11 completed, 5 recruiting, 3 terminated, 2 withdrawn. 8 Phase 2, 6 Phase 3, 3 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 8 | Completed 3, Terminated 2, Active not recruiting 1, Recruiting 1, Unknown 1 |
| Phase 3 | 6 | Completed 4, Terminated 1, Withdrawn 1 |
| Phase 1 | 3 | Completed 1, Recruiting 1, Terminated 1 |
Follow alpha-Mannosidosis: new trials by email Literature Review of alpha-Mannosidosis trials Research Report on alpha-Mannosidosis
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 |
|---|---|---|---|---|---|---|
| Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3 Velmanase Alfa - Alpha-Mannosidosis | Recruiting | Chiesi Farmaceutici S.p.A. | 5 | 2025-02-19 | 2025-05-16 | |
| Interventional Study to Assess Efficacy and Safety of Velmanase Alfa in Patients With Alpha Mannosidosis Velmanase Alfa, Placebo - Alpha-Mannosidosis | Phase 3 | Withdrawn | Chiesi Farmaceutici S.p.A. | 2021-01-11 | 2020-12-08 | |
| Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC data collection - Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes | Recruiting | Paul Szabolcs | 50 | 2020-08-20 | 2026-01-13 | |
| Biomarker for Mannosidosis Disease (BioMannosidosis) Alpha-Mannosidase B Deficiency, Lysosomal Alpha B Mannosidosis | Withdrawn | CENTOGENE GmbH Rostock | 2018-08-20 | 2023-02-13 | ||
| 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 |
| Evaluation of Long-term Efficacy of Treatment With Lamazym Lamazym - Alpha-Mannosidosis | Phase 3 | Completed | Zymenex A/S | 18 | 2015-02-01 | 2020-11-20 |
| 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 |
| Clinical Biomarkers in Alpha-mannosidosis Alpha-Mannosidosis | Completed | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 11 | 2014-07-24 | 2019-12-03 | |
| 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 |
| Lamazym Aftercare Study FR Designed to Provide Treatment for French Patients Lamazym - Alpha-Mannosidosis | Phase 3 | Completed | Chiesi Farmaceutici S.p.A. | 13 | 2013-08-01 | 2023-07-28 |
| Lamazym Aftercare Study Lamazym - Alpha-Mannosidosis | Phase 3 | Completed | Chiesi Farmaceutici S.p.A. | 8 | 2013-06-01 | 2023-07-28 |
| A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis Lamazym, Placebo - Alpha-Mannosidosis | Phase 3 | Completed | Zymenex A/S | 25 | 2012-08-01 | 2020-08-03 |
| Longitudinal Study of Neurodegenerative Disorders Palliative Care, Hematopoetic Stem Cell Transplantation - MLD, Krabbe Disease | Recruiting | University of Pittsburgh | 1500 | 2012-01-11 | 2026-02-09 | |
| 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 |
| 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 |
| 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 |
Showing 20 of 24. Sign in to see all of them.
Leukodystrophy, Globoid Cell (9) · Leukodystrophy, Metachromatic (8) · Mucopolysaccharidosis I (7) · Adrenoleukodystrophy (6) · Mucopolysaccharidosis II (6) · Fucosidosis (5) · Sudden Infant Death (5) · Aspartylglucosaminuria (4) · Mucopolysaccharidosis VI (4) · Osteopetrosis (4) · Sandhoff Disease (4) · Tay-Sachs Disease (4) · Gaucher Disease (3) · Lysosomal Storage Diseases (3)
Carbohydrate Metabolism, Inborn Errors (24) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (24) · Genetic Diseases, Inborn (24) · Nutritional and Metabolic Diseases (24) · Metabolic Diseases (23) · Mannosidase Deficiency Diseases (22) · Metabolism, Inborn Errors (22) · Lysosomal Storage Diseases (21) · Brain Diseases (10) · Brain Diseases, Metabolic (10) · Central Nervous System Diseases (10) · Lysosomal Storage Diseases, Nervous System (10) · Nervous System Diseases (10) · Brain Diseases, Metabolic, Inborn (9) · Connective Tissue Diseases (9)
Stem Cell Transplantation (4) · Alemtuzumab (3) · Busulfan (2) · Cyclophosphamide (2) · Bone Marrow Transplantation (1) · Cyclosporine (1) · Data Collection (1) · Hydroxyurea (1) · Melphalan (1) · Mycophenolic Acid (1) · Palliative Care (1) · Regimen B (1) · Thiotepa (1) · fludarabine (1) · fludarabine phosphate (1)
Chiesi Farmaceutici S.p.A. (6) · Zymenex A/S (6) · Masonic Cancer Center, University of Minnesota (3) · Paul Szabolcs (2) · Aldagen (1) · CENTOGENE GmbH Rostock (1) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (1) · Greenwood Genetic Center (1) · Joanne Kurtzberg, MD (1) · Talaris Therapeutics Inc. (1) · University of Pittsburgh (1)
United States (12) · Denmark (6) · Germany (4) · France (3) · United Kingdom (3) · Austria (1) · Belgium (1) · Czechia (1) · India (1) · Italy (1) · Norway (1) · Poland (1) · Spain (1) · Sri Lanka (1)
Jens Fogh (4) · Allan M Lund, MD (3) · Allan M. Lund, MD (2) · Joanne Kurtzberg, MD (2) · Paul Orchard, MD (2) · Paul Szabolcs, MD (2) · Dag Malm, MD (1) · Ed Wraith, MD (1) · James Hinson, MD (1) · Jens M Fogh, DVM (1) · Jiri Zeman, MD (1) · Michael Beck, MD (1) · Paul Harmatz, MD (1) · Paul Orchard, M.D. (1) · Peter Bauer, Prof. (1)
PMID 26048034, PMID 19617216, PMID 21586746, PMID 23494656, PMID 23786919, PMID 29716835, PMID 29725868, PMID 29846843, PMID 32634238, PMID 32750127
2016 (1)