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

To evaluate the efficacy of nemolizumab in systemic sclerosis patients. To evaluate also the safety and pharmacokinetics.

Study Overview

• Status: Completed
• Conditions: Systemic Sclerosis
• Intervention / Treatment: Drug: nemolizumab

• Study Type: Interventional
• Enrollment (Actual): 6
• Phase: Phase 2

Contacts and Locations

Study Locations

• Japan
  • Tokyo, Japan
    • The University of Tokyo Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 20 years to 70 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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 Sclerosis
• Patients with body weight less than 30.0kg
• Pregnant or lactating women.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: nemolizumab	Drug: nemolizumab; nemolizumab will be administered subcutaneous injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in Modified Rodnan Total Skin Thickness Score (Total mRSS) at Week 24	Compare pre- and post-treatment total mRSS at Week 24. The investigator assessed the degree of skin thickening for each of 17 sites (fingers of both hands, dorsum of both hands, both forearms, both upper arms, face, anterior chest, abdomen, both thighs, both lower legs, and dorsum of both feet) with a 4-point scale from 0 to 3; where 0 = normal skin, 1 = mild thickness, 2 = moderate thickness, and 3 = severe thickness. The total mRSS is the sum of the mRSS of 17 site	Baseline and week 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in Modified Rodnan Total Skin Thickness Score (Total mRSS) at Week 52	Compare pre- and post-treatment total mRSS at Week 52. The investigator assessed the degree of skin thickening for each of 17 sites (fingers of both hands, dorsum of both hands, both forearms, both upper arms, face, anterior chest, abdomen, both thighs, both lower legs, and dorsum of both feet) with a 4-point scale from 0 to 3; where 0 = normal skin, 1 = mild thickness, 2 = moderate thickness, and 3 = severe thickness. The total mRSS is the sum of the mRSS of 17 site	From baseline up to Week 52
Change From Baseline in Health Assessment Questionnaire Disability Index (HAQ-DI) at Week 24	Compare pre- and post-treatment HAQ-DI at Week 24. The HAQ is a questionnaire that patients with chronic diseases answer themselves to assess physical function related to activities of daily living. The questionnaire consists of 20 questions divided into eight categories (dressing and grooming, standing, eating, walking, hygiene, movement, grip strength, and others), which are rated on a four-point scale from 0 to 3. 0 = without any difficulty, 1 = with some difficulty, 2 = with much difficulty, and 3 = unable to do. The highest value in each category is the index for that category, and the average of these is the HAQ-DI. The higher the HAQ-DI, the greater the degree of functional impairment.	From baseline up to Week 24
Change From Baseline in Health Assessment Questionnaire Disability Index (HAQ-DI) at Week 52	Compare pre- and post-treatment HAQ-DI at Week 52. The HAQ is a questionnaire that patients with chronic diseases answer themselves to assess physical function related to activities of daily living. The questionnaire consists of 20 questions divided into eight categories (dressing and grooming, standing, eating, walking, hygiene, movement, grip strength, and others), which are rated on a four-point scale from 0 to 3. 0 = without any difficulty, 1 = with some difficulty, 2 = with much difficulty, and 3 = unable to do. The highest value in each category is the index for that category, and the average of these is the HAQ-DI. The higher the HAQ-DI, the greater the degree of functional impairment.	From baseline up to Week 52
Change From Baseline in Percent Forced Vital Capacity (% FVC) at Week 24	Compare pre- and post-treatment %FVC at Week 24. %FVC is the ratio of the actual measured FVC to the predicted FVC (predicted based on gender, age, and height).	From baseline up to Week 24
Change From Baseline in Percent Forced Vital Capacity (% FVC) at Week 52	Compare pre- and post-treatment %FVC at Week 52. %FVC is the ratio of the actual measured FVC to the predicted FVC (predicted based on gender, age, and height).	From baseline up to Week 52
Change From Baseline in Percent Diffusing Capacity of Lung for Carbon Monoxide (%DLco) at Week 24	Compare pre- and post-treatment %DLco at Week 24. Pulmonary diffusion capacity refers to the ability to supply gases such as oxygen from alveoli to the alveolar capillaries. DLco is measured for carbon monoxide (CO) and is expressed as the volume (mL) of CO that transfers from alveolar air to the blood per minute per 1mmHg of alveolar partial pressure. %DLco is the ratio of the actual measured DLco to the predicted DLco (predicated based on age, sex, and height)	From baseline up to Week 24
Change From Baseline in Percent Diffusing Capacity of Lung for Carbon Monoxide (%DLco) at Week 52	Compare pre- and post-treatment %DLco at Week 52. Pulmonary diffusion capacity refers to the ability to supply gases such as oxygen from alveoli to the alveolar capillaries. DLco is measured for carbon monoxide (CO) and is expressed as the volume (mL) of CO that transfers from alveolar air to the blood per minute per 1mmHg of alveolar partial pressure. %DLco is the ratio of the actual measured DLco to the predicted DLco (predicated based on age, sex, and height)	From baseline up to Week 52
Change From Baseline in Physician's Global Assessment at Week 24	Compare pre- and post-treatment physician's global assessment at Week 24. Physician's global assessment is a visual analogue scale in which the attending physician assess the condition of systemic sclerosis based on severity, damage, and overall disease status. 0 is the best condition and 10 is the worst condition.	From baseline up to Week 24
Change From Baseline in Physician's Global Assessment at Week 52	Compare pre- and post-treatment physician's global assessment at Week 52. Physician's global assessment is a visual analogue scale in which the attending physician assess the condition of systemic sclerosis based on severity, damage, and overall disease status. 0 is the best condition and 10 is the worst condition.	From baseline up to Week 52
Change From Baseline in Patient's Global Assessment at Week 24	Compare pre- and post-treatment patient's global assessment at Week 24. Patient's Global Assessment is a visual analogue scale in which patients rate their systemic sclerosis condition, with 0 being the best and 10 being the worst.	From baseline up to Week 24
Change From Baseline in Patient's Global Assessment at Week 52	Compare pre- and post-treatment patient's global assessment at Week 52. Patient's Global Assessment is a visual analogue scale in which patients rate their systemic sclerosis condition, with 0 being the best and 10 being the worst.	From baseline up to Week 52

Collaborators and Investigators

• Sponsor: Maruho Co., Ltd.
• Investigators: Principal Investigator: Ayumi Yoshizaki, MD, PhD, Tokyo University

Study record dates

Study Major Dates

• Study Start (Actual): April 20, 2022
• Primary Completion (Actual): June 4, 2024
• Study Completion (Actual): July 30, 2024

Study Registration Dates

• First Submitted: January 17, 2022
• First Submitted That Met QC Criteria: January 17, 2022
• First Posted (Actual): January 31, 2022

Study Record Updates

• Last Update Posted (Actual): February 13, 2026
• Last Update Submitted That Met QC Criteria: January 27, 2026
• Last Verified: November 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Connective Tissue Diseases; Skin Diseases; Skin and Connective Tissue Diseases; Scleroderma, Systemic; nemolizumab
• Other Study ID Numbers: M525101-21

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No