Clinical trial NCT03981744: A Study of Ustekinumab in Participants With Active Polymyositis and Dermatomyositis Who Have Not Adequately Responded to One or More Standard-of-care Treatments
Official title: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study of Ustekinumab in Participants With Active Polymyositis and Dermatomyositis Who Have Not Adequately Responded to One or More Standard-of-care Treatments
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Polymyositis
Dermatomyositis
Lead sponsor: Janssen Pharmaceutical K.K.
Sponsor class: INDUSTRY
First posted: 2019-06-11
Enrollment: 51
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
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 efficacy of ustekinumab in participants with active polymyositis (PM)/dermatomyositis (DM) despite receiving 1 or more standard-of-care treatments (for example, glucocorticoids and/or immunomodulators).


Eligibility criteria: Inclusion Criteria:

* Has a diagnosis of polymyositis (PM)/ dermatomyositis (DM) made or confirmed by a physician (such as a rheumatologist, neurologist, or dermatologist) experienced in treatment of PM/DM at least 6 weeks prior to first dose of the study drug
* Has PM or DM which is considered active despite receiving at least 1 standard-of-care treatment by the investigator
* Must be receiving 1 or more of the following protocol-permitted, systemic standard-of-care treatments: i) glucocorticoids, ii) 1 or 2 of the following immunomodulatory drugs: mycophenolate mofetil, azathioprine, oral methotrexate, oral tacrolimus, or oral cyclosporine A
* Regular or as needed treatment with topical use of glucocorticoids are permitted to treat skin lesions on a stable dose for greater than or equal to (\>=) 2 weeks prior to first dose of the study drug
* Contraceptive (birth control) use by men or women should be consistent with local regulations regarding the acceptable methods of contraception for those participating in clinical studies
* Must be medically stable on the basis of clinical laboratory tests performed at screening. If the results of the clinical laboratory tests are outside the normal reference ranges, the participant may be included only if the investigator judges the abnormalities or deviations from normal to be not clinically significant
* Demonstrable muscle weakness at screening and Week 0 measured by the Manual Muscle Testing (MMT)-8 less than or equal to (\<=)135 units
* Demonstrable muscle weakness at screening measured by any 2 or more of the followings: (i) PhGA greater than or equal to (\>=) 1.5 centimeter (cm), (ii) 1 or more muscle enzymes (Creatine kinase \[CK\], and aldolase) \>=1.4\*upper limit of normal (ULN), (iii) Myositis disease activity assessment tool (MDAAT)-Extramuscular Global Assessment \>=1.5 cm

Exclusion Criteria:

* Has myositis other than PM/DM, including but not limited to amyopathic dermatomyositis (ADM), clinically amyopathic DM, juvenile DM, inclusion body myositis (IBM) immune-mediated necrotizing myopathy diagnosed based on muscle biopsy findings and positive anti-SRP or anti-HMGCR antibody, drug-induced myositis, PM associated with human immunodeficiency virus (HIV), and muscular dystrophy, congenital myopathy, metabolic myopathy, and mitochondrial myopathy
* Has other inflammatory diseases that might confound the evaluations of efficacy, including but not limited to rheumatoid arthritis (RA), psoriatic arthritis (PsA), systemic lupus erythematosus (SLE), psoriasis, or Crohn's disease
* Has severe respiratory muscle weakness confirmed by the investigator based on the consultation with a pulmonologist and the measures of respiratory muscle strength such as maximal inspiratory pressure (MIP) and maximal expiratory pressure (MEP) and/or maximal voluntary ventilation (MVV) measurements and lung capacity such as forced vital capacity (FVC). The results need to be within population appropriate normal limits
* Has severe muscle damage (Myositis Damage Index-VAS \[Muscle Damage\] greater than (\>) 7 centimeter \[cm\]), permanent weakness due to a non-IIM cause, or myositis with cardiac dysfunction
* Has glucocorticoid-induced myopathy which the investigator considers the primary cause of muscle weakness
* Has positive test result of anti-melanoma differentiation-associated protein 5 (MDA5) antibody (anti clinically amyopathic dermatomyositis (C-ADM)-140 antibody).
* Has had a nontuberculous mycobacterial infection or opportunistic infection
* Has a history of, or ongoing, chronic or recurrent infectious disease
* Has past history of severe Interstitial lung disease (ILD) flare, severe non-infectious lung inflammation which required active intervention, or multiple relapses of these conditions
* Presence or history of malignancy within 5 years before screening

Primary outcomes:
measure: Percentage of Participants Who Achieved Minimal Improvement in International Myositis Assessment and Clinical Studies Total Improvement Score (IMACS TIS) at Week 24
timeFrame: Week 24
Secondary outcomes:
measure: Change From Baseline in Functional Index-2 (FI-2) at Week 24
timeFrame: Baseline, Week 24
measure: Percentage of Participants Who Experienced Disease Worsening Up to Week 24 Based on Consensus Criteria for Worsening
timeFrame: Up to Week 24
measure: Change From Baseline in Manual Muscle Testing (MMT)-8 Score at Week 24
timeFrame: Baseline, Week 24
measure: Change From Baseline in Physician Global Activity (PhGA) at Week 24
timeFrame: Baseline, Week 24
measure: Change From Baseline in Extramuscular Assessment by Myositis Disease Activity Assessment Tool (MDAAT) at Week 24
timeFrame: Baseline, Week 24
measure: Change From Baseline in Muscle Enzyme Levels at Week 24
timeFrame: Baseline, Week 24
Other outcomes:
Locations:
city: Bunkyo Ku
country: Japan
facility: Tokyo Medical and Dental University Hospital
city: Fukushima
country: Japan
facility: Fukushima Medical University Hospital
city: Hyogo
country: Japan
facility: Shinko Hospital
city: Isehara
country: Japan
facility: Tokai University Hospital
city: Kagoshima City
country: Japan
facility: Kagoshima University Hospital
city: Kanagawa
country: Japan
facility: St Marianna University Hospital
city: Kawachi Nagano
country: Japan
facility: National Hospital Organization Osaka Minami Medical Center
city: Kita-kyushu
country: Japan
facility: Hospital of the University of Occupational and Enviromental Health
city: Kumamoto
country: Japan
facility: Kumamoto University Hospital
city: Kurashiki
country: Japan
facility: Kurashiki Central Hospital
city: Matsumoto
country: Japan
facility: Shinshu University Hospital
city: Nagano
country: Japan
facility: Minaminagano Medical Center Shinonoi General Hospital
city: Nagasaki-shi
country: Japan
facility: Nagasaki University Hospital
city: Nagoya-shi
country: Japan
facility: National Hospital Organization Nagoya Medical Center
city: Niigata
country: Japan
facility: Niigata University Medical And Dental Hospital
city: Okayama
country: Japan
facility: Okayama City General Medical Center Okayama City Hospital
city: Sagamihara
country: Japan
facility: Kitasato University Hospital
city: Saga
country: Japan
facility: Saga University Hospital
city: Sakai City
country: Japan
facility: Sakai City Medical Center
city: Sendai shi
country: Japan
facility: Tohoku University Hospital
city: Sendai
country: Japan
facility: Tohoku Medical And Pharmaceutical University Hospital
city: Shimotsuga Gun
country: Japan
facility: Dokkyo Medical University Hospital
city: Shinjuku ku
country: Japan
facility: Center Hospital of the National Center for Global Health and Medicine
city: Tokyo
country: Japan
facility: The Jikei University Hospital
city: Tokyo
country: Japan
facility: Nippon Medical School Hospital
city: Tokyo
country: Japan
facility: Juntendo University Hospital
city: Tokyo
country: Japan
facility: Tokyo Medical University Hospital
city: Toon
country: Japan
facility: Ehime University Hospital
city: Toyoake
country: Japan
facility: Fujita Health University Hospital
city: Tsukuba
country: Japan
facility: University of Tsukuba Hospital
city: Ube
country: Japan
facility: Yamaguchi University Hospital
city: Yokohama
country: Japan
facility: Yokohama Rosai Hospital
Study officials:
affiliation: Janssen Pharmaceutical K.K.
name: Janssen Pharmaceutical K.K., Japan Clinical Trial
role: STUDY_DIRECTOR
References: