Clinical trials
Metabolism, Inborn Errors trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
85 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 3 |
| 2004 | 3 |
| 2005 | 2 |
| 2006 | 3 |
| 2007 | 3 |
| 2008 | 5 |
| 2009 | 5 |
| 2011 | 2 |
| 2012 | 1 |
| 2013 | 3 |
| 2014 | 2 |
| 2015 | 3 |
| 2016 | 2 |
| 2017 | 1 |
| 2018 | 2 |
| 2019 | 2 |
| 2020 | 1 |
| 2021 | 7 |
| 2022 | 4 |
| 2023 | 2 |
| 2024 | 2 |
| 2025 | 2 |
| 2026 | 2 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 47 |
| Recruiting | 12 |
| Terminated | 11 |
| Unknown status | 7 |
| Active, not recruiting | 3 |
| Withdrawn | 2 |
| No longer available | 1 |
| Not yet recruiting | 1 |
| Approved for marketing | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Center for Research Resources (NCRR) | 14 |
| ModernaTX, Inc. | 5 |
| National Human Genome Research Institute (NHGRI) | 5 |
| Kastle Therapeutics, LLC | 3 |
| Masonic Cancer Center, University of Minnesota | 3 |
| Regeneron Pharmaceuticals | 3 |
| Alexion Pharmaceuticals, Inc. | 2 |
| Children's Hospital of Philadelphia | 2 |
| Duke University | 2 |
| Fred Hutchinson Cancer Center | 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 |
|---|---|
| Metabolism, Inborn Errors | 85 |
| Genetic Diseases, Inborn | 34 |
| Metabolic Diseases | 30 |
| Rare Diseases | 19 |
| Lysosomal Storage Diseases | 15 |
| Sphingolipidoses | 11 |
| Lipid Metabolism Disorders | 9 |
| Mucopolysaccharidosis I | 9 |
| Brain Diseases, Metabolic, Inborn | 8 |
| Gaucher Disease | 8 |
Trials
Showing 1 to 25 of 85. 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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