Clinical trials
Mucopolysaccharidosis IV trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
56 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2001 | 1 |
| 2008 | 2 |
| 2009 | 2 |
| 2010 | 1 |
| 2011 | 4 |
| 2012 | 6 |
| 2013 | 3 |
| 2014 | 2 |
| 2016 | 1 |
| 2017 | 1 |
| 2018 | 2 |
| 2019 | 3 |
| 2020 | 3 |
| 2021 | 5 |
| 2022 | 2 |
| 2023 | 4 |
| 2024 | 1 |
| 2025 | 6 |
| 2026 | 4 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 29 |
| Recruiting | 11 |
| Terminated | 7 |
| Enrolling by invitation | 4 |
| Active, not recruiting | 2 |
| Unknown status | 1 |
| Approved for marketing | 1 |
| Not yet recruiting | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| BioMarin Pharmaceutical | 8 |
| QED Therapeutics, a BridgeBio company | 8 |
| University of California, San Francisco | 3 |
| Emory University | 2 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 2 |
| Nadia Ali, PhD | 2 |
| Pfizer | 2 |
| Supplement Formulators, Inc. | 2 |
| Acuity Polymers, Inc. | 1 |
| Albert Einstein College of Medicine | 1 |
Top conditions
MeSH conditions the trials were registered with, not their broader categories. A condition with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Mucopolysaccharidosis IV | 56 |
| Dwarfism | 11 |
| Genetic Diseases, Inborn | 10 |
| Osteochondrodysplasias | 10 |
| Bone Diseases | 9 |
| Lysosomal Storage Diseases | 9 |
| Mucopolysaccharidosis VI | 9 |
| Musculoskeletal Diseases | 9 |
| Achondroplasia | 7 |
| Diarrhea | 7 |
Trials
Showing 26 to 50 of 56. Each trial links to its page on Pharma Observatory.
Recently changed trials
Changes observed in the last 7 days, newest first, site-count changes left out. Source: ClinicalTrials.gov.
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