Trial to Evaluate Efficacy and Safety of Lenabasum in Dermatomyositis
- 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
-
Fulfill at least one of the following criteria for dermatomyositis:
- Bohan and Peter criteria (Bohan and Peter, 1975a; Bohan and Peter 1975b)
- ACR/EULAR criteria (Lundberg et al, 2017)
-
Disease activity/severity fulfills at least one of the following three criteria:
- MDGA ≥ 3 cm (0 - 10 cm Visual Analog Scale [VAS]) and MMT-8 score ≤ 142 (out of 150 total possible)
- Sum of MDGA, PtGA and EMGA VAS scores is ≥ 10 cm (0-10 cm VAS for each)
- MDGA ≥ 3 cm (0-10 cm VAS) and CDASI activity score of > 14
-
Stable doses of immunosuppressive medications for DM as defined by:
- Unchanged dose of oral corticosteroids ≤ 20 mg per day prednisone or equivalent for ≥ 4 weeks before Visit 1
- Unchanged dose of immunosuppressive medications other than oral corticosteroids for ≥ 8 weeks before Screening
-
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:
- A positive pregnancy test (or at Visit 1)
- Hemoglobin < 9 g/dL in males and < 8 g/dL in females
- Neutrophils < 1.0 × 10^9/L
- Platelets < 75 × 10^9/L
- 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
Group Intervention Description Lenabasum 20 mg Lenabasum 20 mg Subjects will receive lenabasum 20 mg twice daily Lenabasum 5 mg Lenabasum 5 mg Subjects will receive lenabasum 5 mg twice daily Placebo Placebo Subjects will receive placebo twice daily
- Primary Outcome Measures
Name Time Method 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
Name Time Method 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 Baseline Week 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 activity Week 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 10 Week 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 score Week 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 placebo 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.
Trial Locations
- Locations (52)
Johns Hopkins Bayview Medical Center
🇺🇸Baltimore, Maryland, United States
Cleveland Clinic
🇺🇸Cleveland, Ohio, United States
University of Pittsburgh, Division of Rheumatology
🇺🇸Pittsburgh, Pennsylvania, United States
Kliniczny Szpital Wojewodzki Nr 1. im Fryderyka Chopina Klinika Dermatologii
🇵🇱Rzeszow, Poland
UCLA Division of Rheumatology
🇺🇸Los Angeles, California, United States
University of Miami
🇺🇸Miami, Florida, United States
Attune Health Center
🇺🇸Beverly Hills, California, United States
Hospital for Special Surgery
🇺🇸New York, New York, United States
University Hospital "Kaspela" Rheumatology Clinic
🇧🇬Plovdiv, Bulgaria
University Hospital Erlangen Nuremberg
🇩🇪Erlangen, Germany
Fondazione Policlinico Universitario A.Gemelli-IRCCS
🇮🇹Roma, Italy
Hospital 12 Octubre
🇪🇸Madrid, Spain
KLIMED
🇵🇱Łomża, Poland
Osaka University Hospital
🇯🇵Osaka, Japan
University Medical Center Goettingen
🇩🇪Göttingen, Germany
UMHAT "St. Ivan Rilski"
🇧🇬Sofia, Bulgaria
Northwestern University
🇺🇸Chicago, Illinois, United States
Washington University in St. Louis
🇺🇸Saint Louis, Missouri, United States
University of Pennsylvania
🇺🇸Philadelphia, Pennsylvania, United States
Tohoku University Hospital
🇯🇵Miyagi, Japan
Rheumatic Disease Center
🇺🇸Glendale, Wisconsin, United States
UMHAT
🇧🇬Stara Zagora, Bulgaria
University of Debrecen
🇭🇺Debrecen, Hungary
Revmatologicky ustav
🇨🇿Prague, Czechia
Charite-Universitatsmedizin
🇩🇪Berlin, Germany
University Hospital Policlinico-Vittorio Emanuele
🇮🇹Catania, Italy
Gunma University Hospital
🇯🇵Gunma, Japan
Hokkaido University Hospital
🇯🇵Hokkaido, Japan
Yokohama City University Hospital
🇯🇵Kanagawa, Japan
Kyoto University Hospital
🇯🇵Kyoto, Japan
Wakayama Medical Hospital
🇯🇵Wakayama, Japan
Keio University Hospital
🇯🇵Tokyo, Japan
Nippon Medical School Hospital
🇯🇵Tokyo, Japan
Seoul National University Hospital
🇰🇷Seoul, Korea, Republic of
Vall d'Hebron General Hospital
🇪🇸Barcelona, Spain
Karolinska University Hospital, Rheumatology Clinic
🇸🇪Stockholm, Sweden
King's College Hospital NHS Foundation Trust
🇬🇧London, United Kingdom
Inha University Hospital
🇰🇷Incheon, Korea, Republic of
Hanyang University Hospital
🇰🇷Seoul, Korea, Republic of
Seoul St. Mary's Hospital
🇰🇷Seoul, Korea, Republic of
University of British Columbia, Dept. of Dermatology and Skin Science
🇨🇦Vancouver, British Columbia, Canada
Mayo Clinic
🇺🇸Jacksonville, Florida, United States
Austin Neuromuscular Center
🇺🇸Austin, Texas, United States
HonorHealth Neurology
🇺🇸Phoenix, Arizona, United States
Denver Arthritis Clinic
🇺🇸Denver, Colorado, United States
University of South Florida
🇺🇸Tampa, Florida, United States
DelRicht Research
🇺🇸New Orleans, Louisiana, United States
University of Minnesota, Division of Rheumatic and Autoimmune Diseases
🇺🇸Minneapolis, Minnesota, United States
MUSC: Department of Neurology
🇺🇸Charleston, South Carolina, United States
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
Georgetown University
🇺🇸Washington, District of Columbia, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States