Clinical trials
Mucopolysaccharidosis II trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
51 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2003 | 1 |
| 2004 | 1 |
| 2005 | 2 |
| 2008 | 4 |
| 2009 | 5 |
| 2010 | 1 |
| 2011 | 2 |
| 2012 | 1 |
| 2013 | 1 |
| 2014 | 6 |
| 2015 | 4 |
| 2017 | 3 |
| 2018 | 2 |
| 2019 | 1 |
| 2020 | 2 |
| 2021 | 3 |
| 2022 | 6 |
| 2023 | 3 |
| 2025 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 24 |
| Active, not recruiting | 9 |
| Terminated | 8 |
| Enrolling by invitation | 3 |
| Recruiting | 3 |
| Withdrawn | 3 |
| Available | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Shire | 13 |
| REGENXBIO Inc. | 6 |
| Denali Therapeutics Inc. | 4 |
| Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | 3 |
| Takeda | 3 |
| Duke University | 2 |
| JCR Pharmaceuticals Co., Ltd. | 2 |
| Joanne Kurtzberg, MD | 2 |
| Masonic Cancer Center, University of Minnesota | 2 |
| Sangamo Therapeutics | 2 |
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 II | 51 |
| Sudden Infant Death | 27 |
| Mucopolysaccharidosis I | 17 |
| Mucopolysaccharidoses | 13 |
| Mucopolysaccharidosis VI | 13 |
| Lysosomal Storage Diseases | 11 |
| Leukodystrophy, Globoid Cell | 8 |
| Leukodystrophy, Metachromatic | 8 |
| Mucopolysaccharidosis III | 6 |
| Mucopolysaccharidosis VII | 6 |
Trials
Showing 1 to 25 of 51. 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.
No changes to these trials in the last 7 days.
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