Clinical Trial Readiness for Rare Diseases, Disorders and Syndromes

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

Funded Research

Principal Investigators Institutions Project Titles
Arnold, Steven E. Massachusetts General Hospital Biomarkers to Enable ASO Prevention Trials in Genetic Prion Disease Carriers
Byiers, Breanne J. University of Minnesota Evaluating the Psychometric Properties of Three Clinical Trial Outcome Measures for Rett Syndrome
Caldovic, Ljubica; Ah Mew, Nicholas Children’s Research Institute Systemic Biomarkers of Brain Injury from Hyperammonemia
Lynch, David Robinson Children’s Hospital of Philadelphia Frataxin Measurement for Clinical Trial Readiness in Friedreich Ataxia
Muir, Amanda Brooke; Menard-Katcher, Calies D. Children’s Hospital of Philadelphia Distensibility and Remodeling as Markers of Therapeutic Response in Eosinophilic Esophagitis
Nihalani, Deepak Medical University of South Carolina Clinical Assessment of a Novel Non-Invasive Array for Diagnosing Recurrent Focal and Segmental Glomerulosclerosis
Shy, Michael E. University of Iowa Clinical Trial Readiness for Rare Diseases, Disorders and Syndromes

Related Information

Read more about the goals of the CTR grants program.

Learn about related programs: