The Scleroderma Research Foundation (SRF) has announced the launch of a new clinical trial platform aimed at advancing treatments for the rare autoimmune disease of the skin and organs.
Using a model created over a decade ago to accelerate oncology drug development but not previously used for autoimmune disorders, the CONQUEST platform is designed to identify agents that are ready to progress from phase 2b to phase 3 clinical trials.
The initiative, conceived and led by the SRF, will initially focus on interstitial lung disease secondary to scleroderma but will be expanded in the future to address other manifestations of the disease.
The first iteration, which is expected to begin in the fourth quarter of this year, will include approximately 400 patients and will test two drugs, including one from Sanofi. The trial will measure forced vital capacity, the total volume of air that can be exhaled during a maximal forced expiration effort, as the primary endpoint.
The effort will enrol patients across more than 130 centres in more than 22 countries, the SRF said, adding that the platform offers key advantages over traditional clinical trial protocols.
The master protocol for CONQUEST will allow new agents to enter and leave the platform without halting patient enrolment or having to resubmit the entire trial protocol for regulatory review. The trial infrastructure will also remain open, meaning the cycle can continue efficiently, and a common control group will serve multiple experimental arms.
“By assembling a global network of high-performing centres that are dedicated to treating scleroderma, the SRF and its pharmaceutical partners expect to make enduring contributions to the scleroderma community by creating an ecosystem designed to enable success in new drug development,” the organisation said.
Affecting approximately 100,000 patients in the US, scleroderma is a group of rare diseases that involve the hardening and tightening of the skin. It may also cause problems in the blood vessels, internal organs and digestive tract.
Luke Evnin, chairman of the SRF, said: “As with all of our research efforts, the incubation and assembly of CONQUEST was underwritten by generous SRF donors. With essential contributions from expert clinicians other as well as our potential pharmaceutical partners, the platform is ready.
“We are confident that this initiative will pay huge dividends for the global scleroderma community – from patients, to clinicians, to our pharmaceutical partners.”