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| Withdrawn | 1 |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 1 to 25 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.