Systemic sclerosis
Conditions
Interventions
Dose expansion phase (Phase II) : Contrast group:basic treatment plus placebo
Dose escalation phase (Phase I) : high dose group:RY_SW01 cell injection 2.0×10^6 cells /kg treatment
Sponsors
Nanjing Drum Tower Hospital
Eligibility
Sex/Gender
All
Age
18 Years to 65 Years
Inclusion criteria
Inclusion criteria: 1.Voluntarily sign the informed consent form; 2.Aged between 18 and 65 years (inclusive), regardless of gender; 3.Diagnosed with systemic sclerosis (SSc) based on the 2013 American College of Rheumatology (ACR)/European League Against Rheumatism (EULAR) criteria for SSc; 4. Patients diagnosed with diffuse scleroderma during screening, with a disease duration of <=5 years (the onset of the disease is defined as the time of the first presentation of non-Raynaud's phenomenon); 5. Previously treated with two or more methods such as hormones, immunosuppressants, biological agents, with a skin score of 10 <= mRSS <= 30 points.
Exclusion criteria
Exclusion criteria: 1.At screening, subjects with a forced vital capacity (FVC) predicted percentage 25mmHg measured by right heart catheterization or had a systolic pulmonary artery pressure >45mmHg measured by echocardiography at screening; 3.Presence of clinical symptoms requiring hospitalization for one of the following conditions at screening, whether newly occurring or worsening of pre-existing symptoms within 6 months: myocardial infarction, stroke, renal crisis, severe uncontrolled hypertension (>=160/100mmHg); or within 3 months: unstable ischemic heart disease, uncontrolled arrhythmia, heart failure (New York Heart Association III/IV stage), left ventricular ejection fraction 3 times the upper limit of normal (ULN); Total bilirubin >3 times ULN; 3)Renal function abnormalities: Estimated glomerular filtration rate (eGFR) =5 cigarettes within the 3 months prior to screening. 2)Alcohol abuse defined as consuming more than 14 units of alcohol per week (1 unit of alcohol = 350ml of beer, or 45ml of spirits, or 150ml of wine) within the 3 months prior to screening. 3)Drug abuse defined as having a history of drug abuse; 13.Planning to conceive within at least 2 years after the last injection, or unwilling to take effective contraceptive measures with
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Dose escalation phase (Phase I) : incidence of DLT;Dose expansion phase (Phase II) : Incidence of adverse events at 24 weeks after infusion;Dose expansion phase (Phase II) : Change in mRSS from baseline at 24 weeks after infusion; | — |
Secondary
| Measure | Time frame |
|---|---|
| Dose expansion phase (Phase II) : Change in mRSS from baseline at 12 weeks after infusion;Dose expansion phase (Phase II) : Change in lung function (FVC predicted percentage) from baseline at 24 weeks after infusion;The proportion of subjects who failed treatment 24 weeks after infusion;The proportion of subjects with an improvement in mRSS from baseline at 24 weeks post-infusion;The change in HAQ-DI from baseline at 24 weeks after infusion;Change from baseline in the proportion of TNFR1, anti-Scl-70 antibody, IL-6, TGF-ß, VEGF, TNF-a and Th17 cell subsets in blood at 12 and 24 weeks after transfusion; | — |
Countries
China
Contacts
Public ContactSun Lingyun
Nanjing Drum Tower Hospital
Outcome results
None listed