Clinical trials
Phase 3 Genetic Diseases, Inborn trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
65 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 1 |
| 2004 | 1 |
| 2005 | 1 |
| 2007 | 1 |
| 2008 | 2 |
| 2009 | 6 |
| 2012 | 1 |
| 2013 | 3 |
| 2014 | 2 |
| 2016 | 3 |
| 2017 | 6 |
| 2018 | 7 |
| 2019 | 1 |
| 2020 | 1 |
| 2021 | 3 |
| 2022 | 5 |
| 2023 | 3 |
| 2024 | 3 |
| 2025 | 1 |
| 2026 | 4 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 42 |
| Active, not recruiting | 9 |
| Recruiting | 7 |
| Terminated | 3 |
| Withdrawn | 3 |
| Enrolling by invitation | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| BioMarin Pharmaceutical | 7 |
| Novo Nordisk A/S | 6 |
| Regeneron Pharmaceuticals | 6 |
| Vertex Pharmaceuticals Incorporated | 5 |
| GenSight Biologics | 3 |
| PTC Therapeutics | 3 |
| FDA Office of Orphan Products Development | 2 |
| Genzyme, a Sanofi Company | 2 |
| Kastle Therapeutics, LLC | 2 |
| Merz North America, Inc. | 2 |
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 | 65 |
| Hematologic Diseases | 13 |
| Musculoskeletal Diseases | 13 |
| Nervous System Diseases | 13 |
| Genetic Diseases, X-Linked | 11 |
| Metabolic Diseases | 10 |
| Metabolism, Inborn Errors | 9 |
| Muscular Dystrophies | 8 |
| Muscular Dystrophy, Duchenne | 8 |
| Neuromuscular Diseases | 8 |
Trials
Showing 26 to 50 of 65. 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.
Go deeper on Pharma Observatory
Pharma Observatory connects clinical trials, sponsors, sites and investigators in one searchable platform.