NCT07377058招募中不适用
A Randomized, Double-Blind, Placebo-Controlled Clinical Study on the Efficacy and Safety of Tocilizumab in the Treatment of Anti-MDA5 Antibody-Positive Adult Dermatomyositis
适应症
干预措施
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 110
- 试验地点
- 1
- 主要终点
- TIS improvement
研究概览
简要总结
The goal of this clinical trial is to learn if tocilizumab works to treat anti-MDA5+ dermatomyositis (anti-MDA5+DM) in adults. It will also learn about the safety of tocilizumab. The main questions it aims to answer are:
Does tocilizumab improve patients' clinical symptoms? Does tocilizumab improve patients' respiratory failure? What medical problems do participants have when taking tocilizumab? Researchers will compare tocilizumab to a placebo (a look-alike substance that contains no drug) to see if tocilizumab works to treat patients with anti-MDA5+ DM.
Participants will:
Take tocilizumab or a placebo every two weeks for 2 months Visit the clinic once every 2 weeks for checkups and tests
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All inclusion criteria must be met for eligibility:
- •Age ≥ 18 years and < 65 years; no gender restriction; total body weight ≥ 45 kg;
- •Diagnostic Criteria for Anti-MDA5-DM: Refer to the "2023 Chinese Expert Consensus on Diagnosis and Treatment of Anti-Melanoma Differentiation-Related Gene 5 Antibody-Positive Dermatomyositis in China." Dermatomyositis patients exhibiting one of the following manifestations-Gottron's papules, Gottron's rash, or erythema ab igne-along with positive serum anti-MDA5 antibodies, may be diagnosed with anti-MDA5-DM;
- •If patients have concomitant ILD, the following conditions must be met: i) Pulse oxygen saturation (SpO₂) ≥ 90% or PaO₂ ≥ 60 mmHg; ii) Pulmonary function tests showing forced vital capacity percentage of predicted (FVC%) ≥ 60% and carbon monoxide diffusion capacity percentage of predicted (DLco%) ≥ 40%; iii) High-resolution chest CT demonstrating pulmonary interstitial lesions involving < 50% of lung fields;
- •Patients must have received oral prednisone (< 1 mg/kg/day, or equivalent dose of other glucocorticoids) for ≥ 4 weeks prior to randomization;
- •Patients must have received a stable dose of a calcineurin inhibitor (CNI, such as cyclosporine or tacrolimus) for ≥4 weeks prior to randomization; if immunosuppressive therapy was discontinued prior to the screening visit, a washout period of at least 4 weeks is required;
- •Patients must receive prophylactic treatment with trimethoprim-sulfamethoxazole (TMP-SMZ, 400mg trimethoprim/80mg sulfamethoxazole) 1-2 tablets daily during treatment;
- •Women of childbearing potential must have a negative pregnancy test at study entry. If sexually active, they must agree to use effective contraception throughout the study period and must not intend to become pregnant during the study.
- •Patients voluntarily participate in this study and sign an informed consent form.
排除标准
- •1. Polymyositis, anti-synthetase syndrome, immune-mediated necrotizing myositis, or overlap myositis with other connective tissue diseases;
- •Patients with life-threatening complications, including but not limited to acute coronary syndrome (e.g., myocardial infarction, unstable angina) within 24 weeks prior to screening or any history of significant cerebrovascular disease;
- •Any of the following laboratory abnormalities at screening: white blood cell count <3.0×10⁹/L, neutrophil count <1.0×10⁹/L, lymphocyte count <0.5×10⁹/L, hemoglobin <90 g/L, platelet count <50×10⁹/L; severe hepatic impairment (ALT or AST ≥3 times ULN, total bilirubin ≥1.5 times ULN, excluding serum ALT or AST elevation due to dermatomyositis); severe renal impairment (creatinine clearance ≤45 mL/min);
- •Patients hospitalized for severe infection within 60 days prior to screening, or who received intravenous antibiotics (patients who used intravenous antibiotics must complete a five-half-life washout period and confirm absence of active infection before enrollment), but may receive empirical oral antibiotics or topical antibiotics;
- •Active tuberculosis infection that is untreated or inadequately treated; Latent tuberculosis infection (LTBI) requires at least 2 weeks of preventive antituberculosis therapy (including at least 2 antituberculosis drugs) prior to randomization, continuing through study completion. LTBI is defined as: Positive IGRA result (acceptable IGRA assays include: QFT-GIT, QFT-G, and T-spot® TB test);
- •Active viral hepatitis at screening: HBsAg-positive, HBeAg-positive, or HBV-DNA >10³ copies/L (HBV-DNA testing required if HBcAb-positive); HCVAb-positive;
- •Documented HIV infection, evidenced by positive serological test results or positive HIV serological test results at screening;
- •If the patient develops ILD-related clinical manifestations or progressive radiographic worsening within 4 weeks, RP-ILD should be considered. RP-ILD is defined as the presence of any one of the following four conditions within 1 month after the onset of respiratory symptoms: ① Acute and progressive worsening of dyspnea requiring hospitalization or supplemental oxygen; ② Decline in pulmonary function, manifested as a decrease in FVC% >10% with or without a decrease in DLco% >15%; ③ Increased interstitial abnormalities on chest HRCT scan >20%; ④ Decrease in arterial blood gas or partial pressure of oxygen >10 mmHg, indicating respiratory failure; and PaO₂/FiO₂≥200 mmHg.
- •9. Allergy to the active ingredient tocilizumab or any of its excipients;
- •Patients with sulfonamide allergy;
- •Patients unable to complete pulmonary function testing at baseline;
- •Patients receiving prednisone at a dose exceeding 2 mg/kg/day prior to screening;
- •Patients receiving intravenous immunoglobulin (IVIG) prior to screening must discontinue treatment for at least 30 days;
- •Patients who used one or more of the following medications within the specified time window prior to screening:
- •Rituximab within 6 months prior to screening;
- •JAK inhibitors within 2 weeks prior to screening;
- •Use of other biologics (including but not limited to anakinra, adalimumab, infliximab) or other immunosuppressive agents (including but not limited to methotrexate, azathioprine, mycophenolate mofetil) within 4 weeks prior to screening;
- •Patients with prior use of the study drug, other IL-6 inhibitors, or analogues;
- •Pregnant or lactating women, or women planning to become pregnant or initiate lactation;
- •History of malignant tumors within the past 5 years (excluding adequately treated basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or cervical carcinoma in situ with no evidence of recurrence within the preceding 5 years);
- •Other conditions deemed ineligible for study inclusion by the investigator.
研究组 & 干预措施
Placebo
Placebo Comparator
干预措施: Standard medical treatment (Drug)
TCZ
Experimental
干预措施: Tocilizumab (Biological)
结局指标
主要结局
TIS improvement
时间窗: at Week 16 of treatment
Proportion of patients achieving minimal improvement in Total Improvement Score (TIS≥20)
ILD improvement
时间窗: at Week 16
Change in PaO2/FiO2 ratio from baseline (patients with concomitant ILD)
次要结局
未报告次要终点
研究者
Wang Qian
Prof
Peking Union Medical College Hospital
研究点 (1)
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