Clinical trials
Observational Metabolism, Inborn Errors trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
25 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 2 |
| 2004 | 1 |
| 2006 | 2 |
| 2007 | 1 |
| 2012 | 1 |
| 2014 | 1 |
| 2015 | 1 |
| 2016 | 1 |
| 2017 | 1 |
| 2018 | 1 |
| 2019 | 1 |
| 2020 | 1 |
| 2021 | 2 |
| 2022 | 1 |
| 2024 | 1 |
| 2025 | 1 |
| 2026 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 12 |
| Recruiting | 6 |
| Terminated | 3 |
| Unknown status | 3 |
| Active, not recruiting | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Center for Research Resources (NCRR) | 5 |
| National Human Genome Research Institute (NHGRI) | 5 |
| Alnylam Pharmaceuticals | 1 |
| Amicus Therapeutics | 1 |
| Celgene Corporation | 1 |
| Duke University | 1 |
| Fairview University Medical Center | 1 |
| HemoShear Therapeutics | 1 |
| JCR Pharmaceuticals Co., Ltd. | 1 |
| ModernaTX, Inc. | 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 |
|---|---|
| Metabolism, Inborn Errors | 25 |
| Genetic Diseases, Inborn | 11 |
| Metabolic Diseases | 10 |
| Lysosomal Storage Diseases | 6 |
| Brain Diseases, Metabolic, Inborn | 5 |
| Rare Diseases | 5 |
| Sphingolipidoses | 4 |
| Brain Diseases | 3 |
| Brain Diseases, Metabolic | 3 |
| Central Nervous System Diseases | 3 |
Trials
Showing 1 to 25 of 25. 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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