Clinical trials
Genetic Diseases, Inborn trials in Australia
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
51 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2009 | 2 |
| 2010 | 1 |
| 2011 | 3 |
| 2013 | 1 |
| 2014 | 2 |
| 2015 | 1 |
| 2016 | 2 |
| 2017 | 5 |
| 2018 | 5 |
| 2019 | 4 |
| 2020 | 2 |
| 2021 | 4 |
| 2022 | 1 |
| 2023 | 4 |
| 2024 | 6 |
| 2025 | 3 |
| 2026 | 5 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 23 |
| Recruiting | 9 |
| Terminated | 7 |
| Active, not recruiting | 6 |
| Enrolling by invitation | 5 |
| Unknown status | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| BioMarin Pharmaceutical | 8 |
| QED Therapeutics, a BridgeBio company | 7 |
| Novartis Pharmaceuticals | 4 |
| Vertex Pharmaceuticals Incorporated | 4 |
| PTC Therapeutics | 3 |
| Genzyme, a Sanofi Company | 2 |
| Murdoch Childrens Research Institute | 2 |
| Pfizer | 2 |
| Quince Therapeutics S.p.A. | 2 |
| Spark Therapeutics, 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 | 51 |
| Musculoskeletal Diseases | 20 |
| Bone Diseases | 13 |
| Osteochondrodysplasias | 13 |
| Nervous System Diseases | 12 |
| Dwarfism | 11 |
| Genetic Diseases, X-Linked | 10 |
| Achondroplasia | 9 |
| Mucopolysaccharidosis IV | 8 |
| Blood Coagulation Disorders | 7 |
Trials
Showing 51 to 51 of 51. 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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