Accelerating Clinical Trials Evaluating Disease-Modifying Therapies for T1D

Breakthrough T1D (formerly JDRF) is committed to accelerating the development of curative
therapies from discovery research through clinical development and approval for type 1 diabetes
(T1D). To this end, they invite applications that propose clinical trials to test small molecules,
biologics, and combination therapies to delay, halt, or reverse the progression of T1D.

Objectives

• Breakthrough T1D is requesting applications that propose Proof of Concept (POC) human subject research trials to provide the data necessary to enable further clinical development, and Phase 1 or 2 trials to determine safety and efficacy. Trials should be powered for the stage of clinical development proposed and include the appropriate and justified endpoints. Phase 3 trials may be considered, however, approval must be obtained from the scientific contact below prior to submission of an LOI so that the budgetary and timeline needs of the trial can be assessed.
• Examples of research proposals appropriate for this RFA include, but are not limited to:
  • Trials evaluating novel small molecule drugs or biologics in people with T1D.
  • Clinical trials evaluating the repurposing of small molecule drugs or biologics with demonstrated efficacy in non-T1D populations that have clear rationale or preclinical data for assessment in T1D.
  • Inclusion of people with T1D in existing or planned clinical trials evaluating small molecule drugs or biologics in a non-T1D population.
  • The inclusion of an additional arm to an ongoing T1D trial to test an additional intervention or new patient population.
  • T1D-focused real world studies collecting efficacy and safety data on drugs or biologics with expected benefits for people with T1D.
• Trials should be designed to generate critical data regarding drug or biologic mechanism, efficacy, and safety in people with T1D. Trial results should allow for determination of whether selected drugs or biologics merit further development toward the ultimate goal of regulatory approval and/or enhancement of clinical guidelines for people with T1D.
• Priority consideration will be given to trials that:
  • Evaluate an immune agent or therapy with established efficacy in another autoimmune disorder for a novel assessment of an ability to disrupt the immune attack on islets and preserve beta cell function in individuals newly diagnosed with T1D.
  • Evaluate multiple agents that have individually shown efficacy in reducing/halting autoimmunity or supporting beta cell health in a combinatorial approach with a clear rationale for increasing efficacy through synergistic or additive mechanisms.
  • Evaluate proven beta cell regeneration agents with novel targeting strategies based upon strong preclinical data with a focus on establishing a safety profile for the targeted regenerative therapy.

Priorities

• Priority consideration will be given to trials that:
  • Evaluate an immune agent or therapy with established efficacy in another autoimmune disorder for a novel assessment of an ability to disrupt the immune attack on islets and preserve beta cell function in individuals newly diagnosed with T1D.
  • Evaluate multiple agents that have individually shown efficacy in reducing/halting autoimmunity or supporting beta cell health in a combinatorial approach with a clear rationale for increasing efficacy through synergistic or additive mechanisms.
  • Evaluate proven beta cell regeneration agents with novel targeting strategies based upon strong preclinical data with a focus on establishing a safety profile for the targeted regenerative therapy.

Eligibility Criteria

• Applications may be submitted by domestic and foreign non-profit organizations, public and private, such as universities, colleges, hospitals, laboratories, units of state and local governments, and eligible agencies of the federal government.
• Applicants must hold an M.D., D.M.D, D.V.M., Ph.D., or equivalent and have a faculty position or equivalent at a college, university, medical school, or other research facility. Please note that applications from for-profit entities or industry collaborations with academia may be submitted to this RFA; however, additional information will be requested from for-profit entities if a full application is invited.
• There are no citizenship requirements for this program. To assure continued excellence and diversity among applicants and awardees, Breakthrough T1D welcomes applications from all qualified individuals and encourages applications from persons with disabilities, women, and members of minority groups underrepresented in the sciences.

For more information, visit Breakthrough T1D.