RTI International is submitting this application for the Clinical Trials Development Resource for Hematologic Disorders (U24), RFA-HL-12-016. The Resource will support the development of clinical trials by investigators who have ideas for new treatments of blood disorders but lack access to the resources needed to design trials to test these ideas. The RTI Resource team includes highly experienced individuals with diverse backgrounds that cover all aspects of multicenter clinical trials. Our biostatisticians have collaborated extensively with clinical investigators on trial design, monitoring, and analysis, including trials in hematological diseases (sickle cell disease, hemophilia and TTP). Clinical study managers will advise on the practical aspects of trial implementation (recruitment of study sites and subjects, and development of manuals of operations and other study materials). The team includes expert programmers to advise on data entry and data management systems, regulatory specialists with experience interacting with FDA and other regulatory agencies, and bioethicists to advise on ethical conduct of clinical trials. Our team also includes several hematology consultants experienced in designing clinical trials for a wide range of blood disorders. Finally, our team includes groups with considerable experience offering formal training on trial design and management. Drawing on this broad-based expertise, the RTI Resource will accomplish two broad Aims. First, advice from the Resource will provide the R34/U34 investigators with the tools and knowledge that they need to develop high-quality, effective proposals at the end of the trial planning phase. Also, that advice will lead to development by the R34/U34 investigators of feasible, scientifically sound, well-designed protocols that can be implemented efficiently through an R01/U01 mechanism. We will offer training and mentoring to improve the design skills of the investigators so as to improve future research studies beyond the term of the Resource. Scientific leadership will be provided Dr. Donald Brambilia as the Resource Principal Investigator who has over 25 years of experience as a statistician in leadership of clinical trials including 20 years in hematological studies working with many of the leading research hematologists in the nation. Dr. Nigel Key is our lead hematology consultant cooperating with 5 other hematologists to support the U34/R34 investigators. We also include personnel with experience in all aspects for clinical trials design, development, implementation and operations.
Clinicians with knowledge of blood disorder and ideas for new treatments face several obstacles that retard progress toward effective development of the required clinical trials. The lack of access to consultative resources that support trial design is major among the obstacles. This Resource will provide essential support to the investigators developing new therapies and clinical trials that is not always available to them.