12 completed, 3 terminated, 2 unknown, 1 active not recruiting. 20 Phase 2, 9 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 20 | Completed 12, Terminated 3, Unknown 2, Active not recruiting 1, Recruiting 1, Withdrawn 1 |
| Phase 1 | 9 | Completed 5, Unknown 2, Terminated 1, Withdrawn 1 |
| Phase 3 | 1 | Completed 1 |
Follow Sulfatidosis: new trials by email Literature Review of Sulfatidosis trials Research Report on Sulfatidosis
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 |
| 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 |
| MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT) MGTA-456 - Inherited Metabolic Disorders (IMD) | Phase 2 | Completed | Magenta Therapeutics, Inc. | 8 | 2018-02-09 | 2021-11-03 |
| A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD) OTL-200 - Lysosomal Storage Disease, Metachromatic Leukodystrophy | Phase 2 | Completed | Orchard Therapeutics | 10 | 2018-01-25 | 2026-03-16 |
| 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(IMD) Umbilical cord blood transplantation with HSC835 - Inherited Metabolic Disorders IMD | Phase 1 / Phase 2 | Withdrawn | Novartis Pharmaceuticals | 2017-10-10 | 2018-11-28 | |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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation rhARSA - Metachromatic Leukodystrophy | Phase 2 | Completed | Assistance Publique - Hôpitaux de Paris | 1 | 2008-10-01 | 2026-03-27 |
| Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism Stem Cell Transplantation, Cyclophosphamide, Campath-1H - Hurler's Syndrome, Maroteaux-Lamy Syndrome | Phase 2 | Terminated | Masonic Cancer Center, University of Minnesota | 18 | 2008-03-01 | 2017-12-28 |
| Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD HGT-1111 - Late Infantile Metachromatic Leukodystrophy | Phase 2 | Terminated | Shire | 11 | 2008-02-20 | 2021-06-08 |
| 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 |
| HSCT for High Risk Inherited Inborn Errors Clofarabine, Total body Irradiation, Melphalan - Adrenoleukodystrophy, Metachromatic Leukodystrophy | Phase 2 | Completed | Masonic Cancer Center, University of Minnesota | 38 | 2006-09-01 | 2019-07-11 |
| Stem Cell Transplant for Inborn Errors of Metabolism Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin - Adrenoleukodystrophy, Metachromatic Leukodystrophy | Phase 2 / Phase 3 | Completed | Masonic Cancer Center, University of Minnesota | 135 | 1995-01-01 | 2017-12-28 |
Leukodystrophy, Metachromatic (19) · Leukodystrophy, Globoid Cell (8) · Mucopolysaccharidosis I (7) · Adrenoleukodystrophy (6) · alpha-Mannosidosis (5) · Fucosidosis (4) · Mucopolysaccharidosis II (4) · Aspartylglucosaminuria (3) · Mucopolysaccharidosis VI (3) · Mucopolysaccharidosis VII (3) · Peroxisomal Disorders (3) · Sandhoff Disease (3) · Sphingolipidoses (3) · Tay-Sachs Disease (3) · Wolman Disease (3)
Brain Diseases (20) · Brain Diseases, Metabolic (20) · Brain Diseases, Metabolic, Inborn (20) · Central Nervous System Diseases (20) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (20) · Genetic Diseases, Inborn (20) · Lipid Metabolism Disorders (20) · Lipid Metabolism, Inborn Errors (20) · Lipidoses (20) · Lysosomal Storage Diseases, Nervous System (20) · Metabolic Diseases (20) · Nervous System Diseases (20) · Nutritional and Metabolic Diseases (20) · Demyelinating Diseases (19)
Stem Cell Transplantation (5) · Alemtuzumab (4) · Busulfan (3) · Cyclophosphamide (3) · Bone Marrow Transplantation (2) · Hydroxyurea (2) · Melphalan (2) · Antilymphocyte Serum (1) · Clofarabine (1) · Cyclosporine (1) · Hematopoietic Stem Cell Transplantation (1) · Mycophenolic Acid (1) · Regimen B (1) · Thiotepa (1) · Whole-Body Irradiation (1)
Masonic Cancer Center, University of Minnesota (5) · Shire (5) · Orchard Therapeutics (2) · Assistance Publique - Hôpitaux de Paris (1) · Institut National de la Santé Et de la Recherche Médicale, France (1) · Magenta Therapeutics, Inc. (1) · Novartis Pharmaceuticals (1) · Paul Szabolcs (1) · Shenzhen Second People's Hospital (1) · Smart Immune SAS (1) · Talaris Therapeutics Inc. (1)
United States (9) · France (6) · Denmark (4) · Germany (3) · Italy (3) · Japan (3) · Australia (2) · Brazil (2) · Argentina (1) · Belgium (1) · Canada (1) · China (1) · Czechia (1) · Greece (1) · Israel (1)
Paul Orchard, MD (4) · Caroline Sevin, MD-PhD (1) · GuangFu Chen, M.D.,Ph.D (1) · Hua Jiang, M.D,Ph.D (1) · Jiacai Zhuo (1) · Michel Zerah, MD, PhD (1) · Nathalie Cartier, MD, PhD (1) · Patrick Aubourg, MD, PhD (1) · Patrick Aubourg, MD-PhD (1) · Paul Orchard, M.D. (1) · Paul Szabolcs, MD (1)
PMID 21586746, PMID 33332761, PMID 40267426, PMID 11231629, PMID 11399225, PMID 19892975, PMID 23845948, PMID 23966770, PMID 25758611, PMID 27289174, PMID 32634238, PMID 32750127, PMID 32792226, PMID 35065785
2008 (1), 2017 (1), 2018 (1)