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| Withdrawn | 5 |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 1 to 25 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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