Systemic Sclerosis
Conditions
Brief summary
To evaluate the efficacy of nemolizumab in systemic sclerosis patients. To evaluate also the safety and pharmacokinetics.
Interventions
DRUGnemolizumab
nemolizumab will be administered subcutaneous injection
Sponsors
Maruho Co., Ltd.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
20 Years to 70 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Modified Rodnan Total Skin Thickness Score (Total mRSS) at Week 24 | Baseline and 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Modified Rodnan Total Skin Thickness Score (Total mRSS) at Week 52 | From baseline up to 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 |
| Change From Baseline in Health Assessment Questionnaire Disability Index (HAQ-DI) at Week 24 | From baseline up to 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. |
| Change From Baseline in Health Assessment Questionnaire Disability Index (HAQ-DI) at Week 52 | From baseline up to 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. |
| Change From Baseline in Percent Forced Vital Capacity (% FVC) at Week 24 | From baseline up to 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). |
| Change From Baseline in Percent Forced Vital Capacity (% FVC) at Week 52 | From baseline up to 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). |
| Change From Baseline in Percent Diffusing Capacity of Lung for Carbon Monoxide (%DLco) at Week 24 | From baseline up to 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) |
| Change From Baseline in Percent Diffusing Capacity of Lung for Carbon Monoxide (%DLco) at Week 52 | From baseline up to 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) |
| Change From Baseline in Physician's Global Assessment at Week 24 | From baseline up to 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. |
| Change From Baseline in Physician's Global Assessment at Week 52 | From baseline up to 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. |
| Change From Baseline in Patient's Global Assessment at Week 24 | From baseline up to 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. |
| Change From Baseline in Patient's Global Assessment at Week 52 | From baseline up to 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. |
Countries
Japan
Contacts
PRINCIPAL_INVESTIGATORAyumi Yoshizaki, MD, PhD
Tokyo University
Participant flow
Recruitment details
The study was conducted at one site in Japan from April 2022 to July 2024
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 1 Participants |
| Age, Categorical Between 18 and 65 years | 5 Participants |
| Age, Continuous | 53.5 year |
| Classification of Primary Disease Diffuse Cutaneous SSc | 4 Participants |
| Classification of Primary Disease Limited Cutaneous SSc | 2 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 6 Participants |
| Modified Rodnan Total Skin Thickness Score (Total mRSS) | 16 scores on scale |
| percent diffusing capacity of lung for carbon monoxide (% DLco) | 101.75 percentage of actual to predicted DLco |
| percent forced vital capacity (%FVC) | 99.7 percentage of actual to predicted FCV |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 6 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 0 Participants |
| Region of Enrollment Japan | 6 participants |
| Sex: Female, Male Female | 6 Participants |
| Sex: Female, Male Male | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 6 |
| other Total, other adverse events | 6 / 6 |
| serious Total, serious adverse events | 0 / 6 |
Outcome results
None listed