Clinical trial NCT03813160: Trial to Evaluate Efficacy and Safety of Lenabasum in Dermatomyositis
Official title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 3 Trial to Evaluate Efficacy and Safety of Lenabasum in Dermatomyositis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Dermatomyositis
Keywords: dermatomyositis
cannabinoid receptor type 2 agonist
JBT-101
lenabasum
Lead sponsor: Corbus Pharmaceuticals Inc.
Sponsor class: INDUSTRY
First posted: 2019-01-23
Enrollment: 176
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Efficacy of lenabasum 20 mg BID compared to placebo BID as measured by Total Improvement Score (TIS)
timeFrame: Week 28
Secondary outcomes:
measure: Subjects who achieve Definition of Improvement (DOI)
timeFrame: Week 28
measure: Subjects who improve by at least one category on the Investigator Global Assessment (IGA) scale of skin activity
timeFrame: Week 28
measure: Change in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) activity score
timeFrame: Week 28
measure: Subjects who achieve TIS >= 40 (at least moderate improvement)
timeFrame: Week 28
measure: TIS in subjects receiving immunosuppressive therapies (including corticosteroids) for > 1 year at Baseline
timeFrame: Week 52
measure: Change in Forced vital capacity (FVC) absolute, in all subjects and those with interstitial lung disease (ILD) at Baseline.
timeFrame: Week 28
measure: Change in Forced vital capacity (FVC) percent predicted, in all subjects and those with interstitial lung disease (ILD) at Baseline.
timeFrame: Week 28
measure: TIS at Visit 10
timeFrame: Week 52
measure: TIS, lenabasum 5 mg BID versus placebo
timeFrame: Week 28
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: HonorHealth Neurology
state: Arizona
city: Scottsdale
country: United States
facility: Mayo Clinic
state: Arizona
city: Beverly Hills
country: United States
facility: Attune Health Center
state: California
city: Los Angeles
country: United States
facility: UCLA Division of Rheumatology
state: California
city: Denver
country: United States
facility: Denver Arthritis Clinic
state: Colorado
city: Washington
country: United States
facility: Georgetown University
state: District of Columbia
city: Jacksonville
country: United States
facility: Mayo Clinic
state: Florida
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: New Orleans
country: United States
facility: DelRicht Research
state: Louisiana
city: Baltimore
country: United States
facility: Johns Hopkins Bayview Medical Center
state: Maryland
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Minneapolis
country: United States
facility: University of Minnesota, Division of Rheumatic and Autoimmune Diseases
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University in St. Louis
state: Missouri
city: New York
country: United States
facility: Hospital for Special Surgery
state: New York
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh, Division of Rheumatology
state: Pennsylvania
city: Charleston
country: United States
facility: MUSC: Department of Neurology
state: South Carolina
city: Austin
country: United States
facility: Austin Neuromuscular Center
state: Texas
city: Glendale
country: United States
facility: Rheumatic Disease Center
state: Wisconsin
city: Plovdiv
country: Bulgaria
facility: University Hospital "Kaspela" Rheumatology Clinic
city: Sofia
country: Bulgaria
facility: UMHAT "St. Ivan Rilski"
city: Stara Zagora
country: Bulgaria
facility: UMHAT
city: Vancouver
country: Canada
facility: University of British Columbia, Dept. of Dermatology and Skin Science
state: British Columbia
city: Prague
country: Czechia
facility: Revmatologicky ustav
city: Berlin
country: Germany
facility: Charite-Universitatsmedizin
city: Erlangen
country: Germany
facility: University Hospital Erlangen Nuremberg
city: Göttingen
country: Germany
facility: University Medical Center Goettingen
city: Debrecen
country: Hungary
facility: University of Debrecen
city: Catania
country: Italy
facility: University Hospital Policlinico-Vittorio Emanuele
city: Roma
country: Italy
facility: Fondazione Policlinico Universitario A.Gemelli-IRCCS
city: Gunma
country: Japan
facility: Gunma University Hospital
city: Hokkaido
country: Japan
facility: Hokkaido University Hospital
city: Kanagawa
country: Japan
facility: Yokohama City University Hospital
city: Kyoto
country: Japan
facility: Kyoto University Hospital
city: Miyagi
country: Japan
facility: Tohoku University Hospital
city: Osaka
country: Japan
facility: Osaka University Hospital
city: Tokyo
country: Japan
facility: Keio University Hospital
city: Tokyo
country: Japan
facility: Nippon Medical School Hospital
city: Wakayama
country: Japan
facility: Wakayama Medical Hospital
city: Incheon
country: Korea, Republic of
facility: Inha University Hospital
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital
city: Seoul
country: Korea, Republic of
facility: Hanyang University Hospital
city: Seoul
country: Korea, Republic of
facility: Seoul St. Mary's Hospital
city: Bialystok
country: Poland
facility: KLIMED
city: Rzeszow
country: Poland
facility: Kliniczny Szpital Wojewodzki Nr 1. im Fryderyka Chopina Klinika Dermatologii
city: Łomża
country: Poland
facility: KLIMED
city: Barcelona
country: Spain
facility: Vall d'Hebron General Hospital
city: Madrid
country: Spain
facility: Hospital 12 Octubre
city: Stockholm
country: Sweden
facility: Karolinska University Hospital, Rheumatology Clinic
city: London
country: United Kingdom
facility: King's College Hospital NHS Foundation Trust
Study officials:
affiliation: University of Pennsylvania
name: Victoria P Werth, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Pittsburgh Department of Medicine/Division of Rheumatology
name: Chester V Oddis, MD
role: PRINCIPAL_INVESTIGATOR
References: