Clinical trials
Phase 1/2 Rare Diseases trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
12 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2009 | 1 |
| 2016 | 1 |
| 2018 | 1 |
| 2021 | 2 |
| 2022 | 1 |
| 2024 | 1 |
| 2025 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 6 |
| Recruiting | 3 |
| Active, not recruiting | 1 |
| Suspended | 1 |
| Terminated | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Gemma Biotherapeutics | 2 |
| Amgen | 1 |
| Aspa Therapeutics | 1 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 1 |
| Fred Hutchinson Cancer Center | 1 |
| Hoffmann-La Roche | 1 |
| Klus Pharma Inc. | 1 |
| Kyowa Kirin Co., Ltd. | 1 |
| National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | 1 |
| REGENXBIO 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 |
|---|---|
| Rare Diseases | 12 |
| Genetic Diseases, Inborn | 3 |
| Anemia, Sickle Cell | 2 |
| Hematologic Diseases | 2 |
| Lysosomal Storage Diseases | 2 |
| Metabolic Diseases | 2 |
| Atherosclerosis | 1 |
| Bile Duct Neoplasms | 1 |
| Bone Diseases | 1 |
| Breast Neoplasms | 1 |
Trials
Showing 1 to 12 of 12. 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.