5 completed, 3 recruiting, 2 terminated, 1 active not recruiting. 4 Phase 2, 2 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Terminated 2, Active not recruiting 1, Completed 1 |
| Phase 1 | 2 | Active not recruiting 1, Recruiting 1 |
| Phase 3 | 1 | Completed 1 |
Follow MPS IVA: new trials by email Literature Review of MPS IVA trials Research Report on MPS IVA
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 |
|---|---|---|---|---|---|---|
| Cardiac Structure and Function in MPS MPS I, MPS IV A | Recruiting | Children's Hospital of Orange County | 240 | 2025-09-01 | 2026-10-21 | |
| 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 |
| Non-invasive Functional Assessment and Pathogenesis of Morquio A Imaging, gait analysis, growth, joint test, hearing test, questionnaire, etc. - Mucopolysaccharidosis IV Type A, Morquio A Syndrome | Recruiting | Nemours Children's Clinic | 60 | 2021-05-01 | 2026-05-13 | |
| 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 | |
| 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 | |
| Gait Analysis in MPS IVA MPS IVA, Morquio Syndrome | Completed | Oregon Health and Science University | 10 | 2013-08-01 | 2019-10-17 | |
| 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 |
| BMN 110 US Expanded Access Program BMN 110 - Mucopolysaccharidosis IVA, Morquio A Syndrome | Approved for marketing | BioMarin Pharmaceutical | 2014-04-02 |
Mucopolysaccharidosis IV (12) · Lysosomal Storage Diseases (5) · Mucopolysaccharidosis VI (3) · Dwarfism (1) · Gaucher Disease (1) · Mucopolysaccharidoses (1) · Mucopolysaccharidosis VII (1) · Sudden Infant Death (1) · Wolman Disease (1)
Carbohydrate Metabolism, Inborn Errors (12) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (12) · Connective Tissue Diseases (12) · Genetic Diseases, Inborn (12) · Metabolic Diseases (12) · Metabolism, Inborn Errors (12) · Mucinoses (12) · Nutritional and Metabolic Diseases (12) · Skin and Connective Tissue Diseases (12) · Mucopolysaccharidoses (11) · Lysosomal Storage Diseases (7) · Bone Diseases (1) · Bone Diseases, Developmental (1) · Brain Diseases (1) · Brain Diseases, Metabolic (1)
GALNS protein, human (7) · Chondroitinases and Chondroitin Lyases (4) · Enzyme Replacement Therapy (4) · Galns protein, mouse (4) · Sulfatases (3) · Diagnostic Imaging (1) · GAA protein, human (1) · Gait Analysis (1) · Growth (1) · Hearing Tests (1) · Iduronidase (1) · Sebelipase alfa (1) · galsulfase (1) · idursulfase (1) · vestronidase alfa (1)
BioMarin Pharmaceutical (6) · University of California, San Francisco (2) · Children's Hospital of Orange County (1) · Greenwood Genetic Center (1) · Nemours Children's Clinic (1) · Oregon Health and Science University (1)
United States (12) · United Kingdom (5) · Canada (3) · Germany (3) · Italy (3) · Taiwan (3) · Denmark (2) · France (2) · Netherlands (2) · Portugal (2) · Puerto Rico (2) · Argentina (1) · Australia (1) · Austria (1) · Belgium (1)
Debra Lounsbury (2) · Adam Shaywitz, MD PhD (1) · Celeste Decker, M.D. (1) · Ellen B Fung, PhD (1) · Richard Curtis Rogers, MD (1) · Shunji Tomatsu, MD PhD (1) · Tippi MacKenzie, MD (1)
PMID 15324318, PMID 18412124, 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
R01HD102545 (1), R01HL173371 (1)