It has now enrolled 211 patients worldwide for a trial that will be conducted at 106 sites.
The focuSSced study (NCT02453256) will be assessing the effectiveness and safety of Actemra, compared with a placebo, as a scleroderma therapy. The autoimmune disease causes thickening and scarring of the skin as well as respiratory problems.
The U.S. Food and Drug Administration has yet to approve a scleroderma treatment. It granted Actemra breakthrough therapy designation in 2015, however.
Genentech made the breakthrough therapy announcement at the European League Against Rheumatism Conference in Rome when it presented Phase 2 trial data that paved the way for the Phase 3 study.
In the Phase 2 trial, Actemra reduced patients’ skin thickening after 24 weeks — and the improvement continued in a second 24-week round of treatment.
The FDA approved Actemra as a treatment for giant cell arteritis in May 2017. It was the first FDA approval for the condition, but the sixth for Actemra.
The randomized, placebo-controlled Phase 3 trial will be evaluating the effectiveness of Actemra versus a placebo at 48 weeks. Researchers will use the modified Rodnan skin score as the measuring stick.
Researchers will also look at Actemra’s ability to preserve lung function, using a measure known as forced vital capacity. After the 48-week trial, patients receiving Actemra will have a chance to continue the therapy in a 48-week extension trial.
“I am extremely delighted by recruitment of the last subject in the focuSSced trial,” Dr. Dinesh Khanna, director of the University of Michigan Scleroderma Program, said in a press release. “The trial recruited 211 subjects with early systemic sclerosis, a disease with high unmet need,” said Khanna, the study’s lead investigator.
“Having reached our first milestone of completing enrollment, we continue to push forward with the determination that we can potentially deliver a therapeutic option to a patient population that has serious unmet need,” said Dr. Jeffrey Siegel, the global head of Genentech’s rheumatology and rare diseases operation. “We hope the Phase III trial results will show that treatment with Actemra not only shows improvement in skin thickening over time, which is crucial for these patients, but also the preservation of lung function.”