2 completed, 1 active not recruiting, 1 recruiting, 1 unknown. 2 Phase 1, 2 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 2 | Active not recruiting 1, Recruiting 1 |
| Phase 2 | 2 | Active not recruiting 1, Unknown 1 |
Follow MPS VI: new trials by email Literature Review of MPS VI trials Research Report on MPS VI
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 |
|---|---|---|---|---|---|---|
| Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI Vosoritide Injection [Voxzogo] - MPS IVA, MPS VI | Phase 1 / Phase 2 | Active not recruiting | University of California, San Francisco | 6 | 2023-09-25 | 2026-03-10 |
| PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) Aldurazyme (laronidase) - MPS I, MPS II | Phase 1 | Recruiting | University of California, San Francisco | 10 | 2021-07-01 | 2026-03-17 |
| 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 |
| Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome Morquio Syndrome A, Maroteaux Lamy Syndrome | Completed | Greenwood Genetic Center | 17 | 2013-10-01 | 2015-08-13 | |
| Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02 MPS VI | Completed | BioMarin Pharmaceutical | 59 | 2011-06-01 | 2014-04-02 |
Mucopolysaccharidosis VI (5) · Mucopolysaccharidosis IV (4) · Gaucher Disease (1) · Mucopolysaccharidoses (1) · Mucopolysaccharidosis VII (1) · Sudden Infant Death (1) · Wolman Disease (1)
Carbohydrate Metabolism, Inborn Errors (5) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5) · Connective Tissue Diseases (5) · Genetic Diseases, Inborn (5) · Lysosomal Storage Diseases (5) · Metabolic Diseases (5) · Metabolism, Inborn Errors (5) · Mucinoses (5) · Nutritional and Metabolic Diseases (5) · Skin and Connective Tissue Diseases (5) · Mucopolysaccharidoses (4) · Brain Diseases (1) · Brain Diseases, Metabolic (1) · Brain Diseases, Metabolic, Inborn (1) · Central Nervous System Diseases (1)
GAA protein, human (1) · GALNS protein, human (1) · Iduronidase (1) · Losartan (1) · Sebelipase alfa (1) · galsulfase (1) · idursulfase (1) · vestronidase alfa (1) · vosoritide (1)
University of California, San Francisco (2) · BioMarin Pharmaceutical (1) · Greenwood Genetic Center (1) · Hospital de Clinicas de Porto Alegre (1)
United States (4) · Brazil (2) · Australia (1) · France (1) · Germany (1) · Portugal (1) · United Kingdom (1)
Ellen B Fung, PhD (1) · Guilherme Baldo, PhD (1) · Richard Curtis Rogers, MD (1) · Roberto Giugliani, MD, PhD (1) · Tippi MacKenzie, MD (1)
PMID 15324318, PMID 19948615, PMID 21637107, PMID 23510062, PMID 24764221, PMID 28374935, PMID 28657663, PMID 30337566, PMID 32102934, PMID 37955580, PMID 39891377, PMID 9918480