Basket Clinical Trials of Drugs Targeting Shared Molecular Etiologies in Multiple Rare Diseases (U44 Clinical Trial Required)

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NCATS seeks to facilitate rare diseases research by enabling efficient and effective movement toward clinical trials in multiple rare diseases. The purpose of this NOFO is to provide support for basket clinical trials of drugs targeting shared molecular etiologies in more than one rare disease, and in the process to identify and overcome challenges in adapting the oncology basket trial model to rare diseases. Projects proposed for this NOFO will require individuals with expertise in carrying out clinical trials in rare diseases. Applicants are expected to collaborate with clinical investigators at academic institutions. Projects involving clinical investigators who are part of the Rare Disease Clinical Research Network (RDCRN) and which focus on diseases under study in the RDCRN are strongly encouraged. See https://www.rarediseasesnetwork.org/ for additional information.

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