6 unknown. 6 Phase 2, 3 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 6 | Unknown 6 |
| Phase 3 | 3 | Unknown 3 |
Follow Mucinoses: new trials by email Literature Review of Mucinoses trials Research Report on Mucinoses
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 |
|---|---|---|---|---|---|---|
| An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day - Sanfilippo Syndrome, MPS3 | Phase 2 / Phase 3 | Unknown | Ozlem Goker-Alpan | 10 | 2024-12-01 | 2025-08-20 |
| Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) LYS-SAF302 - Mucopolysaccharidosis Type IIIA | Phase 2 / Phase 3 | Unknown | LYSOGENE | 20 | 2018-12-17 | 2021-08-31 |
| Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI Losartan, Placebo - Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI | Phase 2 | Unknown | Hospital de Clinicas de Porto Alegre | 10 | 2018-11-07 | 2022-12-19 |
| A Treatment Extension Study of Mucopolysaccharidosis Type IIIB AX 250 - Mucopolysaccharidosis Type IIIB, MPS III B | Phase 2 | Unknown | Allievex Corporation | 20 | 2018-02-19 | 2023-01-18 |
| Study of Idursulfase-beta (GC1111) in Hunter Syndrome idursulfase beta, idursulfase - Mucopolysaccharidosis II | Phase 2 | Unknown | Green Cross Corporation | 20 | 2016-12-01 | 2016-01-26 |
| 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 |
Mucopolysaccharidosis III (3) · Mucopolysaccharidoses (2) · Lysosomal Storage Diseases (1) · Mucopolysaccharidosis II (1) · Mucopolysaccharidosis IV (1) · Mucopolysaccharidosis VI (1)
Carbohydrate Metabolism, Inborn Errors (6) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (6) · Connective Tissue Diseases (6) · Genetic Diseases, Inborn (6) · Metabolic Diseases (6) · Metabolism, Inborn Errors (6) · Nutritional and Metabolic Diseases (6) · Skin and Connective Tissue Diseases (6) · Lysosomal Storage Diseases (5) · Mucopolysaccharidoses (4) · Genetic Diseases, X-Linked (1) · Heredodegenerative Disorders, Nervous System (1) · Intellectual Disability (1) · Nervous System Diseases (1)
Ambroxol (1) · Antilymphocyte Serum (1) · Cyclophosphamide (1) · Losartan (1) · Stem Cell Transplantation (1) · Tablets (1) · Transplantation Conditioning (1) · idursulfase (1) · idursulfase beta, mouse (1)
Allievex Corporation (1) · Green Cross Corporation (1) · Hospital de Clinicas de Porto Alegre (1) · LYSOGENE (1) · Ozlem Goker-Alpan (1) · Tehran University of Medical Sciences (1)
United States (3) · Germany (2) · United Kingdom (2) · Brazil (1) · Colombia (1) · France (1) · Iran (1) · Netherlands (1) · Spain (1) · Taiwan (1) · Turkey (Türkiye) (1)
Guilherme Baldo, PhD (1) · Ozlem Goker-Alpan, MD (1) · Roberto Giugliani, MD, PhD (1)