Clinical trials
Observational Genetic Diseases, Inborn trials in Australia
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
6 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2019 | 2 |
| 2023 | 2 |
| 2024 | 2 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Recruiting | 3 |
| Completed | 2 |
| Enrolling by invitation | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| QED Therapeutics, a BridgeBio company | 2 |
| BioMarin Pharmaceutical | 1 |
| Friedreich's Ataxia Research Alliance | 1 |
| Neuroscience Research Australia | 1 |
| Spark Therapeutics, Inc. | 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 | 6 |
| Blood Coagulation Disorders | 2 |
| Blood Coagulation Disorders, Inherited | 2 |
| Bone Diseases | 2 |
| Coagulation Protein Disorders | 2 |
| Dwarfism | 2 |
| Hematologic Diseases | 2 |
| Hemophilia A | 2 |
| Hemorrhagic Disorders | 2 |
| Mucopolysaccharidosis IV | 2 |
Trials
Showing 1 to 6 of 6. 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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