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

NCT03981744 | Terminated Phase 3 Results DMC

• 2 other identifiers: CR108618CNTO1275DMY3001
• Study Type: interventional
• Target: 51
• Locations: 1 country
• Sites: 32

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

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants Who Achieved Minimal Improvement in International Myositis Assessment and Clinical Studies Total Improvement Score (IMACS TIS) at Week 24

  Minimal improvement was defined as IMACS TIS greater than or equal to (\>=) 20 in participants with polymyositis (PM)/dermatomyositis (DM). Criteria used the 6 IMACS core set measures: physician global activity (PhGA)(0-10), patient global activity (PtGA)(0-10), manual muscle testing-8 (MMT-8)(0-80), muscle enzymes (creatine kinase, Aldolase, lactate dehydrogenase, aspartate aminotransferase, alanine aminotransferase), extramuscular assessment of myositis disease activity assessment tool (MDAAT)(0-10), and health assessment questionnaire disability index (HAQ-DI)(0-3). Absolute percent change in each core set measure was calculated as final value minus baseline value divided by range\*100. Total improvement score was calculated by sum of 6 core set improvement scores. Total improvement score ranged from 0 to 100 where higher scores indicated greater improvement. This was categorized into 3 categories (minimal \[improvement \>=20\], moderate \[improvement \>=40\] and major \[improvement \>=60\]).

  Week 24

Secondary Outcomes (6)

• Change From Baseline in Functional Index-2 (FI-2) at Week 24

  Baseline, Week 24

• Percentage of Participants Who Experienced Disease Worsening Up to Week 24 Based on Consensus Criteria for Worsening

  Up to Week 24

• Change From Baseline in Manual Muscle Testing (MMT)-8 Score at Week 24

  Baseline, Week 24

• Change From Baseline in Physician Global Activity (PhGA) at Week 24

  Baseline, Week 24

• Change From Baseline in Extramuscular Assessment by Myositis Disease Activity Assessment Tool (MDAAT) at Week 24

  Baseline, Week 24

Study Arms (2)

Group 1: Ustekinumab

EXPERIMENTAL

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.

Drug: Ustekinumab 6 mg/kg; Drug: Ustekinumab 90 mg; Drug: Placebo IV

Group 2: Placebo

PLACEBO COMPARATOR

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.

Drug: Ustekinumab 6 mg/kg; Drug: Ustekinumab 90 mg; Drug: Placebo IV; Drug: Placebo SC

Interventions

Ustekinumab 6 mg/kg 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.

Also known as: STELARA

Group 1: Ustekinumab; Group 2: Placebo

Ustekinumab 90 mg 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.

Also known as: STELARA

Group 1: Ustekinumab; Group 2: Placebo

Placebo IV DRUG

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

Group 1: Ustekinumab; Group 2: Placebo

Placebo SC DRUG

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

Group 2: Placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Janssen Pharmaceutical K.K.: lead

Study Sites (32)

Tokyo Medical and Dental University Hospital

Bunkyō City, 113 8519, Japan

Location

Fukushima Medical University Hospital

Fukushima, 960 1295, Japan

Location

Shinko Hospital

Hyōgo, 651-0072, Japan

Location

Tokai University Hospital

Isehara, 259-1193, Japan

Location

Kagoshima University Hospital

Kagoshima, 890-8544, Japan

Location

St Marianna University Hospital

Kanagawa, 216 8511, Japan

Location

National Hospital Organization Osaka Minami Medical Center

Kawachi-Nagano, 586 8521, Japan

Location

Hospital of the University of Occupational and Enviromental Health

Kitakyushu, 807-8555, Japan

Location

Kumamoto University Hospital

Kumamoto, 860-8556, Japan

Location

Kurashiki Central Hospital

Kurashiki, 710-8602, Japan

Location

Shinshu University Hospital

Matsumoto, 390-8621, Japan

Location

Minaminagano Medical Center Shinonoi General Hospital

Nagano, 388-8004, Japan

Location

Nagasaki University Hospital

Nagasaki, 852-8501, Japan

Location

National Hospital Organization Nagoya Medical Center

Nagoya, 460-0001, Japan

Location

Niigata University Medical And Dental Hospital

Niigata, 951 8520, Japan

Location

Okayama City General Medical Center Okayama City Hospital

Okayama, 700-8557, Japan

Location

Saga University Hospital

Saga, 849-8501, Japan

Location

Kitasato University Hospital

Sagamihara, 252-0375, Japan

Location

Sakai City Medical Center

Sakai, 593-8304, Japan

Location

Tohoku University Hospital

Sendai, 980 8574, Japan

Location

Tohoku Medical And Pharmaceutical University Hospital

Sendai, 983-8512, Japan

Location

Dokkyo Medical University Hospital

Shimotsuga Gun, 321 0293, Japan

Location

Center Hospital of the National Center for Global Health and Medicine

Shinjuku Ku, 162 8655, Japan

Location

The Jikei University Hospital

Tokyo, 105 8471, Japan

Location

Nippon Medical School Hospital

Tokyo, 113 8603, Japan

Location

Juntendo University Hospital

Tokyo, 113-8431, Japan

Location

Tokyo Medical University Hospital

Tokyo, 160-0023, Japan

Location

Fujita Health University Hospital

Toyoake, 470-1192, Japan

Location

Ehime University Hospital

Tōon, 791-0295, Japan

Location

University of Tsukuba Hospital

Tsukuba, 305 8576, Japan

Location

Yamaguchi University Hospital

Ube, 755-8505, Japan

Location

Yokohama Rosai Hospital

Yokohama, 222-0036, Japan

Location

Related Publications (1)

• 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.

  PMID: 37652554 DERIVED

MeSH Terms

Conditions

Polymyositis; Dermatomyositis

Interventions

Ustekinumab

Condition Hierarchy (Ancestors)

Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Limitations and Caveats

This study was terminated early due to lack of efficacy found at Week 24.

Results Point of Contact

• Title: PRODUCT DEVELOPMENT PORTFOLIO LEADER
• Organization: Janssen Pharmaceutical K.K.

ClinicalTrialDisclosure@its.jnj.com

844-434-4210

Study Officials

• Janssen Pharmaceutical K.K., Japan Clinical Trial

  Janssen Pharmaceutical K.K.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 7, 2019
• First Posted: June 11, 2019
• Study Start: July 26, 2019
• Primary Completion: January 24, 2022
• Study Completion: July 12, 2022
• Last Updated: April 29, 2025
• Results First Posted: May 13, 2024

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will share

Locations

JP (32)