5 completed, 3 recruiting, 2 terminated, 2 unknown. 2 Phase 2, 1 Not applicable, 1 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 2 | Completed 2 |
| Not applicable | 1 | Completed 1 |
| Phase 1 | 1 | Enrolling by invitation 1 |
| Phase 3 | 1 | Terminated 1 |
Follow Mucolipidoses: new trials by email Literature Review of Mucolipidoses trials Research Report on Mucolipidoses
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 AAV9. hMCOLN1co For Patients With Mucolipidosis Type IV AAV9.hMCOLN1co - Mucolipidosis Type IV | Phase 1 | Enrolling by invitation | The Children's Hospital of Zhejiang University School of Medicine | 1 | 2026-01-13 | 2026-02-10 |
| Mucolipidosis Type IV Natural History Study Mucolipidosis Type IV | Active not recruiting | Massachusetts General Hospital | 50 | 2023-03-15 | 2026-04-23 | |
| The Natural History of Sialidosis Type I Observational study - Observational Study | Recruiting | National Taiwan University Hospital | 30 | 2022-03-15 | 2025-05-20 | |
| Registry Gangliosidoses Gangliosidoses, GM1 Gangliosidosis | Unknown | SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt) | 40 | 2020-06-08 | 2020-11-12 | |
| Biomarker for Glycogen Storage Diseases (BioGlycogen) Fructose Metabolism, Inborn Errors, Glycogen Storage Disease | Withdrawn | CENTOGENE GmbH Rostock | 2018-08-20 | 2023-02-13 | ||
| Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders Campath-1H, Clofarabine, Melphalan - Lysosomal Storage Disease, Peroxisomal Disorder | Not applicable | Completed | Masonic Cancer Center, University of Minnesota | 3 | 2012-07-11 | 2017-12-05 |
| 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 | |
| Longitudinal Studies of the Glycoproteinoses Non-interventional. - Aspartylglucosaminuria, Fucosidosis | Completed | Greenwood Genetic Center | 100 | 2009-08-01 | 2023-09-13 | |
| The Natural History of Mucolipidosis Type IV Mucolipidosis Type IV | Terminated | Baylor Research Institute | 7 | 2009-02-19 | 2026-01-26 | |
| 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 |
| 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 |
| Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders Neurological Regression, Myoclonus | Recruiting | National Human Genome Research Institute (NHGRI) | 200 | 2002-02-06 | 2026-10-20 | |
| The Natural History and Pathogenesis of Mucolipidosis Type IV Mucolipidosis Type IV | Completed | National Institute of Neurological Disorders and Stroke (NINDS) | 30 | 2001-04-27 | 2017-07-02 | |
| 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 Transplantation for Hurler Stem Cell Transplant, Busulfan, Cyclophosphamide, ATG - Mucopolysaccharidosis I, Mucopolysaccharidosis VI | Phase 2 | Completed | Masonic Cancer Center, University of Minnesota | 41 | 1999-05-01 | 2017-12-28 |
Lysosomal Storage Diseases (6) · Gangliosidosis, GM1 (5) · Leukodystrophy, Metachromatic (5) · Adrenoleukodystrophy (4) · Leukodystrophy, Globoid Cell (4) · Sandhoff Disease (4) · Tay-Sachs Disease (4) · Fucosidosis (3) · Metabolism, Inborn Errors (3) · Mucopolysaccharidosis I (3) · Mucopolysaccharidosis III (3) · Mucopolysaccharidosis VI (3) · Wolman Disease (3) · Gangliosidoses, GM2 (2)
Bone Diseases (15) · Bone Diseases, Metabolic (15) · Brain Diseases (15) · Brain Diseases, Metabolic (15) · Brain Diseases, Metabolic, Inborn (15) · Carbohydrate Metabolism, Inborn Errors (15) · Central Nervous System Diseases (15) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (15) · Lysosomal Storage Diseases, Nervous System (15) · Musculoskeletal Diseases (15) · Nervous System Diseases (15) · Nutritional and Metabolic Diseases (15) · Genetic Diseases, Inborn (14) · Metabolic Diseases (13) · Metabolism, Inborn Errors (12)
Alemtuzumab (2) · Clofarabine (2) · Hematopoietic Stem Cell Transplantation (2) · Melphalan (2) · Whole-Body Irradiation (2) · Bone Marrow Transplantation (1) · Busulfan (1) · Cyclophosphamide (1) · Cyclosporine (1) · Hydroxyurea (1) · Mycophenolic Acid (1) · Observation (1) · Radiotherapy, Intensity-Modulated (1) · Stem Cell Transplantation (1) · thymoglobulin (1)
Masonic Cancer Center, University of Minnesota (3) · Aldagen (1) · Baylor Research Institute (1) · CENTOGENE GmbH Rostock (1) · Fairview University Medical Center (1) · Greenwood Genetic Center (1) · Massachusetts General Hospital (1) · National Human Genome Research Institute (NHGRI) (1) · National Institute of Neurological Disorders and Stroke (NINDS) (1) · National Taiwan University Hospital (1) · Sanford Health (1) · SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt) (1) · The Children's Hospital of Zhejiang University School of Medicine (1)
United States (11) · Germany (2) · China (1) · India (1) · Sri Lanka (1) · Taiwan (1)
Paul Orchard, MD (2) · Cynthia J Tifft, M.D. (1) · James Hinson, MD (1) · Joanne Kurtzberg, MD (1) · K. Scott Baker (1) · Patricia Musolino, MD, PHD (1) · Peter Bauer, Prof. (1) · Sara Cathey, MD (1) · Weston Miller, MD (1)
PMID 19617216, PMID 21586746, PMID 31883529, PMID 32753397, PMID 36194207, PMID 36461157, PMID 36700853, PMID 38641994
U54NS065768 (1)