Safety and Efficacy of Lenabasum, a Cannabinoid Receptor Type 2 Agonist, in Patients with Dermatomyositis with Refractory Skin Disease: A Randomized Clinical Trial.

Abstract

BACKGROUND:

Treatment options are limited for skin disease in dermatomyositis. Lenabasum is a cannabinoid receptor type 2 agonist that triggers the resolution of inflammation.

OBJECTIVE:

The objective of this study was to evaluate the safety and efficacy of lenabasum in patients with refractory cutaneous dermatomyositis.

DESIGN:

This study was a single-center, double-blind, randomized, placebo-controlled phase 2 study conducted from July 2015 to August 2017.

POPULATION:

The population included subjects aged ≥18 years with at least moderately active dermatomyositis skin activity by Cutaneous Dermatomyositis Disease Area and Severity Index activity ≥ 14 and failure or intolerance to hydroxychloroquine.

INTERVENTION:

Participants received 20 mg lenabasum daily for 28 days and then 20 mg twice per day for 56 days or placebo.

MAIN OUTCOMES AND MEASURES:

The primary outcome was a change in Cutaneous Dermatomyositis Disease Area and Severity Index activity. Safety and other secondary efficacy assessments were performed till day 113.

RESULTS:

A total of 22 subjects were randomized to lenabasum (n = 11) or placebo (n = 11). No serious or severe adverse events were related to lenabasum, and no participants discontinued the study. The adjusted least-squares mean for Cutaneous Dermatomyositis Disease Area and Severity Index activity decreased more for lenabasum, and the difference was significant on day 113 (least-squares mean [standard error] difference = ‒6.5 [3.1], P = 0.038). Numerically greater improvements were seen in multiple secondary efficacy outcomes and biomarkers with lenabasum.

CONCLUSION:

Lenabasum treatment was well tolerated and was associated with greater improvement in Cutaneous Dermatomyositis Disease Area and Severity Index activity and multiple efficacy outcomes.

TRIAL REGISTRATION:

This study was registered at ClinicalTrials.gov, NCT02466243.

• Authors: Victoria P Werth, Emily Hejazi, Sandra M Pena, Jessica Haber, Majid Zeidi, Nithin Reddy, Joyce Okawa, Rui Feng, Muhammad M Bashir, Kirubel Gebre, Arvin S Jadoo, Josef Symon S Concha, Nancy Dgetluck, Scott Constantine, Barbara White
• Journal: The Journal of investigative dermatology (J Invest Dermatol) Vol. 142 Issue 10 Pg. 2651-2659.e1 (10 2022) ISSN: 1523-1747 [Electronic] United States
• PMID: 35490744 (Publication Type: Clinical Trial, Phase II, Journal Article, Randomized Controlled Trial, Research Support, Non-U.S. Gov't, Research Support, N.I.H., Extramural)
• Copyright: Copyright © 2022 The Authors. Published by Elsevier Inc. All rights reserved.

Chemical References

• Biomarkers
• Cannabinoid Receptor Agonists
• Receptors, Cannabinoid
• Hydroxychloroquine
• Dronabinol
• lenabasum

Topics

• Adolescent
• Adult
• Biomarkers
• Cannabinoid Receptor Agonists (adverse effects)
• Dermatomyositis (diagnosis, drug therapy)
• Double-Blind Method
• Dronabinol (analogs & derivatives)
• Humans
• Hydroxychloroquine (adverse effects)
• Receptors, Cannabinoid
• Treatment Outcome