2 completed, 1 recruiting, 1 terminated. 4 Phase 1, 2 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 4 | Completed 2, Recruiting 1, Terminated 1 |
| Phase 2 | 2 | Completed 1, Terminated 1 |
Follow Mannosidase Deficiency Diseases: new trials by email Literature Review of Mannosidase Deficiency Diseases trials Research Report on Mannosidase Deficiency Diseases
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 |
|---|---|---|---|---|---|---|
| Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation Thiotepa--single daily dose, Thiotepa--escalated dose - Bone Marrow Failure Syndrome, Thalassemia | Phase 1 / Phase 2 | Completed | University of Florida | 6 | 2018-06-15 | 2023-09-15 |
| 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 |
| 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 |
| Safety Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosis Lamazym - Alpha Mannosidosis | Phase 1 | Completed | Zymenex A/S | 10 | 2010-10-01 | 2020-08-03 |
Adrenoleukodystrophy (3) · alpha-Mannosidosis (3) · Leukodystrophy, Globoid Cell (2) · Leukodystrophy, Metachromatic (2) · Mucopolysaccharidosis I (2) · Mucopolysaccharidosis II (2) · Sandhoff Disease (2) · Tay-Sachs Disease (2) · Anemia, Diamond-Blackfan (1) · Anemia, Hemolytic (1) · Anemia, Sickle Cell (1) · Bone Marrow Failure Disorders (1) · Brain Diseases, Metabolic, Inborn (1) · Common Variable Immunodeficiency (1) · Granulomatous Disease, Chronic (1)
Carbohydrate Metabolism, Inborn Errors (4) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Genetic Diseases, Inborn (4) · Lysosomal Storage Diseases (4) · Metabolic Diseases (4) · Metabolism, Inborn Errors (4) · Nutritional and Metabolic Diseases (4) · Adrenal Gland Diseases (3) · Adrenal Insufficiency (3) · Brain Diseases (3) · Brain Diseases, Metabolic (3) · Central Nervous System Diseases (3) · Connective Tissue Diseases (3) · Demyelinating Diseases (3) · Endocrine System Diseases (3)
Joanne Kurtzberg, MD (1) · Talaris Therapeutics Inc. (1) · University of Florida (1) · Zymenex A/S (1)
United States (3)
Allan M. Lund, MD (1) · Biljana Horn, MD (1) · Jens Fogh (1) · Joanne Kurtzberg, MD (1) · Suzanne T Ildstad, MD (1)