Clinical trials are critical to developing and evaluating new treatments for rare diseases. Scientists, however, often do not have enough information about the symptoms and biology of rare diseases to design clinical trials. NCATS, working with the Eunice Kennedy Shriver National Institute of Child Health and Human Development, created the Clinical Trial Readiness for Rare Diseases, Disorders and Syndromes grants to address some of the obstacles scientists face. These obstacles include, among other issues, gaps in our understanding of a rare disease’s natural history and a lack of suitable biomarkers or clinical outcome measures.
Through these grants, NCATS seeks to facilitate rare disease research by enabling efficient and effective movement of candidate therapies or diagnostics toward clinical trials and to increase their likelihood of success. These grants are modeled after a grant program at the National Institute of Neurological Disorders and Stroke.
Contact: Alice Chen Grady, M.D.
Current Funding Opportunities
Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R03 Clinical Trial Not Allowed)
PAR-23-160 · Posted Date: 04/13/2023
Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)
PAR-23-159 · Posted Date: 04/13/2023
Funded Research
Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R03 Clinical Trial Not Allowed)
| Principal Investigator(s) | Year Awarded | Institution | Title |
|---|---|---|---|
| Weese-Mayer, Debra Ellyn | 2022 | Lurie Children's Hospital of Chicago | PHOX2B Congenital Central Hypoventilation Syndrome (CCHS) Physiologic Signatures in Readiness for Future Clinical Trials |
| Erkan, Elif | 2021 | Cincinnati Children's Hospital Medical Center | Urinary Lipidomic Profile in FSGS: A Novel Biomarker |
| Wheeler, Anne Caroline; Sadhwani, Anjali | 2021 | Research Triangle Institute | Determining Minimal Clinically Important Differences for Neurodevelopmental Outcome Measures in Angelman Syndrome |
| Dwyer, Andrew Alois | 2020 | Boston College | Identifying Predictors of Reversible Congenital Hypogonadotropic Hypogonadism |
| Nihalani, Deepak | 2019 | Medical University of South Carolina | Clinical Assessment of a Novel Non-Invasive Array for Diagnosing Recurrent Focal and Segmental Glomerulosclerosis |
Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)
Related Information
Read more about the goals of the CTR grants program.
Learn about related programs:
- Rare Diseases Clinical Research Network – The RDCRN supports consortia of medical research centers that work together to investigate groups of related rare diseases.
- Therapeutics for Rare and Neglected Diseases – The TRND program supports preclinical development of therapeutic candidates intended to treat rare or neglected disorders.