Systemic Scleroderma
Conditions
Keywords
Systemic Scleroderma, SSc
Brief summary
This study is a single-center, single-arm, open-label, exploratory clinical trial. A total of 30 patients with diffuse cutaneous systemic sclerosis (dcSSc) will be enrolled. A historical control cohort will be established to evaluate the efficacy and safety of Firsekibart by comparing with historical data.
Interventions
DRUGFirsekibart injection
Firsekibart, independently developed by GeneScience, was officially approved for marketing by the NMPA in July 2025 as China's first domestically developed fully human monoclonal antibody targeting IL-1β.
Sponsors
Tongji Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
1. Age 18-70 years (inclusive), male or female. 2. Diagnosis of systemic sclerosis (SSc) according to the 2013 ACR/EULAR diagnostic criteria. 3. Disease duration of diffuse cutaneous systemic sclerosis (dcSSc), as defined by LeRoy \& Medsger (2001), of ≤ 5 years (from the time of first onset of non-Raynaud's phenomenon). 4. Modified Rodnan skin score (mRSS) ≥10; 5. Voluntarily signed informed consent form and ability to comply with the requirements of the study protocol.
Exclusion criteria
1. Allergy to the active ingredient of Firsekibart or any of its excipients, or a history of allergy to monoclonal antibodies. 2. Presence of any rheumatic disease other than SSc. 3. Moderate to severe lung disease with FVC \< 60% or DLCO \< 50% of predicted value. 4. Use of medications that may interfere with the evaluation of the efficacy and safety of Firsekibart, except for stable use of permitted concomitant therapies that have been maintained for at least 4 weeks prior to screening and are kept at a stable dose throughout the study period. 5. Use of biological agents or stem cell therapy within 3 months prior to screening or within 5 half-lives of the known drug. 6. Receipt of live or attenuated vaccines within two months prior to screening. 7. Severe hepatic impairment, renal impairment, or hematologic abnormalities at screening. 8. Acute or chronic infection (excluding infection complicated by finger ulceration), active infection, history of malignant tumor, or immunodeficiency disorder. 9. Women who are pregnant or breastfeeding, or subjects planning to become pregnant during the study period. 10. Any other conditions that, in the investigator's judgment, render the subject ineligible for this trial.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in the modified Rodnan Skin Score (mRSS) from baseline | Week 12 | The mRSS is independently assessed by two physicians, evaluating the thickness of skin in 17 anatomic areas rated from 0 to 3, with a total score ranging from 0 to 51. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in modified Rodnan skin score (mRSS) from baseline | Week 16, 24 | The mRSS is independently assessed by two physicians, evaluating the thickness of skin in 17 anatomic areas rated from 0 to 3, with a total score ranging from 0 to 51. |
| Change in the Composite Response Index in Systemic Sclerosis (CRISS) from baseline | Week 12, 16, 24 | CRISS is a weighted score and includes five core set measures: modified Rodnan skin score, FVC% predicted, health assessment questionnaire-disability index, and patient and clinician global assessments. |
| Change from baseline in pulmonary function (FVC) | Week 12, 16, 24 | Forced vital capacity (FVC) is the amount of air that can be forcibly exhaled after the deepest possible breath. |
| Change from baseline in pulmonary function (DLCO) | Week 12, 16, 24 | Diffusing capacity of the lungs for carbon monoxide (DLCO)is a key measure of gas diffusion across the alveolar-capillary membrane, determined by the single-breath method. |
| Change in serum IL-1β levels from baseline | Week 12, 16, 24 | IL-1β plays an important role in inflammation and fibrosis, and its expression is aberrantly regulated in various autoimmune diseases. IL-1β is considered an effective target for diseases associated with fibrosis and tissue remodeling. |
| Change in serum IL-6 levels from baseline | Week 12, 16, 24 | IL-1β acts as a potent upstream stimulus for IL-6 production. IL-6 activates fibroblasts, promoting their proliferation and driving the abundant synthesis of collagen and extracellular matrix components. In parallel, IL-6 induces endothelial-to-mesenchymal transition in endothelial cells, thereby exacerbating the vicious cycle between microvascular pathology and fibrosis. |
Countries
China
Contacts
CONTACTLingli Dong, Professor
Outcome results
None listed