5 completed, 2 not yet recruiting, 2 terminated, 1 recruiting. 5 Phase 2, 4 Phase 1, 2 Not applicable.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Completed 4, Terminated 1 |
| Phase 1 | 4 | Completed 3, Terminated 1 |
| Not applicable | 2 | Not yet recruiting 2 |
Follow Metachromatic Leukodystrophy (MLD): new trials by email Literature Review of Metachromatic Leukodystrophy (MLD) trials Research Report on Metachromatic Leukodystrophy (MLD)
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 |
|---|---|---|---|---|---|---|
| Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD Lentiviral TYF-ARSA correction of patient's autologous HSCs - Metachromatic Leukodystrophy (MLD) | Not applicable | Not yet recruiting | Shenzhen Geno-Immune Medical Institute | 10 | 2027-06-01 | 2026-11-02 |
| Direct Lentiviral Injection Gene Therapy for MLD Intrathecal and intravenous LV gene therapy - Metachromatic Leukodystrophy (MLD) | Not applicable | Not yet recruiting | Shenzhen Geno-Immune Medical Institute | 10 | 2027-05-31 | 2026-11-02 |
| A Study to Learn About Metachromatic Leukodystrophy (MLD) in Children in Spain No Intervention - Metachromatic Leukodystrophy (MLD) | Withdrawn | Takeda | 2023-11-30 | 2024-01-12 | ||
| A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy SHP611 - Metachromatic Leukodystrophy (MLD) | Phase 2 | Completed | Shire | 36 | 2019-05-13 | 2026-07-10 |
| Natural History Study of Children With Metachromatic Leukodystrophy Lipid Metabolism Disorders, Metachromatic Leukodystrophy (MLD) | Terminated | Shire | 1 | 2015-11-02 | 2021-03-17 | |
| An Efficacy and Safety Study of HGT-1110 in Participants With Metachromatic Leukodystrophy HGT-1110 - Metachromatic Leukodystrophy (MLD) | Phase 1 / Phase 2 | Completed | Shire | 24 | 2013-05-23 | 2026-04-21 |
| Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD) Recombinant human arylsulfatase A - Metachromatic Leukodystrophy (MLD) | Phase 1 / Phase 2 | Completed | Shire | 24 | 2012-02-02 | 2021-06-14 |
| Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders hematopoietic stem cell infusion - Hurler Syndrome (MPS I), Hurler-Scheie Syndrome | Phase 1 / Phase 2 | Terminated | Talaris Therapeutics Inc. | 3 | 2011-04-01 | 2023-04-12 |
| 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 | |
| Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Campath-1H, Cyclophosphamide, Busulfan - Mucopolysaccharidosis, Hurler Syndrome | Phase 2 | Completed | Masonic Cancer Center, University of Minnesota | 46 | 2009-12-01 | 2018-02-05 |
| Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD) rhASA - Dose Level 1, rhASA - Dose Level 2, rhASA - Dose Level 3 - Metachromatic Leukodystrophy (MLD) | Phase 1 | Completed | Shire | 13 | 2007-01-22 | 2021-06-25 |
Leukodystrophy, Metachromatic (11) · Adrenoleukodystrophy (2) · Leukodystrophy, Globoid Cell (2) · Mucopolysaccharidosis I (2) · Mucopolysaccharidosis II (2) · Sphingolipidoses (2) · alpha-Mannosidosis (2) · 3-Methylglutaconic Aciduria, Type V (1) · Achalasia Addisonianism Alacrimia syndrome (1) · Acrodysostosis (1) · Activated PI3K-delta Syndrome (1) · Addison Disease (1) · Alagille Syndrome (1) · Alstrom Syndrome (1) · Amnesia, Transient Global (1)
Brain Diseases, Metabolic (11) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (11) · Leukoencephalopathies (11) · Lipid Metabolism, Inborn Errors (11) · Lipidoses (11) · Lysosomal Storage Diseases, Nervous System (11) · Nutritional and Metabolic Diseases (11) · Brain Diseases (10) · Brain Diseases, Metabolic, Inborn (10) · Central Nervous System Diseases (10) · Demyelinating Diseases (10) · Genetic Diseases, Inborn (10) · Hereditary Central Nervous System Demyelinating Diseases (10) · Lipid Metabolism Disorders (10) · Lysosomal Storage Diseases (10)
Stem Cell Transplantation (2) · Alemtuzumab (1) · Busulfan (1) · Cyclophosphamide (1) · Cyclosporine (1) · Mycophenolic Acid (1)
Shire (5) · Shenzhen Geno-Immune Medical Institute (2) · Masonic Cancer Center, University of Minnesota (1) · Sanford Health (1) · Takeda (1) · Talaris Therapeutics Inc. (1)
United States (5) · Denmark (4) · France (4) · Germany (4) · Japan (4) · Brazil (3) · Argentina (2) · Australia (2) · Belgium (2) · Canada (2) · China (2) · Spain (2) · Czechia (1) · Greece (1) · Israel (1)
Lung-Ji Chang, Ph.D (1) · Paul Orchard, MD (1) · Suzanne T Ildstad, MD (1)
PMID 26000324, PMID 32792226, PMID 33332761
2018 (1)