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Clinical Trial
. 2023 Aug;9(3):e003268.
doi: 10.1136/rmdopen-2023-003268.

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

Kimito Kawahata  1 , Tomonori Ishii  2 , Takahisa Gono  3 , Yumi Tsuchiya  4 , Hiroki Ohashi  5 , Katsunori Yoshizawa  5 , Richuan Zheng  6 , Maori Ayabe  7 , Kazuko Nishikawa  5
Affiliations
Clinical Trial

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

Kimito Kawahata et al. RMD Open. 2023 Aug.

Abstract

Objectives: To evaluate the efficacy and safety of ustekinumab (UST) in a multicentre, randomised, double-blind, placebo-controlled trial in adult Japanese patients with active polymyositis (PM) and dermatomyositis (DM).

Methods: Fifty-one Japanese adults diagnosed with active PM/DM who did not respond adequately to one or more standard-of-care treatments were randomised 1:1 to receive UST (n=25) or placebo (n=26). Participants received body weight-range based intravenous administration of UST (6 mg/kg) or placebo at week 0 followed by 90 mg subcutaneous (SC) administration of UST or placebo every 8 weeks from week 8 to week 24. At week 24, placebo group crossed over to receive body weight-range based intravenous administration of UST, and thereafter, all participants received/were to receive SC administration of UST 90 mg every 8 weeks (week 32 through to week 72). The primary efficacy endpoint was the proportion of participants who achieved minimal improvement (≥20) in the International Myositis Assessment and Clinical Studies Total Improvement Score (IMACS TIS) at week 24.

Results: No statistically significant difference was seen in the proportion of participants who achieved minimal improvement (≥20) in IMACS TIS at week 24 between the treatment groups (UST 64.0% vs placebo 61.5%, p=0.94) based on the primary estimand of the primary endpoint analysis.

Conclusions: UST was safe and well tolerated but did not meet the primary efficacy endpoint in adult Japanese participants with active PM/DM based on the primary analysis at week 24 in the study.

Trial registration number: NCT03981744.

Keywords: dermatomyositis; inflammation; polymyositis.

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Conflict of interest statement

Competing interests: KK has received consulting fee as medical advisor, honoraria/ speaking fees and support for conducting clinical studies (paid to the hospital) from Janssen. TI has received honoraria/ speaking fees from Asahi Kasei, Astellas, Boehringer Ingelheim, Janssen, and Ono Pharmaceuticals and support for conducting clinical studies (paid to the hospital) from Janssen. TG has received honoraria/speaking fee from Asahi Kasei, Astellas, Boehringer Ingelheim, Bristol-Myers Squibb, Chugai, Eisai, Janssen, MBL, Nippon Shinyaku, Pfizer, and Ono Pharmaceuticals and support for conducting clinical studies (paid to the hospital) from Janssen. YT, HO, RZ, MA and KN are employees of Janssen Pharmaceuticals. HO and KY are Janssen Pharmaceuticals employees and shareholders of Johnson & Johnson.

Figures

Figure 1
Figure 1
Study design. DBL, database lock; GC, glucocorticoids; IMACS TIS, International Myositis Assessment and Clinical Studies Group Total Improvement Score; IV, intravenous; q8w, every 8 weeks; SC, subcutaneous. (R): randomisation; white broken arrow:~6mg/kg IV load; black arrow: sc study agent administration.
Figure 2
Figure 2
Consort diagram.
Figure 3
Figure 3
Primary endpoint: proportion of subjects who achieved minimal improvement (≥20) in IMACS TIS at week 24 and subgroup analysis: proportion of subjects who achieved minimal improvement (≥20) in IMACS TIS at week 24 by disease type.
Figure 4
Figure 4
Proportion of subjects achieving minimal improvement (≥20) in IMACS TIS over time through week 24.
Figure 5
Figure 5
Post-hoc analysis: summary of IMACS TIS over time at week 24.
Figure 6
Figure 6
Proportion of subjects who achieved moderate (≥40) or major (≥60) improvement in IMACS TIS at week 24.
See this image and copyright information in PMC

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