Clinical trials
Phase 2 Metabolism, Inborn Errors trials
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 |
|---|---|
| 2004 | 1 |
| 2005 | 1 |
| 2006 | 2 |
| 2007 | 1 |
| 2008 | 1 |
| 2009 | 1 |
| 2010 | 1 |
| 2011 | 1 |
| 2014 | 1 |
| 2015 | 1 |
| 2017 | 1 |
| 2018 | 1 |
| 2021 | 1 |
| 2024 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 18 |
| Terminated | 3 |
| Unknown status | 2 |
| Withdrawn | 2 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Center for Research Resources (NCRR) | 7 |
| Masonic Cancer Center, University of Minnesota | 3 |
| Kastle Therapeutics, LLC | 2 |
| Akcea Therapeutics | 1 |
| Alexion Pharmaceuticals, Inc. | 1 |
| Alnylam Pharmaceuticals | 1 |
| Children's Hospital Medical Center, Cincinnati | 1 |
| Children's Hospital of Philadelphia | 1 |
| Fred Hutchinson Cancer Center | 1 |
| HemoShear 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 |
|---|---|
| Metabolism, Inborn Errors | 25 |
| Rare Diseases | 10 |
| Metabolic Diseases | 8 |
| Genetic Diseases, Inborn | 6 |
| Lipid Metabolism Disorders | 4 |
| Lipid Metabolism, Inborn Errors | 4 |
| Sphingolipidoses | 4 |
| Bone Marrow Failure Disorders | 3 |
| Dyslipidemias | 3 |
| Hyperlipidemias | 3 |
Trials
Showing 51 to 25 of 25. 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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