Clinical trials
Dwarfism trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
55 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2001 | 2 |
| 2004 | 2 |
| 2005 | 6 |
| 2006 | 1 |
| 2007 | 1 |
| 2008 | 2 |
| 2009 | 1 |
| 2010 | 2 |
| 2011 | 1 |
| 2012 | 2 |
| 2013 | 1 |
| 2014 | 2 |
| 2015 | 1 |
| 2016 | 2 |
| 2017 | 1 |
| 2018 | 3 |
| 2019 | 4 |
| 2020 | 4 |
| 2021 | 2 |
| 2022 | 1 |
| 2023 | 2 |
| 2024 | 4 |
| 2025 | 3 |
| 2026 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 31 |
| Terminated | 7 |
| Recruiting | 6 |
| Active, not recruiting | 6 |
| Enrolling by invitation | 3 |
| Unknown status | 1 |
| Withdrawn | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| BioMarin Pharmaceutical | 9 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 7 |
| QED Therapeutics, a BridgeBio company | 7 |
| Nemours Children's Clinic | 5 |
| Ascendis Pharma A/S | 3 |
| Children's Hospital Medical Center, Cincinnati | 2 |
| Massachusetts General Hospital | 2 |
| National Human Genome Research Institute (NHGRI) | 2 |
| Nationwide Children's Hospital | 2 |
| Andrew Dauber | 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 |
|---|---|
| Dwarfism | 55 |
| Achondroplasia | 17 |
| Bone Diseases | 16 |
| Musculoskeletal Diseases | 14 |
| Genetic Diseases, Inborn | 12 |
| Osteochondrodysplasias | 12 |
| Mucopolysaccharidosis IV | 11 |
| Dwarfism, Pituitary | 9 |
| Bone Diseases, Developmental | 8 |
| Failure to Thrive | 4 |
Trials
Showing 1 to 25 of 55. 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.
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