Clinical trials
Terminated Phase 2 Genetic Diseases, Inborn trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
11 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2006 | 1 |
| 2007 | 1 |
| 2009 | 2 |
| 2011 | 1 |
| 2019 | 2 |
| 2021 | 3 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Terminated | 11 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Children's Hospital Los Angeles | 1 |
| Eloxx Pharmaceuticals, Inc. | 1 |
| Fred Hutchinson Cancer Center | 1 |
| HemoShear Therapeutics | 1 |
| Intergroupe Francophone du Myelome | 1 |
| Laboratoires Thea | 1 |
| National Cancer Institute (NCI) | 1 |
| Novartis Pharmaceuticals | 1 |
| Pfizer | 1 |
| The Hospital for Sick Children | 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 | 11 |
| Metabolic Diseases | 3 |
| Neoplasms | 2 |
| Achondroplasia | 1 |
| Anemia, Aplastic | 1 |
| Anemia, Diamond-Blackfan | 1 |
| Bone Diseases | 1 |
| Cystic Fibrosis | 1 |
| Cystinosis | 1 |
| Digestive System Diseases | 1 |
Trials
Showing 1 to 11 of 11. 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
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