Clinical trials
Phase 1 Metabolism, Inborn Errors trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
15 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2005 | 1 |
| 2008 | 1 |
| 2009 | 1 |
| 2013 | 2 |
| 2015 | 1 |
| 2022 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 9 |
| Terminated | 2 |
| Withdrawn | 2 |
| Recruiting | 1 |
| Unknown status | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Center for Research Resources (NCRR) | 5 |
| Joanne Kurtzberg, MD | 2 |
| Children's Hospital of Philadelphia | 1 |
| Duke University | 1 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 1 |
| National Institute of Mental Health (NIMH) | 1 |
| Neurocrine Biosciences | 1 |
| New York Medical College | 1 |
| Synlogic | 1 |
| University of Pennsylvania | 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 | 15 |
| Rare Diseases | 7 |
| Gaucher Disease | 3 |
| Genetic Diseases, Inborn | 3 |
| Mucopolysaccharidosis I | 3 |
| Urea Cycle Disorders, Inborn | 3 |
| Anemia, Aplastic | 2 |
| Anemia, Diamond-Blackfan | 2 |
| Immunologic Deficiency Syndromes | 2 |
| Lysosomal Storage Diseases | 2 |
Trials
Showing 1 to 15 of 15. 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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