Safety, Tolerability, and Efficacy of JBT-101 in Subjects With Dermatomyositis

NCT02466243 | Terminated Phase 2 Results

• 3 other identifiers: JBT101-DM-001116313R21AR066286
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to evaluate the safety, tolerability and efficacy of JBT-101 in adult subjects with skin-predominant, dermatomyositis (DM) that is refractory to at least 3 months treatment with hydroxychloroquine.

Conditions

Dermatomyositis

Keywords

JBT-101, lenabasum, dermatomyositis

Outcome Measures

Primary Outcomes (2)

• Change in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) From Baseline in Part A.

  The CDASI is a validated outcome measure that systematically quantifies cutaneous DM disease activity, In the CDASI, DM skin disease activity is scored from 0 to 100 based on the physician's evaluation of erythema, scale, and erosion or ulceration at 15 anatomic locations as well as alopecia, Gottron's sign or papules on the hands, and periungual changes. A 5-point or greater decrease in the CDASI activity score indicates clinically relevant improvement based on statistical analysis using a receiver operating characteristic curve to maximize sensitivity and specificity

  Part A: 84-day treatment period (Change from the Baseline CDSAI score at Day 84)

• Number of Participants With Treatment Emergent Adverse Events as a Measure of Safety and Tolerability

  Number of participants with treatment emergent adverse events were assessed as a measure of safety and tolerability

  Part A: to Day 84

Secondary Outcomes (1)

• Change in Patient-reported Outcomes From Baseline at 84 Days for Part A

  Part A: 84-day treatment period

Study Arms (2)

JBT-101

EXPERIMENTAL

Part A: JBT-101 20 mg capsule once a day on Days 1-28, then 20 mg capsule twice a day on Days 29-84. Part B: JBT-101 20 mg twice daily on Days 1 - 365 of the OLE.

Drug: JBT-101

Placebo

PLACEBO COMPARATOR

Part A: Placebo capsule once a day on Days 1-28, then placebo capsule twice a day on Days 29-84. Part B: Placebo twice daily on Days 1 - 365 of the OLE.

Drug: Placebo

Interventions

JBT-101 DRUG

Part A: 20 mg once daily on Days 1-28, then 20 mg twice daily on Days 29-84. Part B: JBT-101 20 mg twice daily on Days 1 - 365 of the OLE.

Also known as: lenabasum

JBT-101

Placebo DRUG

Part A: Once daily on Days 1-28, then twice daily on Days 29-84. Part B: Placebo twice daily on Days 1 - 365 of the OLE.

Placebo

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• CDASI activity score ≥ 14;
• No difficulty with lifting or walking, and no more than 1.5 x the upper limit of normal of creatine phosphokinase or aldolase;
• Failed at least 3 months treatment with hydroxychloroquine;
• Stable treatment for dermatomyositis for at least 28 days before Visit 1 (Day 1).
• Completion of dosing in Part A without permanent discontinuation of study product because of safety or tolerability reasons

You may not qualify if:

• Significant diseases or conditions other than DM that may influence response to the study product or safety;
• Any one of the following values for laboratory tests at Screening:
• A positive pregnancy test (or at Visit 1);
• Hemoglobin \< 10 g/dL;
• Neutrophils \< 1.0 x 10\^9/L;
• Platelets \< 75 x 10\^9/L;
• Creatinine clearance \< 50 ml/min according to modified Cockcroft-Gault equation;
• Aspartate aminotransferase, alanine aminotransferase, or alkaline phosphatase \> 2.5 x upper normal limit;
• Total bilirubin ≥ 1.5 x upper limit of normal.
• Any other condition that, in the opinion of the Principal Investigator, is clinically significant and may put the subject at greater safety risk, influence response to study product, or interfere with study assessments.

Sponsors & Collaborators

• Corbus Pharmaceuticals Inc.: lead
• National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS): collaborator
• University of Pennsylvania: collaborator

Study Sites (1)

University of Pennsylvania Perlman School of Medicine

Philadelphia, Pennsylvania, 19104, United States

Location

Related Publications (2)

• Stone CJ, Ahuja G, Lopes Almeida Gomes L, Poroye J, Faden DF, Xie L, Feng R, White B, Werth VP. Long-Term Safety and Efficacy of Lenabasum, a Cannabinoid Receptor Type 2 Agonist, in Patients with Dermatomyositis with Refractory Skin Disease: Follow-Up Data from a 3-Year Open-Label Extension Study. JID Innov. 2024 Sep 5;5(1):100311. doi: 10.1016/j.xjidi.2024.100311. eCollection 2025 Jan.

  PMID: 39403556 DERIVED

• Werth VP, Hejazi E, Pena SM, Haber J, Zeidi M, Reddy N, Okawa J, Feng R, Bashir MM, Gebre K, Jadoo AS, Concha JSS, Dgetluck N, Constantine S, White B. Safety and Efficacy of Lenabasum, a Cannabinoid Receptor Type 2 Agonist, in Patients with Dermatomyositis with Refractory Skin Disease: A Randomized Clinical Trial. J Invest Dermatol. 2022 Oct;142(10):2651-2659.e1. doi: 10.1016/j.jid.2022.03.029. Epub 2022 Apr 29.

  PMID: 35490744 DERIVED

MeSH Terms

Conditions

Dermatomyositis

Interventions

lenabasum

Condition Hierarchy (Ancestors)

Polymyositis; Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Results Point of Contact

• Title: Head of Clinical Operations
• Organization: Corbus Pharmaceuticals, Inc.

info@corbuspharma.com

6179630100

Study Officials

• Victoria Werth, M.D.

  University of Pennsylvania Perlman School of Medicine

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 2, 2015
• First Posted: June 9, 2015
• Study Start: June 1, 2015
• Primary Completion: August 1, 2017
• Study Completion: January 29, 2021
• Last Updated: January 19, 2023
• Results First Posted: January 19, 2023

Record last verified: 2022-12

Locations

US (1)