A Study of Ustekinumab in Participants With Active Polymyositis and Dermatomyositis Who Have Not Adequately Responded to One or More Standard-of-care Treatments

NCT ID: NCT03981744

Last Updated: 2025-04-29

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 51 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-07-26
• Study Completion Date: 2022-07-12

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).

Conditions

Polymyositis; Dermatomyositis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

Group 1: Ustekinumab

Participants will receive body weight-range based IV dosing of approximately 6 milligram per kilogram (mg/kg) of ustekinumab at Week 0 followed by ustekinumab 90 milligram (mg) subcutaneously (SC) at Week 8 and every 8 Weeks (q8w) administrations through Week 72. At Week 24, participants will receive IV dosing of placebo.

Group Type: EXPERIMENTAL

Ustekinumab 6 mg/kg

Intervention Type: DRUG

Participants will receive body weight-range based IV dosing of 6 mg/kg of ustekinumab at Week 0 in Group 1 and at Week 24 in Group 2.

Ustekinumab 90 mg

Intervention Type: DRUG

Participants will receive ustekinumab 90 mg SC at Week 8 and every 8 Weeks (q8w) through Week 72 in Group 1 and q8w Week 32 through Week 72 in Group 2.

Placebo IV

Intervention Type: DRUG

Participants will receive IV dosing of placebo at Week 24 in Group 1 and at Week 0 in Group 2.

Group 2: Placebo

Participants will receive IV dosing of placebo at Week 0 followed by placebo SC administrations at Weeks 8,16 and 24. At Week 24, participants will receive body weight-range based IV dosing of approximately 6 mg/kg of ustekinumab, followed by ustekinumab 90 mg SC q8w administrations Week 32 through Week 72.

Group Type: PLACEBO_COMPARATOR

Ustekinumab 6 mg/kg

Intervention Type: DRUG

Participants will receive body weight-range based IV dosing of 6 mg/kg of ustekinumab at Week 0 in Group 1 and at Week 24 in Group 2.

Ustekinumab 90 mg

Intervention Type: DRUG

Participants will receive ustekinumab 90 mg SC at Week 8 and every 8 Weeks (q8w) through Week 72 in Group 1 and q8w Week 32 through Week 72 in Group 2.

Placebo IV

Intervention Type: DRUG

Participants will receive IV dosing of placebo at Week 24 in Group 1 and at Week 0 in Group 2.

Placebo SC

Intervention Type: DRUG

Participants will receive SC dosing of placebo at Weeks 8,16 and 24.

Interventions

Ustekinumab 6 mg/kg

Participants will receive body weight-range based IV dosing of 6 mg/kg of ustekinumab at Week 0 in Group 1 and at Week 24 in Group 2.

Intervention Type: DRUG

Ustekinumab 90 mg

Participants will receive ustekinumab 90 mg SC at Week 8 and every 8 Weeks (q8w) through Week 72 in Group 1 and q8w Week 32 through Week 72 in Group 2.

Intervention Type: DRUG

Placebo IV

Participants will receive IV dosing of placebo at Week 24 in Group 1 and at Week 0 in Group 2.

Intervention Type: DRUG

Placebo SC

Participants will receive SC dosing of placebo at Weeks 8,16 and 24.

Intervention Type: DRUG

Other Intervention Names

STELARA; STELARA

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Janssen Pharmaceutical K.K.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Janssen Pharmaceutical K.K., Japan Clinical Trial

Role: STUDY_DIRECTOR

Janssen Pharmaceutical K.K.

Locations

Tokyo Medical and Dental University Hospital

Bunkyō City, , Japan

Fukushima Medical University Hospital

Fukushima, , Japan

Shinko Hospital

Hyōgo, , Japan

Tokai University Hospital

Isehara, , Japan

Kagoshima University Hospital

Kagoshima, , Japan

St Marianna University Hospital

Kanagawa, , Japan

National Hospital Organization Osaka Minami Medical Center

Kawachi-Nagano, , Japan

Hospital of the University of Occupational and Enviromental Health

Kitakyushu, , Japan

Kumamoto University Hospital

Kumamoto, , Japan

Kurashiki Central Hospital

Kurashiki, , Japan

Shinshu University Hospital

Matsumoto, , Japan

Minaminagano Medical Center Shinonoi General Hospital

Nagano, , Japan

Nagasaki University Hospital

Nagasaki, , Japan

National Hospital Organization Nagoya Medical Center

Nagoya, , Japan

Niigata University Medical And Dental Hospital

Niigata, , Japan

Okayama City General Medical Center Okayama City Hospital

Okayama, , Japan

Saga University Hospital

Saga, , Japan

Kitasato University Hospital

Sagamihara, , Japan

Sakai City Medical Center

Sakai, , Japan

Tohoku University Hospital

Sendai, , Japan

Tohoku Medical And Pharmaceutical University Hospital

Sendai, , Japan

Dokkyo Medical University Hospital

Shimotsuga Gun, , Japan

Center Hospital of the National Center for Global Health and Medicine

Shinjuku Ku, , Japan

The Jikei University Hospital

Tokyo, , Japan

Nippon Medical School Hospital

Tokyo, , Japan

Juntendo University Hospital

Tokyo, , Japan

Tokyo Medical University Hospital

Tokyo, , Japan

Fujita Health University Hospital

Toyoake, , Japan

Ehime University Hospital

Tōon, , Japan

University of Tsukuba Hospital

Tsukuba, , Japan

Yamaguchi University Hospital

Ube, , Japan

Yokohama Rosai Hospital

Yokohama, , Japan

Countries

Japan

References

Kawahata K, Ishii T, Gono T, Tsuchiya Y, Ohashi H, Yoshizawa K, Zheng R, Ayabe M, Nishikawa K. Phase 3, multicentre, randomised, double-blind, placebo-controlled, parallel-group study of ustekinumab in Japanese patients with active polymyositis and dermatomyositis who have not adequately responded to one or more standard-of-care treatments. RMD Open. 2023 Aug;9(3):e003268. doi: 10.1136/rmdopen-2023-003268.

Reference Type: DERIVED

PMID: 37652554 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

CNTO1275DMY3001

Identifier Type: OTHER

Identifier Source: secondary_id

CR108618

Identifier Type: -

Identifier Source: org_study_id