Clinical trials
Rare Diseases trials with no applicable phase
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
144 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 17 |
| 2001 | 1 |
| 2009 | 1 |
| 2012 | 1 |
| 2014 | 1 |
| 2016 | 1 |
| 2017 | 2 |
| 2018 | 2 |
| 2019 | 4 |
| 2020 | 2 |
| 2021 | 5 |
| 2022 | 1 |
| 2023 | 4 |
| 2024 | 7 |
| 2025 | 6 |
| 2026 | 5 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 104 |
| Recruiting | 15 |
| Unknown status | 13 |
| Terminated | 6 |
| Not yet recruiting | 3 |
| Withdrawn | 1 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Center for Research Resources (NCRR) | 25 |
| FDA Office of Orphan Products Development | 13 |
| University Hospital Tuebingen | 7 |
| Fairview University Medical Center | 6 |
| Icahn School of Medicine at Mount Sinai | 5 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 4 |
| National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | 4 |
| Case Western Reserve University | 3 |
| Children's Hospital Medical Center, Cincinnati | 3 |
| Children's National Research Institute | 3 |
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 | 144 |
| Mental Disorders | 25 |
| Neurologic Manifestations | 25 |
| Genetic Diseases, Inborn | 22 |
| Respiratory Tract Diseases | 14 |
| Communicable Diseases | 13 |
| Immune System Diseases | 13 |
| Endocrine System Diseases | 11 |
| Hematologic Diseases | 11 |
| Metabolism, Inborn Errors | 10 |
Trials
Showing 76 to 100 of 144. Each trial links to its page on Pharma Observatory.
44 more trials match. See them 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.