MedPath

Trial to Evaluate Efficacy and Safety of Lenabasum in Dermatomyositis

Phase 3
Completed
Conditions
Dermatomyositis
Interventions
Registration Number
NCT03813160
Lead Sponsor
Corbus Pharmaceuticals Inc.
Brief Summary

This is a Phase 3 multicenter, double-blind, randomized, placebo-controlled study assessing the efficacy and safety of lenabasum for the treatment of dermatomyositis. Approximately 150 subjects will be enrolled in this study at about 60 sites in North America, Europe, and Asia. The planned duration of double-blind treatment with study drug is up to 52 weeks.

Detailed Description

Subjects will be randomized to receive lenabasum 20 mg twice per day, lenabasum 5 mg twice per day, or placebo twice per day in a 2:1:2 ratio. The primary efficacy outcome at Week 28 will compare lenabasum 20 mg BID to placebo the Total Improvement Score (TIS), which is a weighted composite measure of improvement from baseline in six endpoints: Physician Global Assessment of Disease Activity, Physician Assessment of Extramuscular Disease Activity, Patient Global Assessment of Disease Activity, Health Assessment Questionnaire (patient-reported disability), Manual Muscle Testing (MMT), and muscle enzymes.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
176
Inclusion Criteria
  • Fulfill at least one of the following criteria for dermatomyositis:

    1. Bohan and Peter criteria (Bohan and Peter, 1975a; Bohan and Peter 1975b)
    2. ACR/EULAR criteria (Lundberg et al, 2017)
  • Disease activity/severity fulfills at least one of the following three criteria:

    1. MDGA ≥ 3 cm (0 - 10 cm Visual Analog Scale [VAS]) and MMT-8 score ≤ 142 (out of 150 total possible)
    2. Sum of MDGA, PtGA and EMGA VAS scores is ≥ 10 cm (0-10 cm VAS for each)
    3. MDGA ≥ 3 cm (0-10 cm VAS) and CDASI activity score of > 14
  • Stable doses of immunosuppressive medications for DM as defined by:

    1. Unchanged dose of oral corticosteroids ≤ 20 mg per day prednisone or equivalent for ≥ 4 weeks before Visit 1
    2. Unchanged dose of immunosuppressive medications other than oral corticosteroids for ≥ 8 weeks before Screening
Exclusion Criteria
  • Unstable DM or DM with end-stage organ involvement at Screening or Visit 1

  • Significant diseases or conditions other than DM that may influence response to the study drug or safety

  • Any of the following values for laboratory tests at Screening:

    1. A positive pregnancy test (or at Visit 1)
    2. Hemoglobin < 9 g/dL in males and < 8 g/dL in females
    3. Neutrophils < 1.0 × 10^9/L
    4. Platelets < 75 × 10^9/L
    5. Creatinine clearance < 50 mL/min on screening blood test, per the Modification of Diet in Renal Disease Study or in 24 hour urine creatine clearance measurement

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Lenabasum 20 mgLenabasum 20 mgSubjects will receive lenabasum 20 mg twice daily
Lenabasum 5 mgLenabasum 5 mgSubjects will receive lenabasum 5 mg twice daily
PlaceboPlaceboSubjects will receive placebo twice daily
Primary Outcome Measures
NameTimeMethod
Efficacy of lenabasum 20 mg BID compared to placebo BID as measured by Total Improvement Score (TIS)Week 28

TIS from IMAC Core Set Measures (CSM) will be calculated following Aggarwal et al (2017) recommendations. Scores are based on a 0 - 100 scale; higher scores indicate better improvement in myositis.

Secondary Outcome Measures
NameTimeMethod
Subjects who achieve Definition of Improvement (DOI)Week 28

Defined as ≥ 3 of 6 core set measures improved by ≥ 20% (relative to Baseline) with no more than 2 core set measures worsening by ≥ 25% (MMT-8 may not decrease by ≥ 25% from baseline)

TIS in subjects receiving immunosuppressive therapies (including corticosteroids) for > 1 year at BaselineWeek 52

TIS from IMAC Core Set Measures (CSM) will be calculated following Aggarwal et al (2017) recommendations. Scores are based on a 0 - 100 scale; higher scores indicate better improvement in myositis.

Change in Forced vital capacity (FVC) absolute, in all subjects and those with interstitial lung disease (ILD) at Baseline.Week 28

ILD is defined as a history of fibrosis on chest x-ray, a history of ILD on CT of lungs, and/or FVC% predicted \<80% at Screening or Visit 1

Subjects who improve by at least one category on the Investigator Global Assessment (IGA) scale of skin activityWeek 28

The IGA is used by the investigator to score overall skin disease on a 0 to 4 scale; higher scores indicate greater skin disease.

Subjects who achieve TIS >= 40 (at least moderate improvement)Week 28

TIS from IMAC Core Set Measures (CSM) will be calculated following Aggarwal et al (2017) recommendations. Scores are based on a 0 - 100 scale; higher scores indicate better improvement in myositis.

Change in Forced vital capacity (FVC) percent predicted, in all subjects and those with interstitial lung disease (ILD) at Baseline.Week 28

ILD is defined as a history of fibrosis on chest x-ray, a history of ILD on CT of lungs, and/or FVC% predicted \<80% at Screening or Visit 1

TIS at Visit 10Week 52

TIS from IMAC Core Set Measures (CSM) will be calculated following Aggarwal et al (2017) recommendations. Scores are based on a 0 - 100 scale; higher scores indicate better improvement in myositis.

Change in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) activity scoreWeek 28

CDASI is a validated outcome measure that systematically quantifies cutaneous DM disease activity and damage (Klein et al, 2007; Yassaee et al, 2010) Disease Activity Score is rated using three activity measures. The activity score ranges from 0 to 100. Higher scores indicate greater disease severity.

TIS, lenabasum 5 mg BID versus placeboWeek 28

TIS from IMAC Core Set Measures (CSM) will be calculated following Aggarwal et al (2017) recommendations. Scores are based on a 0 - 100 scale; higher scores indicate better improvement in myositis.

Trial Locations

Locations (52)

HonorHealth Neurology

🇺🇸

Phoenix, Arizona, United States

Mayo Clinic

🇺🇸

Jacksonville, Florida, United States

Attune Health Center

🇺🇸

Beverly Hills, California, United States

UCLA Division of Rheumatology

🇺🇸

Los Angeles, California, United States

Denver Arthritis Clinic

🇺🇸

Denver, Colorado, United States

Georgetown University

🇺🇸

Washington, District of Columbia, United States

University of Miami

🇺🇸

Miami, Florida, United States

University of South Florida

🇺🇸

Tampa, Florida, United States

Northwestern University

🇺🇸

Chicago, Illinois, United States

University of Kansas Medical Center

🇺🇸

Kansas City, Kansas, United States

Scroll for more (42 remaining)
HonorHealth Neurology
🇺🇸Phoenix, Arizona, United States
© Copyright 2025. All Rights Reserved by MedPath