Clinical trials
Mucopolysaccharidosis I trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
53 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 1 |
| 2001 | 1 |
| 2003 | 1 |
| 2004 | 2 |
| 2005 | 2 |
| 2006 | 1 |
| 2007 | 1 |
| 2008 | 7 |
| 2009 | 4 |
| 2010 | 1 |
| 2011 | 2 |
| 2012 | 2 |
| 2013 | 1 |
| 2014 | 5 |
| 2015 | 2 |
| 2017 | 2 |
| 2018 | 4 |
| 2019 | 2 |
| 2020 | 3 |
| 2021 | 1 |
| 2022 | 2 |
| 2023 | 3 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 26 |
| Terminated | 13 |
| Active, not recruiting | 5 |
| Recruiting | 4 |
| No longer available | 1 |
| Suspended | 1 |
| Enrolling by invitation | 1 |
| Unknown status | 1 |
| Withdrawn | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Masonic Cancer Center, University of Minnesota | 9 |
| Genzyme, a Sanofi Company | 4 |
| Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | 3 |
| Patricia I. Dickson, M.D. | 3 |
| University of Minnesota | 3 |
| JCR Pharmaceuticals Co., Ltd. | 2 |
| Magenta Therapeutics, Inc. | 2 |
| Paul Szabolcs | 2 |
| REGENXBIO Inc. | 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 I | 53 |
| Mucopolysaccharidosis II | 17 |
| Mucopolysaccharidosis VI | 16 |
| Mucopolysaccharidoses | 14 |
| Leukodystrophy, Globoid Cell | 13 |
| Leukodystrophy, Metachromatic | 13 |
| Metabolism, Inborn Errors | 9 |
| Adrenoleukodystrophy | 8 |
| Sudden Infant Death | 8 |
| Fucosidosis | 7 |
Trials
Showing 1 to 25 of 53. 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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