Clinical trials
Rare Diseases trials with unknown status
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
74 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 5 |
| 2001 | 2 |
| 2010 | 1 |
| 2014 | 1 |
| 2015 | 2 |
| 2016 | 1 |
| 2017 | 1 |
| 2018 | 3 |
| 2019 | 3 |
| 2020 | 3 |
| 2021 | 8 |
| 2022 | 4 |
| 2023 | 3 |
| 2024 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Unknown status | 74 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Center for Research Resources (NCRR) | 28 |
| National Institute of Neurological Disorders and Stroke (NINDS) | 5 |
| University Hospital Tuebingen | 5 |
| Fairview University Medical Center | 3 |
| Assistance Publique - Hôpitaux de Paris | 2 |
| Northwestern Memorial Hospital | 2 |
| Wuerzburg University Hospital | 2 |
| Bicetre Hospital | 1 |
| Centre Hospitalier Universitaire de Liege | 1 |
| Cystinosis Research Foundation | 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 | 74 |
| Genetic Diseases, Inborn | 12 |
| Mental Disorders | 9 |
| Neurologic Manifestations | 9 |
| Respiratory Tract Diseases | 7 |
| Communicable Diseases | 6 |
| Connective Tissue Diseases | 6 |
| Endocrine System Diseases | 6 |
| Hematologic Diseases | 6 |
| Immune System Diseases | 6 |
Trials
Showing 1 to 25 of 74. 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.