la Recherche Médicale, France (1) · Smart Immune SAS (1) · University of Pittsburgh (1)
United States (3) · France (2)
U01NS106845 (1), U54NS115052 (1)