7 completed, 1 recruiting, 1 terminated, 1 unknown. 11 Phase 1, 8 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 11 | Completed 7, Recruiting 1, Terminated 1, Unknown 1, Withdrawn 1 |
| Phase 2 | 8 | Completed 5, Terminated 1, Unknown 1, Withdrawn 1 |
Follow Leukodystrophy, Metachromatic: new trials by email Literature Review of Leukodystrophy, Metachromatic trials Research Report on Leukodystrophy, Metachromatic
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 Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD) Umbilical cord blood transplantation with HSC835 - Inherited Metabolic Disorders IMD | Phase 1 / Phase 2 | Withdrawn | Novartis Pharmaceuticals | 2017-10-10 | 2018-11-28 | |
| Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy transduced CD34+ hematopoietic stem cell - Metachromatic Leukodystrophy, Adrenoleukodystrophy | Phase 1 / Phase 2 | Unknown | Shenzhen Second People's Hospital | 50 | 2015-01-01 | 2022-05-31 |
| UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells DUOC-01 - Adrenoleukodystrophy, Batten Disease | Phase 1 | Recruiting | Joanne Kurtzberg, MD | 40 | 2014-09-01 | 2026-09-03 |
| Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy intracerebral administration of AAVrh.10cuARSA - Metachromatic Leukodystrophy | Phase 1 / Phase 2 | Completed | Institut National de la Santé Et de la Recherche Médicale, France | 5 | 2014-06-01 | 2026-03-02 |
| 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 |
| Human Placental-Derived Stem Cell Transplantation Human Placental Derived Stem Cell - Mucopolysaccharidosis I, Mucopolysaccharidosis VI | Phase 1 | Completed | New York Medical College | 43 | 2013-04-01 | 2022-10-25 |
| 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 |
| Gene Therapy for Metachromatic Leukodystrophy (MLD) OTL-200 Gene Therapy - Lysosomal Storage Disease, Metachromatic Leukodystrophy | Phase 1 / Phase 2 | Completed | Orchard Therapeutics | 20 | 2010-04-09 | 2025-12-05 |
| 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 |
| Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD) Recombinant human Arylsulfatase A (rhASA) - Late Infantile Metachromatic Leukodystrophy | Phase 1 / Phase 2 | Completed | Shire | 13 | 2007-01-22 | 2021-06-14 |
Adrenoleukodystrophy (4) · Leukodystrophy, Globoid Cell (3) · Mucopolysaccharidosis I (3) · Mucopolysaccharidosis II (2) · Neuronal Ceroid-Lipofuscinoses (2) · Niemann-Pick Diseases (2) · Sandhoff Disease (2) · Tay-Sachs Disease (2) · alpha-Mannosidosis (2) · Anemia, Aplastic (1) · Anemia, Diamond-Blackfan (1) · Brain Diseases, Metabolic, Inborn (1) · Fucosidosis (1) · Gaucher Disease (1)
Brain Diseases (11) · Brain Diseases, Metabolic (11) · Central Nervous System Diseases (11) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (11) · Demyelinating Diseases (11) · Genetic Diseases, Inborn (11) · Hereditary Central Nervous System Demyelinating Diseases (11) · Leukoencephalopathies (11) · Lipid Metabolism Disorders (11) · Lipid Metabolism, Inborn Errors (11) · Lipidoses (11) · Lysosomal Storage Diseases, Nervous System (11) · Metabolic Diseases (11) · Nervous System Diseases (11) · Nutritional and Metabolic Diseases (11)
Shire (4) · Institut National de la Santé Et de la Recherche Médicale, France (1) · Joanne Kurtzberg, MD (1) · New York Medical College (1) · Novartis Pharmaceuticals (1) · Orchard Therapeutics (1) · Shenzhen Second People's Hospital (1) · Talaris Therapeutics Inc. (1)
Denmark (4) · France (3) · United States (3) · Australia (2) · Germany (2) · Japan (2) · Brazil (1) · China (1) · Czechia (1) · Italy (1)
Caroline Sevin, MD-PhD (1) · GuangFu Chen, M.D.,Ph.D (1) · Hua Jiang, M.D,Ph.D (1) · Jiacai Zhuo (1) · Joanne Kurtzberg, MD (1) · Michel Zerah, MD, PhD (1) · Mitchell S Cairo, MD (1) · Nathalie Cartier, MD, PhD (1) · Patrick Aubourg, MD-PhD (1) · Qizhou Lian, M.D.,Ph.D. (1) · Suzanne T Ildstad, MD (1) · Thomas Roujeau, MD, PhD (1) · Xin Du, M.D.,Ph.D. (1)
PMID 33332761, PMID 11231629, PMID 11399225, PMID 19892975, PMID 23845948, PMID 23966770, PMID 25758611, PMID 26000324, PMID 27289174, PMID 32792226, PMID 35065785, PMID 40267426