Clinical trials
Genetic Diseases, Inborn trials in the United States with unknown status
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
16 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 1 |
| 2013 | 1 |
| 2017 | 1 |
| 2019 | 3 |
| 2021 | 1 |
| 2023 | 2 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Unknown status | 16 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Center for Research Resources (NCRR) | 5 |
| Fairview University Medical Center | 2 |
| Boston Children's Hospital | 1 |
| Editas Medicine, Inc. | 1 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 1 |
| GenEmbryomics Pty. Ltd | 1 |
| Illumina, Inc. | 1 |
| M.D. Anderson Cancer Center | 1 |
| REGENXBIO Inc. | 1 |
| Rady Pediatric Genomics & Systems Medicine Institute | 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 | 16 |
| Rare Diseases | 9 |
| Respiratory Tract Diseases | 3 |
| Communicable Diseases | 2 |
| Cystic Fibrosis | 2 |
| Immune System Diseases | 2 |
| Primary Immunodeficiency Diseases | 2 |
| Abnormalities, Multiple | 1 |
| Adrenoleukodystrophy | 1 |
| Amyloidosis | 1 |
Trials
Showing 1 to 16 of 16. 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.