Clinical trials
Completed Genetic Diseases, Inborn trials in Canada
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
36 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2006 | 1 |
| 2007 | 2 |
| 2008 | 1 |
| 2009 | 4 |
| 2010 | 1 |
| 2011 | 1 |
| 2012 | 1 |
| 2013 | 1 |
| 2014 | 1 |
| 2015 | 2 |
| 2017 | 5 |
| 2018 | 6 |
| 2019 | 4 |
| 2020 | 1 |
| 2022 | 2 |
| 2023 | 2 |
| 2024 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 36 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Vertex Pharmaceuticals Incorporated | 5 |
| QED Therapeutics, a BridgeBio company | 3 |
| Kastle Therapeutics, LLC | 2 |
| Novartis Pharmaceuticals | 2 |
| PTC Therapeutics | 2 |
| Pfizer | 2 |
| Regeneron Pharmaceuticals | 2 |
| Spark Therapeutics, Inc. | 2 |
| University of Washington, the Collaborative Health Studies Coordinating Center | 2 |
| Akcea 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 |
|---|---|
| Genetic Diseases, Inborn | 36 |
| Infant, Newborn, Diseases | 8 |
| Metabolic Diseases | 8 |
| Musculoskeletal Diseases | 8 |
| Cystic Fibrosis | 6 |
| Hematologic Diseases | 6 |
| Lipid Metabolism Disorders | 6 |
| Lung Diseases | 6 |
| Metabolism, Inborn Errors | 6 |
| Pancreatic Diseases | 6 |
Trials
Showing 1 to 25 of 36. 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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