3 recruiting, 2 terminated, 1 active not recruiting, 1 enrolling by invitation. 3 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 3 | Active not recruiting 1, Recruiting 1, Terminated 1 |
Follow Hereditary Diffuse Leukoencephalopathy with Spheroids: new trials by email Literature Review of Hereditary Diffuse Leukoencephalopathy with Spheroids trials Research Report on Hereditary Diffuse Leukoencephalopathy with Spheroids
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 |
|---|---|---|---|---|---|---|
| A Study of VGL101 in Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia VGL101 - ALSP | Phase 2 | Terminated | Vigil Neuroscience, Inc. | 20 | 2022-12-14 | 2025-06-11 |
| Natural History Study in Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia No intervention - ALSP | Terminated | Vigil Neuroscience, Inc. | 56 | 2021-09-13 | 2025-08-13 | |
| Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia Blood sample collection - Adrenoleukodystrophy, Adrenomyeloneuropathy | Recruiting | Assistance Publique - Hôpitaux de Paris | 100 | 2021-08-30 | 2025-09-24 | |
| A Study to Assess CSF1R-related Leukoencephalopathy After Stem Cell Transplantation CSF1R-related Leukoencephalopathy, ALSP | Enrolling by invitation | Mayo Clinic | 20 | 2020-07-21 | 2026-01-08 | |
| 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 |
| Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT Hydroxyurea, Alemtuzumab, Fludarabine - Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes | Phase 2 | Recruiting | Paul Szabolcs | 100 | 2014-02-04 | 2025-12-15 |
| Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford Rare Disorders, Undiagnosed Disorders | Recruiting | Sanford Health | 20000 | 2010-07-01 | 2026-10-28 |
Leukodystrophy, Metachromatic (4) · Leukoencephalopathies (3) · Adrenoleukodystrophy (2) · Leukodystrophy, Globoid Cell (2) · Lymphohistiocytosis, Hemophagocytic (2) · Mucopolysaccharidosis I (2) · Mucopolysaccharidosis II (2) · Osteopetrosis (2) · Syndrome (2) · alpha-Mannosidosis (2) · 3-Methylglutaconic Aciduria, Type V (1) · Achalasia Addisonianism Alacrimia syndrome (1) · Acrodysostosis (1) · Activated PI3K-delta Syndrome (1)
Brain Diseases (7) · Central Nervous System Diseases (6) · Nervous System Diseases (6) · Brain Diseases, Metabolic (4) · Brain Diseases, Metabolic, Inborn (4) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Demyelinating Diseases (4) · Genetic Diseases, Inborn (4) · Genetic Diseases, X-Linked (4) · Hereditary Central Nervous System Demyelinating Diseases (4) · Heredodegenerative Disorders, Nervous System (4) · Intellectual Disability (4) · Leukoencephalopathies (4) · Lipid Metabolism Disorders (4) · Lipid Metabolism, Inborn Errors (4)
Alemtuzumab (1) · Hydroxyurea (1) · Melphalan (1) · Regimen B (1) · Stem Cell Transplantation (1) · Thiotepa (1) · fludarabine (1) · fludarabine phosphate (1)
Vigil Neuroscience, Inc. (2) · Assistance Publique - Hôpitaux de Paris (1) · Masonic Cancer Center, University of Minnesota (1) · Mayo Clinic (1) · Paul Szabolcs (1) · Sanford Health (1)
United States (6) · France (2) · Germany (2) · Netherlands (2) · United Kingdom (2) · Brazil (1) · Canada (1)
Paul Orchard, M.D. (1) · Paul Szabolcs, MD (1) · Zbigniew K Wszolek, MD (1)