Clinical trials
Metabolism, Inborn Errors trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
151 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 4 |
| 2004 | 3 |
| 2005 | 2 |
| 2006 | 6 |
| 2007 | 3 |
| 2008 | 5 |
| 2009 | 6 |
| 2010 | 1 |
| 2011 | 2 |
| 2012 | 1 |
| 2013 | 4 |
| 2014 | 3 |
| 2015 | 4 |
| 2016 | 3 |
| 2017 | 5 |
| 2018 | 7 |
| 2019 | 8 |
| 2020 | 1 |
| 2021 | 12 |
| 2022 | 6 |
| 2023 | 3 |
| 2024 | 8 |
| 2025 | 3 |
| 2026 | 6 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 87 |
| Recruiting | 18 |
| Terminated | 15 |
| Unknown status | 12 |
| Withdrawn | 7 |
| Active, not recruiting | 4 |
| Not yet recruiting | 4 |
| No longer available | 2 |
| Approved for marketing | 2 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Center for Research Resources (NCRR) | 27 |
| ModernaTX, Inc. | 6 |
| National Human Genome Research Institute (NHGRI) | 5 |
| Kastle Therapeutics, LLC | 4 |
| Regeneron Pharmaceuticals | 4 |
| Alexion Pharmaceuticals, Inc. | 3 |
| Alnylam Pharmaceuticals | 3 |
| CENTOGENE GmbH Rostock | 3 |
| Children's Hospital of Philadelphia | 3 |
| Masonic Cancer Center, University of Minnesota | 3 |
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 | 151 |
| Genetic Diseases, Inborn | 48 |
| Metabolic Diseases | 43 |
| Rare Diseases | 42 |
| Lysosomal Storage Diseases | 16 |
| Sphingolipidoses | 13 |
| Lipid Metabolism Disorders | 12 |
| Lipid Metabolism, Inborn Errors | 12 |
| Amino Acid Metabolism, Inborn Errors | 11 |
| Brain Diseases, Metabolic, Inborn | 10 |
Trials
Showing 26 to 50 of 151. Each trial links to its page on Pharma Observatory.
51 more trials match. See them 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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