5 completed, 2 terminated, 1 active not recruiting, 1 recruiting. 10 Phase 2, 2 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 10 | Completed 5, Terminated 2, Active not recruiting 1, Recruiting 1, Unknown 1 |
| Phase 1 | 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 |
| Trial on Safety and Efficacy of Velmanase Alfa Treatment in Pediatric Patients With Alpha-Mannosidosis Velmanase Alfa (e.g. Lamazym) - Alpha-Mannosidosis | Phase 2 | Completed | Chiesi Farmaceutici S.p.A. | 5 | 2016-12-01 | 2021-10-26 |
| 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 |
| 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 |
| Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis Lamazym - Alpha-Mannosidosis | Phase 2 | Completed | Zymenex A/S | 10 | 2012-01-01 | 2017-03-29 |
| 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 |
| Dose Finding Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosis Lamazym - Alpha Mannosidosis | Phase 2 | Unknown | Zymenex A/S | 10 | 2011-01-01 | 2012-09-26 |
| 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 |
| 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 |
| 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 |
alpha-Mannosidosis (8) · Mucopolysaccharidosis I (7) · Leukodystrophy, Globoid Cell (5) · Leukodystrophy, Metachromatic (5) · Adrenoleukodystrophy (4) · Mucopolysaccharidosis II (4) · Mucopolysaccharidosis VI (4) · Aspartylglucosaminuria (3) · Fucosidosis (3) · Mucopolysaccharidosis VII (3) · Peroxisomal Disorders (3) · Sphingolipidoses (3) · Anemia, Diamond-Blackfan (2) · Anemia, Sickle Cell (2) · Granulomatous Disease, Chronic (2)
Carbohydrate Metabolism, Inborn Errors (10) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (10) · Genetic Diseases, Inborn (10) · Lysosomal Storage Diseases (10) · Metabolic Diseases (10) · Nutritional and Metabolic Diseases (10) · Metabolism, Inborn Errors (9) · Brain Diseases (7) · Brain Diseases, Metabolic (7) · Brain Diseases, Metabolic, Inborn (7) · Central Nervous System Diseases (7) · Connective Tissue Diseases (7) · Mucinoses (7) · Nervous System Diseases (7)
Stem Cell Transplantation (5) · Alemtuzumab (3) · Busulfan (3) · Cyclophosphamide (3) · Bone Marrow Transplantation (2) · Cyclosporine (1) · Hydroxyurea (1) · Melphalan (1) · Mycophenolic Acid (1) · Regimen B (1) · Thiotepa (1) · fludarabine (1) · fludarabine phosphate (1) · thymoglobulin (1)
Masonic Cancer Center, University of Minnesota (4) · Zymenex A/S (2) · Chiesi Farmaceutici S.p.A. (1) · Paul Szabolcs (1) · Talaris Therapeutics Inc. (1) · University of Florida (1)
United States (7) · Denmark (3) · Austria (1) · Belgium (1) · France (1) · Germany (1) · Italy (1) · Spain (1) · United Kingdom (1)
Paul Orchard, MD (3) · Jens Fogh (2) · Allan M Lund, MD (1) · Allan M. Lund, MD (1) · Biljana Horn, MD (1) · Paul Orchard, M.D. (1) · Paul Szabolcs, MD (1) · Suzanne T Ildstad, MD (1)
PMID 21586746, PMID 32634238, PMID 32750127
2016 (1)