5 completed, 2 terminated, 1 active not recruiting, 1 recruiting. 5 Phase 2, 2 Phase 1, 2 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Completed 3, Active not recruiting 1, Terminated 1 |
| Phase 1 | 2 | Completed 2 |
| Phase 3 | 2 | Completed 1, Terminated 1 |
Follow Fucosidosis: new trials by email Literature Review of Fucosidosis trials Research Report on Fucosidosis
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 Long-Term Observational Study of Patients With Fucosidosis Fucosidosis | Recruiting | JCR Pharmaceuticals Co., Ltd. | 57 | 2026-03-25 | 2026-09-17 | |
| 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 | 2026-01-07 |
| 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 |
| 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 |
| Longitudinal Studies of the Glycoproteinoses Non-interventional. - Aspartylglucosaminuria, Fucosidosis | Completed | Greenwood Genetic Center | 100 | 2009-08-01 | 2023-09-13 | |
| Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells Hematopoietic stem cell transplantation - Sickle Cell Disease, Thalassemia | Phase 1 / Phase 2 | Completed | Children's Hospital Los Angeles | 25 | 2008-08-01 | 2016-06-23 |
| 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 |
| ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases ALD-101 - Inherited Metabolic Diseases, Lysosomal Storage Disorders | Phase 3 | Terminated | Aldagen | 40 | 2008-03-01 | 2014-07-08 |
| Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation I Cell Disease, Fucosidosis | Unknown | Fairview University Medical Center | 10 | 1999-08-01 | 2005-06-24 | |
| 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, Globoid Cell (7) · Leukodystrophy, Metachromatic (7) · Mucopolysaccharidosis I (7) · Mucopolysaccharidosis VI (6) · Adrenoleukodystrophy (5) · Metabolism, Inborn Errors (5) · alpha-Mannosidosis (5) · Aspartylglucosaminuria (4) · Niemann-Pick Diseases (4) · Sphingolipidoses (4) · Wolman Disease (4) · Gaucher Disease (3) · Genetic Diseases, Inborn (3) · Lysosomal Storage Diseases (3)
Brain Diseases (10) · Brain Diseases, Metabolic (10) · Central Nervous System Diseases (10) · Nervous System Diseases (10) · Nutritional and Metabolic Diseases (10) · Brain Diseases, Metabolic, Inborn (9) · Carbohydrate Metabolism, Inborn Errors (9) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (9) · Lysosomal Storage Diseases, Nervous System (9) · Metabolic Diseases (9) · Lipid Metabolism Disorders (8) · Lipid Metabolism, Inborn Errors (8) · Lipidoses (8) · Connective Tissue Diseases (7) · Demyelinating Diseases (7)
Stem Cell Transplantation (4) · Busulfan (3) · Cyclophosphamide (3) · Alemtuzumab (2) · Bone Marrow Transplantation (2) · Antilymphocyte Serum (1) · Cyclosporine (1) · Hematopoietic Stem Cell Transplantation (1) · Mycophenolic Acid (1) · Regimen B (1)
Masonic Cancer Center, University of Minnesota (4) · Aldagen (1) · Children's Hospital Los Angeles (1) · Fairview University Medical Center (1) · Greenwood Genetic Center (1) · JCR Pharmaceuticals Co., Ltd. (1) · New York Medical College (1)
United States (10) · India (1) · Netherlands (1) · Tunisia (1) · Turkey (Türkiye) (1) · United Kingdom (1)
Paul Orchard, MD (3) · Hisham Abdel-Azim, MD (1) · James Hinson, MD (1) · Joanne Kurtzberg, MD (1) · K. Scott Baker (1) · Karolina M Stepien, MD (1) · Mitchell S Cairo, MD (1) · Paul Orchard, M.D. (1) · Sara Cathey, MD (1)