The Scleroderma Research Foundation Launches Clinical Trial Platform

Rare Daily Staff

The Scleroderma Research Foundation said Sanofi agreed to contribute the first experimental agent to CONQUEST, an innovative clinical trial platform created by the foundation designed to rapidly advance promising treatments for scleroderma and to identify agents that should progress to late-stage clinical trials.

CONQUEST is based on a model first created more than a decade ago to accelerate the development of cancer therapies. The group said it is the first of its kind in rare autoimmune diseases. In its initial iteration, the platform will focus on interstitial lung disease secondary to scleroderma.

SRF said it will enroll patients across more than 130 centers in more than 22 countries. The foundation plans to expand the platform to address other manifestations of scleroderma.

Scleroderma is a systemic autoimmune disease that affects approximately 100,000 patients in the United States with high morbidity and mortality rates and for which there are limited treatment options. The disease presents multiple challenges for patients with a spectrum of symptoms that prominently include vascular disease and internal organ fibrosis.

CONQUEST uses a master protocol that provides a regulatory framework to study multiple potential treatments for scleroderma, and like other platform trials, this provides multiple efficiencies not available in traditional clinical trial protocols, including:

• New agents may enter and leave the platform without halting patient enrollment or resubmitting the entire clinical trial protocol for regulatory review.
• The trial infrastructure is built once but remains open with substantial efficiencies for each cycle and enabling knowledge gained to improve the platform over time.
• A common control group is enrolled that serves multiple experimental arms.

In all, the platform provides advantages over conventional trials including smaller sample sizes, fewer patients on placebo, reduced costs, faster timelines to data, better inferences for regulators, and the ability to share the accumulated learnings across the entire scleroderma research community.

By assembling a global network of high-performing centers 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.

“Our commitment to innovation in scleroderma spans basic, translational, and clinical research. A decade ago, we began to plan how we could adapt some of the impressive clinical development innovations in cancer to scleroderma,” said Luke Evnin, chairman of the Scleroderma Research Foundation. “Recent advances in understanding drivers of autoimmunity and fibrosis have generated a rich pipeline of promising therapeutics across autoimmunity and specifically, for scleroderma. However, among other issues, the breadth and depth of opportunities pose clinical development challenges in a rare disease setting such as scleroderma, and CONQUEST arrives at the right moment to address those challenges.”

Photo: Luke Evnin, chairman of the Scleroderma Research Foundation