5 completed, 2 unknown, 1 terminated. 4 Phase 3, 3 Phase 2, 1 Not applicable.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 4 | Completed 2, Terminated 1, Unknown 1 |
| Phase 2 | 3 | Completed 2, Unknown 1 |
| Not applicable | 1 | Completed 1 |
Follow Mucopolysaccharidosis: new trials by email Literature Review of Mucopolysaccharidosis trials Research Report on Mucopolysaccharidosis
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 |
|---|---|---|---|---|---|---|
| The Effect of Enzyme Replacement Therapy in Mucopolysaccharidosis Enzyme Replacement Agent - Mucopolysaccharidosis | Not applicable | Completed | Hasan Kalyoncu University | 12 | 2020-02-01 | 2021-08-16 |
| A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) UX003 - Sly Syndrome, MPS VII | Phase 3 | Completed | Ultragenyx Pharmaceutical Inc | 12 | 2015-11-10 | 2020-07-30 |
| Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age UX003 - Sly Syndrome, MPS VII | Phase 2 | Completed | Ultragenyx Pharmaceutical Inc | 8 | 2015-07-21 | 2019-10-30 |
| A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) UX003, Placebo - MPS 7, Sly Syndrome | Phase 3 | Completed | Ultragenyx Pharmaceutical Inc | 12 | 2014-12-01 | 2020-07-30 |
| Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer. Mucopolysaccharidosis, Fabry Disease | Unknown | Johannes Gutenberg University Mainz | 75 | 2012-09-01 | 2013-03-01 | |
| 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 for Mucopolysaccharidosis Conditioning regimen, Graft-versus-host disease (GVHD) prophylaxis, Stem Cell Transplantation - Mucopolysaccharidosis | Phase 2 / Phase 3 | Unknown | Tehran University of Medical Sciences | 10 | 2009-09-01 | 2012-06-04 |
| 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 |
Mucopolysaccharidoses (8) · Lysosomal Storage Diseases (4) · Mucopolysaccharidosis VII (4) · Metabolic Diseases (3) · Rare Diseases (3) · Adrenoleukodystrophy (2) · Fucosidosis (2) · Leukodystrophy, Globoid Cell (2) · Leukodystrophy, Metachromatic (2) · Mucopolysaccharidosis I (2) · Mucopolysaccharidosis II (2) · Mucopolysaccharidosis VI (2) · alpha-Mannosidosis (2) · Aspartylglucosaminuria (1) · Canavan Disease (1)
Carbohydrate Metabolism, Inborn Errors (8) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (8) · Connective Tissue Diseases (8) · Genetic Diseases, Inborn (8) · Mucinoses (8) · Nutritional and Metabolic Diseases (8) · Skin and Connective Tissue Diseases (8) · Metabolism, Inborn Errors (7) · Metabolic Diseases (5) · Lysosomal Storage Diseases (4) · Pathologic Processes (4) · Pathological Conditions, Signs and Symptoms (4) · Brain Diseases (3) · Brain Diseases, Metabolic (3) · Brain Diseases, Metabolic, Inborn (3)
vestronidase alfa (3) · Cyclophosphamide (2) · Stem Cell Transplantation (2) · Alemtuzumab (1) · Antilymphocyte Serum (1) · Busulfan (1) · Cyclosporine (1) · Mycophenolic Acid (1) · Transplantation Conditioning (1)
Ultragenyx Pharmaceutical Inc (3) · Aldagen (1) · Hasan Kalyoncu University (1) · Johannes Gutenberg University Mainz (1) · Masonic Cancer Center, University of Minnesota (1) · Tehran University of Medical Sciences (1)
United States (5) · Portugal (2) · Brazil (1) · Germany (1) · Iran (1) · Mexico (1) · Spain (1) · Turkey (Türkiye) (1)
Chester Whitley, MD (1) · James Hinson, MD (1) · Joanne Kurtzberg, MD (1) · Mislen Bauer, MD (1) · Paul Harmatz, MD (1) · Paul Orchard, MD (1) · Raymond Wang, MD (1) · Susanne Pitz, MD (1) · Yavuz Yakut, Prof (1)
PMID 37415957, PMID 30467742, PMID 32063397, PMID 33874971, PMID 22543891, PMID 24388732, PMID 27561270, PMID 28081172, PMID 29478819, PMID 31383595, PMID 32201668, PMID 35331634
2015 (2)