Clinical trials
Phase 1 Genetic Diseases, Inborn trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
15 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 1 |
| 2011 | 2 |
| 2014 | 1 |
| 2015 | 1 |
| 2019 | 1 |
| 2022 | 1 |
| 2023 | 2 |
| 2026 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 12 |
| Not yet recruiting | 1 |
| Terminated | 1 |
| Withdrawn | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Cancer Institute (NCI) | 3 |
| Adjuvia Therapeutics, Inc. | 1 |
| Edgewise Therapeutics, Inc. | 1 |
| Hillhurst Biopharmaceuticals, Inc. | 1 |
| National Institute of Mental Health (NIMH) | 1 |
| National Institutes of Health Clinical Center (CC) | 1 |
| Neurocrine Biosciences | 1 |
| New York Blood Center | 1 |
| Novo Nordisk A/S | 1 |
| PepGen 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 | 15 |
| Neoplasms | 4 |
| Gastrointestinal Neoplasms | 3 |
| Nervous System Diseases | 3 |
| Neuroectodermal Tumors, Primitive, Peripheral | 3 |
| Neuromuscular Diseases | 3 |
| Adrenal Gland Diseases | 2 |
| Adrenal Hyperplasia, Congenital | 2 |
| Adrenocortical Hyperfunction | 2 |
| Bone Neoplasms | 2 |
Trials
Showing 1 to 15 of 15. 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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