Clinical trials
Observational Rare Diseases trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
220 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 3 |
| 2001 | 3 |
| 2003 | 2 |
| 2004 | 1 |
| 2006 | 1 |
| 2007 | 1 |
| 2009 | 1 |
| 2010 | 3 |
| 2011 | 2 |
| 2013 | 2 |
| 2014 | 1 |
| 2015 | 5 |
| 2016 | 3 |
| 2017 | 4 |
| 2018 | 8 |
| 2019 | 11 |
| 2020 | 9 |
| 2021 | 19 |
| 2022 | 9 |
| 2023 | 16 |
| 2024 | 19 |
| 2025 | 19 |
| 2026 | 13 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 82 |
| Recruiting | 59 |
| Unknown status | 42 |
| Enrolling by invitation | 11 |
| Not yet recruiting | 10 |
| Terminated | 7 |
| Withdrawn | 5 |
| Active, not recruiting | 4 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Center for Research Resources (NCRR) | 57 |
| IRCCS Policlinico S. Donato | 10 |
| Assistance Publique - Hôpitaux de Paris | 8 |
| National Human Genome Research Institute (NHGRI) | 7 |
| Imagine Institute | 6 |
| University Hospital Tuebingen | 6 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 4 |
| Institut National de la Santé Et de la Recherche Médicale, France | 4 |
| Associazione Angela Serra per la ricerca sul cancro | 3 |
| AstraZeneca | 2 |
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 | 220 |
| Genetic Diseases, Inborn | 39 |
| Endocrine System Diseases | 18 |
| Neurologic Manifestations | 16 |
| Mental Disorders | 14 |
| Respiratory Tract Diseases | 12 |
| Metabolism, Inborn Errors | 9 |
| Undiagnosed Diseases | 9 |
| Marfan Syndrome | 7 |
| Connective Tissue Diseases | 6 |
Trials
Showing 51 to 75 of 220. Each trial links to its page on Pharma Observatory.
120 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.
Go deeper on Pharma Observatory
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