Clinical trials
Phase 1 Genetic Diseases, Inborn trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
36 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 2 |
| 2009 | 1 |
| 2011 | 3 |
| 2014 | 1 |
| 2015 | 1 |
| 2016 | 3 |
| 2017 | 2 |
| 2019 | 1 |
| 2020 | 2 |
| 2022 | 1 |
| 2023 | 4 |
| 2024 | 1 |
| 2026 | 2 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 26 |
| Terminated | 4 |
| Recruiting | 3 |
| Not yet recruiting | 1 |
| Unknown status | 1 |
| Withdrawn | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| AO GENERIUM | 3 |
| Eloxx Pharmaceuticals, Inc. | 3 |
| National Cancer Institute (NCI) | 3 |
| National Center for Research Resources (NCRR) | 2 |
| Siena Biotech S.p.A. | 2 |
| University of Alabama at Birmingham | 2 |
| AIRNA Corporation | 1 |
| Adjuvia Therapeutics, Inc. | 1 |
| AlgiPharma AS | 1 |
| Dicerna Pharmaceuticals, Inc., a Novo Nordisk company | 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 | 36 |
| Rare Diseases | 8 |
| Respiratory Tract Diseases | 8 |
| Cystic Fibrosis | 7 |
| Metabolic Diseases | 4 |
| Neoplasms | 4 |
| Nervous System Diseases | 4 |
| Central Nervous System Diseases | 3 |
| Gastrointestinal Neoplasms | 3 |
| Hemoglobinopathies | 3 |
Trials
Showing 26 to 36 of 36. 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.