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A randomised, double-blind, placebo-controlled phase 3 study of lenabasum in diffuse cutaneous systemic sclerosis: RESOLVE-1 design and rationale


1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15

 

  1. Weill Cornell Medical College, New York City, NY, USA. SpieraR@hss.edu
  2. University of Michigan Scleroderma Program, Ann Arbor, MI, USA.
  3. Department of Allergy and Rheumatology, Nippon Medical School Graduate School of Medicine, Tokyo, Japan.
  4. Department of Rheumatology, University of California in Los Angeles, CA, USA.
  5. University of Utah and Salt Lake City VA Health Care System Salt Lake City, UT, USA.
  6. Division of Rheumatology, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
  7. Department of Rheumatology, St Vincent’s Hospital, Melbourne, Victoria, Australia.
  8. Department of Experimental Rheumatology, University of Florence and Division of Rheumatology AOUC, Florence, Italy.
  9. Division of Rheumatology, Jewish General Hospital, Montreal, Canada.
  10. Department of Rheumatology, University Hospital Zurich, Switzerland.
  11. Corbus Pharmaceuticals, Inc. Norwood, MA, USA.
  12. Corbus Pharmaceuticals, Inc. Norwood, MA, USA.
  13. Corbus Pharmaceuticals, Inc. Norwood, MA, USA.
  14. Corbus Pharmaceuticals, Inc. Norwood, MA, USA.
  15. UCL Centre for Rheumatology and Connective Tissue Diseases, Royal Free Campus, University College London, UK.

CER13926
2021 Vol.39, N°4 ,Suppl.131
PI 0124, PF 0133
Treatment

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PMID: 34323681 [PubMed]

Received: 12/08/2020
Accepted : 27/10/2020
In Press: 21/07/2021
Published: 28/07/2021

Abstract

OBJECTIVES:
The multi-systemic, heterogenous nature of diffuse cutaneous systemic sclerosis (dcSSc) presents challenges in designing clinical studies that can demonstrate a treatment effect on overall disease burden. We describe the design of the first Phase 3 study in dcSSc patients where the American College of Rheumatology (ACR) Combined Response Index in diffuse cutaneous Systemic Sclerosis (CRISS) score was chosen prospectively as the primary outcome. The CRISS measures key clinical disease parameters and patient-reported outcomes (PROs).
METHODS:
RESOLVE-1 is a Phase 3, randomised, double-blind, placebo-controlled trial of dcSSc patients evaluating the efficacy and safety of lenabasum. Patients ≥18 years of age with dc-SSc and disease duration ≤6 years were eligible. Patients could continue stable background therapy for dcSSc, including stable immunosuppressive therapies. They were randomised to lenabasum 5 or 20 mg twice daily or placebo. The primary efficacy outcome was the mean change from baseline to 52 weeks in the ACR CRISS score.
RESULTS:
The study enrolled 365 patients over 1.5 years at 77 sites in 13 countries in North America, Europe, Israel, and Asia-Pacific, with the last patient first visit on May 1, 2019.
CONCLUSIONS:
RESOLVE-1 is the first Phase 3 interventional study to date in dcSSc to prospectively use the ACR CRISS as the primary efficacy outcome. Eligibility criteria allowed background therapy as might occur in clinical practice. This approach also facilitated timely patient enrolment. RESOLVE-1 provides a novel study design that may be used for future Phase 3 dcSSc studies to assess the holistic efficacy of therapy.

Rheumatology Article