Clinical trials
Expanded access Metabolic Diseases trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
5 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
No trials to chart.
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Approved for marketing | 2 |
| No longer available | 2 |
| Available | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Akcea Therapeutics | 1 |
| Alnylam Pharmaceuticals | 1 |
| Ionis Pharmaceuticals, Inc. | 1 |
| Joanne Kurtzberg, MD | 1 |
| Joyce Fox | 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 |
|---|---|
| Metabolic Diseases | 5 |
| Genetic Diseases, Inborn | 3 |
| Metabolism, Inborn Errors | 3 |
| Dyslipidemias | 2 |
| Hyperlipidemias | 2 |
| Hyperlipoproteinemia Type I | 2 |
| Hyperlipoproteinemias | 2 |
| Lipid Metabolism Disorders | 2 |
| Lipid Metabolism, Inborn Errors | 2 |
| Adrenoleukodystrophy | 1 |
Trials
Showing 76 to 5 of 5. 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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Go deeper on Pharma Observatory
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