Clinical trials
Phase 1/2 Mucopolysaccharidosis I trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
16 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2008 | 2 |
| 2011 | 1 |
| 2015 | 2 |
| 2016 | 1 |
| 2017 | 3 |
| 2018 | 3 |
| 2019 | 1 |
| 2020 | 1 |
| 2021 | 2 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 9 |
| Active, not recruiting | 2 |
| Terminated | 2 |
| Withdrawn | 2 |
| Suspended | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| ArmaGen, Inc | 2 |
| JCR Pharmaceuticals Co., Ltd. | 2 |
| Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | 2 |
| Children's Hospital Los Angeles | 1 |
| Genzyme, a Sanofi Company | 1 |
| Institut de Recherches Internationales Servier | 1 |
| Novartis Pharmaceuticals | 1 |
| Orchard Therapeutics | 1 |
| REGENXBIO Inc. | 1 |
| Sangamo Therapeutics | 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 I | 16 |
| Mucopolysaccharidosis II | 3 |
| Adrenoleukodystrophy | 2 |
| Anemia, Sickle Cell | 2 |
| Granulomatous Disease, Chronic | 2 |
| Leukodystrophy, Metachromatic | 2 |
| Lymphoproliferative Disorders | 2 |
| Mucopolysaccharidoses | 2 |
| Mucopolysaccharidosis VI | 2 |
| Niemann-Pick Diseases | 2 |
Trials
Showing 26 to 16 of 16. Each trial links to its page on Pharma Observatory.
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Recently changed trials
Changes observed in the last 7 days, newest first, site-count changes left out. Source: ClinicalTrials.gov.
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