Clinical trials
Genetic Diseases, Inborn trials in Brazil
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 |
|---|---|
| 2007 | 1 |
| 2008 | 1 |
| 2013 | 1 |
| 2014 | 2 |
| 2015 | 1 |
| 2017 | 2 |
| 2018 | 1 |
| 2020 | 2 |
| 2022 | 1 |
| 2023 | 2 |
| 2024 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 10 |
| Recruiting | 2 |
| Terminated | 2 |
| Enrolling by invitation | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| BioMarin Pharmaceutical | 4 |
| PTC Therapeutics | 3 |
| Kastle Therapeutics, LLC | 2 |
| Alexion Pharmaceuticals, Inc. | 1 |
| Azafaros B.V. | 1 |
| Friedreich's Ataxia Research Alliance | 1 |
| Intellia Therapeutics | 1 |
| Pfizer | 1 |
| Shire | 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 |
|---|---|
| Genetic Diseases, Inborn | 15 |
| Nervous System Diseases | 6 |
| Metabolic Diseases | 5 |
| Metabolism, Inborn Errors | 5 |
| Blood Coagulation Disorders | 4 |
| Blood Coagulation Disorders, Inherited | 4 |
| Coagulation Protein Disorders | 4 |
| Hematologic Diseases | 4 |
| Hemophilia A | 4 |
| Hemorrhagic Disorders | 4 |
Trials
Showing 76 to 15 of 15. 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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