6 completed, 3 terminated, 1 approved for marketing, 1 recruiting. 5 Phase 2, 3 Phase 1, 2 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Terminated 3, Completed 2 |
| Phase 1 | 3 | Completed 1, Recruiting 1, Terminated 1 |
| Phase 3 | 2 | Completed 2 |
Follow GALNS protein, human: new trials by email Literature Review of GALNS protein, human trials Research Report on GALNS protein, human
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 |
|---|---|---|---|---|---|---|
| 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 |
| Natural History of Atypical Morquio A Disease Elosulfase Alfa 1 MG/ML Intravenous Solution [VIMIZIM] - Mucopolysaccharidosis IV A | Completed | GOIZET | 7 | 2018-02-01 | 2025-10-03 | |
| A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) Vimizim® (elosulfase alfa) - Mucopolysaccharidosis IV Type A, Morquio A Syndrome | Completed | BioMarin Pharmaceutical | 418 | 2014-09-01 | 2024-03-05 | |
| BMN 110 Phase 3B in Australian Patients BMN 110 - Mucopolysaccharidosis IVA (Morquio A Syndrome) | Phase 3 | Completed | BioMarin Pharmaceutical | 13 | 2013-07-01 | 2019-09-26 |
| Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation BMN 110 - Mucopolysaccharidosis IVA, Morquio A Syndrome | Phase 2 | Terminated | BioMarin Pharmaceutical | 16 | 2012-08-01 | 2016-01-12 |
| Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome BMN 110 - Mucopolysaccharidosis IVA, Morquio A Syndrome | Phase 2 | Terminated | BioMarin Pharmaceutical | 25 | 2012-04-01 | 2016-02-01 |
| Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome) BMN 110 - Mucopolysaccharidosis IVA, Morquio A Syndrome | Phase 2 | Completed | BioMarin Pharmaceutical | 15 | 2011-10-01 | 2017-08-10 |
| Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) BMN 110 - Weekly, BMN 110 - Every Other Week - Mucopolysaccharidosis IV A, Morquio A Syndrome | Phase 3 | Completed | BioMarin Pharmaceutical | 173 | 2011-07-01 | 2021-07-21 |
| A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) BMN 110 - MPS IV A, Mucopolysaccharidosis IVA | Phase 1 / Phase 2 | Terminated | BioMarin Pharmaceutical | 20 | 2010-11-01 | 2015-09-30 |
| A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA BMN 110 - MPS IV A | Phase 1 / Phase 2 | Completed | BioMarin Pharmaceutical | 20 | 2009-04-01 | 2014-06-30 |
| BMN 110 US Expanded Access Program BMN 110 - Mucopolysaccharidosis IVA, Morquio A Syndrome | Approved for marketing | BioMarin Pharmaceutical | 2014-04-02 |
Mucopolysaccharidosis IV (11) · Lysosomal Storage Diseases (7) · Gaucher Disease (1) · Mucopolysaccharidosis VI (1) · Mucopolysaccharidosis VII (1) · Sudden Infant Death (1) · Wolman Disease (1)
BioMarin Pharmaceutical (9) · GOIZET (1) · University of California, San Francisco (1)
United Kingdom (7) · United States (7) · Canada (3) · France (3) · Germany (3) · Italy (3) · Taiwan (3) · Australia (2) · Denmark (2) · Netherlands (2) · Portugal (2) · Puerto Rico (2) · Argentina (1) · Austria (1) · Belgium (1)
Celeste Decker, MD (2) · Debra Lounsbury (2) · Adam Shaywitz, MD PhD (1) · Celeste Decker, M.D. (1) · Eric He, MD (1) · Tippi MacKenzie, MD (1) · Tristan Zhang, MD (1)
PMID 29526614, PMID 15324318, PMID 19948615, PMID 21637107, PMID 23510062, PMID 26331768, PMID 27955919, PMID 28374935, PMID 28535791, PMID 28657663, PMID 29159458, PMID 29248359, PMID 30337566, PMID 32102934, PMID 37955580, PMID 39891377, PMID 9918480