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An Open-label, Phase 2 Study to Assess the Efficacy and Safety of Nemolizumab in Subjects with Systemic Sclerosis

An Open-label, Phase 2 Study to Assess the Efficacy and Safety of Nemolizumab in Subjects with Systemic Sclerosis

6

Patient demographics and background who was administered the investigational drug: - Gender: 6 females - Age range: 50 to 66 years - Weight (SD): 54.13 (9.52) kg - Race: 6 Asian - Classification of Primary Disease: diffuse cutaneous systemic sclerosis :4, limited cutaneous systemic sclerosis:2 - Median modified rodman total skin thickness score (mRSS) - (range): 16.0 (12 - 22) - Median percent-predicted forced vital capacity (%FVC) (range): 97.70% (82.7% - 109.0%) - Median percent diffusing capacity of the lung for carbon monoxide (%DLCO) (range): 101.75% (83.2% - 109.2%)

- Consent obtained: 7 - Enrolled: 6 - Investigational drug (IP) administered: 6 - Study completed: 6 Reasons for discontinuation: Withdrawal by subject.

Adverse events (AEs) were observed in 6 patients with 19 events. All of these AEs were mild in severity, and all resolved, with the exception of one event of malaise. Furthermore, all AEs were deemed unrelated to the IP. No death, other serious AEs and AEs leading to discontinuation, interruption, or dose reduction of the IP were observed. AEs of special interest were 3 events of skin infections (one event each of oral herpes, tinea pedis, and paronychia) in 3 patients, and 7 events of non-skin infections (nasopharyngitis in 3 patients with 4 events, and one event each of gastroenteritis, adenoviral conjunctivitis, and COVID-19) in 4 patients. There were no AEs related to clinical laboratory tests, and clinically significant changes in each test parameter were not observed after the administration of the IP.

[Primary outcome] The median change (range) in mRSS from baseline at Week 24 was -9.5 (-13 to -6). [Secondary outcome] - The median change (range) in mRSS from baseline at Week 52 was -10.0 (-14 to -7). - The median change (range) in Health Assessment Questionnaire Disability Index from baseline was 0.000 (-0.13 to 0.38) at Week 24, and 0.063 (-0.13 to 0.25) at Week 52, indicating no significant change in health assessment. - The median change (range) in %FVC from baseline at Week 24 Week 52 was -1.30% (-3.5% to 5.7%) and -0.35% (-6.4% to 5.7%), respectively, indicating no significant change. - The median change (range) in %DLCO from baseline at Week 24 and Week 52 was 0.55% (-4.5% to 8.8%) and -3.00% (-6.6% to 4.4%), respectively, indicating no significant change. - The median change (range) in physician's global assessment (PGA) score from baseline was -3.30 (-5.4 to -0.9) at Week 24 and -3.10 (-6.5 to -0.6) at Week 52, indicating a trend towards improvement in the overall condition of SSc as assessed by the Investigator or sub-investigator. - The median change (range) in patient's global assessment (PtGA) score from baseline was 1.30 (-1.8 to 3.8) at Week 24, and 1.70 (-0.1 to 5.1) at Week 52, indicating no change in the patients' assessment of their overall condition of SSc.

The median change in mRSS from baseline at Week 24 was -9.5 (-13 to -6), confirming an improvement. In the overall improvement assessment, improvement was observed in the PGA, but not in the PtGA. No changes were observed in other assessment items. Thus, the efficacy of the IP for SSc was not clear. AEs were observed in 6 patients with 19 events. All were mild and not related to the IP, indicating no safety concerns.

No

https://jrct.mhlw.go.jp/latest-detail/jRCT2031210481

Nishiura Tomoyuki

Maruho Co.,Ltd. Kyoto R&D Center

93 chudoji Awatacho, Shimogyo-ku, Kyoto

ctinfo@mii.maruho.co.jp

Information Trials Clinical

Maruho Co.,Ltd. Kyoto R&D Center

93 chudoji Awatacho, Shimogyo-ku, Kyoto

+81-753253279

ctinfo@mii.maruho.co.jp

Complete

Dec. 12, 2021

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

-Diagnosis of Systemic Sclerosis according to American College of Rheumatology (ACR) and European League Against Rheumatism (EULAR) 2013 criteria
-Systemic Sclerosis patients with moderate to sever skin sclerosis

-Patients witg a disease considered inappropriate for participation in clinical trials, such as serious cardiac / hepatic / renal / pulmonary / hematologic disease
-Patients with a diseases that could interfere with assessment of Systemic Scleorosis
-Patients with body weight less than 30.0kg
-Pregnant or lactating women.

Both

Systemic Sclerosis

Nemolizumab will be administered subcutaneously

Change from Baseline in modified Rodnan Skin Score (mRSS) at Week 24

+81-3-5800-8743

Dec. 21, 2021

none