With the goal of speeding up the evaluation of new investigational therapies for multiple myeloma (MM), the Multiple Myeloma Research Foundation (MMRF) has started developing what it calls a “master protocol” to allow patients to participate in clinical trials evaluating several investigational therapies at once. MMRF will be working with the FDA, the National Cancer Institute (NCI), pharmaceutical, biotech, and diagnostic industry members, academic center leaders, and patients to develop the protocol.
The MM Master Protocol is a biomarker-driven clinical trial that MMRF says can match patients with relapsed disease with targeted therapies based on the genomic profile of their tumor. The protocol would allow several Phase II trials to take place in parallel within a single study design. Patients would be assigned to an experimental therapy based on an analysis of their genetic alterations, allowing multiple new therapies to be evaluated more rapidly than with a traditional trial design. This, according to MMRF, would give researchers the ability to expedite decisions to advance a drug to Phase III testing in a specific genetic subgroup, or to test a different drug in this subgroup.
The protocol was the topic of discussion at a workshop held October 22, 2014, in Washington, D.C., which included attendees from the FDA and NCI. In addition to discussing protocol design, participants also addressed the application of minimal residual disease (MRD) measurements as surrogate endpoints for MM trials. Multiple reports, according to MMRF, suggest that MRD can a strong predictor of long-term progression-free survival in multiple myeloma.
“We have made real progress in the battle against multiple myeloma, but there is still more work to do to bring new, effective, personalized treatments to patients,” MMRF's president and CEO Walter M. Capone said in a statement. “The development of a Multiple Myeloma Master Protocol offers a promising solution to accelerate the development of critical targeted therapies to patients through a novel research model.”