Clinical trials
Phase 1/2 Metabolism, Inborn Errors trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
13 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2015 | 1 |
| 2019 | 1 |
| 2021 | 4 |
| 2022 | 2 |
| 2023 | 1 |
| 2025 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Recruiting | 4 |
| Completed | 3 |
| Terminated | 3 |
| Active, not recruiting | 1 |
| Not yet recruiting | 1 |
| Withdrawn | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| ModernaTX, Inc. | 5 |
| Alexion Pharmaceuticals, Inc. | 1 |
| Children's Hospital of Philadelphia | 1 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 1 |
| Forge Biologics, Inc | 1 |
| LogicBio Therapeutics, Inc | 1 |
| National Center for Research Resources (NCRR) | 1 |
| NeuroGT | 1 |
| SwanBio Therapeutics, 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 | 13 |
| Genetic Diseases, Inborn | 7 |
| Metabolic Diseases | 6 |
| Lysosomal Storage Diseases | 3 |
| Mucopolysaccharidoses | 3 |
| Acid-Base Imbalance | 2 |
| Acidosis | 2 |
| Amino Acid Metabolism, Inborn Errors | 2 |
| Brain Diseases, Metabolic | 2 |
| Brain Diseases, Metabolic, Inborn | 2 |
Trials
Showing 1 to 13 of 13. 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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