Enrollment Complete in Phase 3 Trial Testing Lenabasum in Diffuse Cutaneous SSc Patients
Enrollment has been completed in an ongoing Phase 3 clinical trial assessing the safety and efficacy of experimental therapy lenabasum in patients with systemic sclerosis (SSc).
Corbus Pharmaceuticals, lenabasum’s developer, expects to announce top-line results from the study during the summer of 2020, and hopes they will support an initial marketing application for the therapy.
“Completion of subject enrollment in this pivotal study is a key milestone to support the potential filing of a New Drug Application (NDA) in the U.S. and marketing authorization applications elsewhere,” Barbara White, MD, chief medical officer of Corbus, said in a press release. “We are grateful to the participants for their commitment and all our investigators and their staffs for their dedication and support of the study.”
The RESOLVE-1 trial (NCT03398837) has enrolled a total of 365 adults diagnosed with diffuse cutaneous SSc at more than 70 clinical centers across North America, Europe, Israel, Japan, South Korea, and Australia.
Participants will be randomized to receive 5 or 20 mg of lenabasum or a placebo twice a day for 52 weeks, in addition to a four-week follow-up period.
The trial was planned to provide detailed information on the safety and efficacy of lenabasum to help manage the symptoms associated with SSc.
In April, the company announced it had changed the primary efficacy outcome of the study in U.S. sites after a meeting with the U.S. Food and Drug Administration (FDA). This adjustment was intended to enhance the chances of the trial’s success and, consequently, of future approval of lenabasum.
Now, the trial’s primary efficacy goal is the change in the American College of Rheumatology Combined Response Index in diffuse cutaneous SSc (ACR CRISS) score after one year (52 weeks) of treatment at each of its 27 U.S. sites.
For the remaining international sites, researchers will determine lenabasum’s efficacy according to changes in the modified Rodnan Skin score (mRSS) after one year of treatment. Changes in mRSS will be determined at U.S. sites as well, but researchers will consider it a secondary measure to show the treatment’s effectiveness.
The ACR CRISS score is a composite measure that takes into consideration different aspects of the patient’s response to treatment in SSc. It was provisionally endorsed by the American College of Rheumatology to test new therapies for diffuse cutaneous SSc.
The score is calculated according to weighted changes from the study’s start in mRSS, Health Assessment Questionnaire — Disability Index (HAQ-DI), forced vital capacity (FVC) percent predicted (a measure of lung function), and patient and physician global assessments of health related to SSc.
All RESOLVE-1 participants will have the opportunity to continue treatment with lenabasum upon completion of the trial — which is expected to end in early 2020 — in an open-label extension study.
Lenabasum has been granted orphan drug status and fast track designation by the FDA, and orphan drug designation by the European Medicines Agency for the treatment of SSc.
