Clinical trial NCT02466243: Safety, Tolerability, and Efficacy of JBT-101 in Subjects With Dermatomyositis
Official title: A Phase 2, Double-blind, Randomized, Placebo-controlled Study to Investigate the Safety, Tolerability, and Efficacy of JBT-101 in Subjects With Dermatomyositis
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Dermatomyositis
Keywords: JBT-101, lenabasum, dermatomyositis
Lead sponsor: Corbus Pharmaceuticals Inc.
Sponsor class: INDUSTRY
Collaborators: name: National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
name: University of Pennsylvania
First posted: 2015-06-09
Enrollment: 22
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Detailed description: Part A: An interventional, double-blind, randomized, placebo-control design will be used to test JBT-101 in about 22 eligible male or female subjects ≥ 18 and ≤ 70 years of age with moderate-to-severe active skin-predominant dermatomyositis.

Part B: A one-year open-label design to test JBT-101 in subjects who completed Part A without permanent discontinuation of study product because of safety or tolerability reasons.

Eligibility criteria: Inclusion Criteria (Part A):

* 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).

Inclusion Criteria (Part B):

* Completion of dosing in Part A without permanent discontinuation of study product because of safety or tolerability reasons

Exclusion Criteria (Part A and B):

* 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:

  1. A positive pregnancy test (or at Visit 1);
  2. Hemoglobin \< 10 g/dL;
  3. Neutrophils \< 1.0 x 10\^9/L;
  4. Platelets \< 75 x 10\^9/L;
  5. Creatinine clearance \< 50 ml/min according to modified Cockcroft-Gault equation;
  6. Aspartate aminotransferase, alanine aminotransferase, or alkaline phosphatase \> 2.5 x upper normal limit;
  7. 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.

Primary outcomes:
measure: Change in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) From Baseline in Part A.
timeFrame: Part A: 84-day treatment period (Change from the Baseline CDSAI score at Day 84)
measure: Number of Participants With Treatment Emergent Adverse Events as a Measure of Safety and Tolerability
timeFrame: Part A: to Day 84
Secondary outcomes:
measure: Change in Patient-reported Outcomes From Baseline at 84 Days for Part A
timeFrame: Part A: 84-day treatment period
Other outcomes:
Locations:
city: Philadelphia
country: United States
facility: University of Pennsylvania Perlman School of Medicine
state: Pennsylvania
Study officials:
affiliation: University of Pennsylvania Perlman School of Medicine
name: Victoria Werth, M.D.
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.