Study of the Telitacicept in Pediatric Patients With Frequently Relapsing or Steroid Dependent Nephrotic Syndrome
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- 1-year relapse-free survival rate
研究概览
简要总结
The main objective is to evaluate the effectiveness of telitacicept in pediatric patients with frequently relapsing or steroid dependent nephrotic syndrome within the 52-week follow-up.
详细描述
Nephrotic syndrome(NS) is the most common glomerular disease in children. Approximately 45-50% of patients with nephrotic syndrome exhibit frequent relapses or are dependent on steroid therapy. Frequent relapses or steroid dependence in nephrotic syndrome have been challenging issues for clinicians. Long-term, repeated, and high-dose oral steroid use can lead to side effects such as obesity, delayed development, hypertension, diabetes, glaucoma, osteoporosis, and increased susceptibility to infections. The addition of traditional immunosuppressants such as cyclophosphamide and tacrolimus can cause severe and irreversible side effects. Therefore, exploring new drugs and their application protocols is particularly important. Telitacicept has a unique dual-target mechanism that can inhibit B cell maturation and differentiation at multiple stages, thereby inhibiting B cell activity. Clinical studies have confirmed its significant efficacy in various kidney diseases, such as lupus nephritis, IgA nephropathy, and adult recurrent minimal change nephrotic syndrome; moreover, it has good safety profiles.
Therefore, through this prospective, single-center, open-label clinical trial, we aim to evaluate whether telitacicept provides superior efficacy compared to existing conventional treatment regimens for childhood frequent relapse (FR) or steroid-dependent (SD) nephrotic syndrome (NS), and assess its safety profile. Our goal is to provide an optimized treatment plan for childhood FRNS or SDNS.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Sensitive but frequent relapses or steroids dependence nephrotic syndrome
- •Age: 2 to 18 years old
- •Normal renal function: estimated glomerular filtration rate ≥90ml/ min/1.73m2
- •Morning urine protein <1+ or urine protein-creatinine ratio <0.2g/g (<20 mg/ mmol) for 3 consecutive days and above when in enroll
- •No rituximab was used within 6 months, no tacrolimus, mycophenolate mofetil, cyclosporine A, or cyclophosphamide was used within 3 months, no ACTH was used within 3 months prior to the enrollment
排除标准
- •Family history of nephrotic syndrome, chronic glomerulonephritis or uremia
- •Leukopenia (White Blood Cells ≤ 3.0 * 10^9 / L)
- •Moderate to severe anemia (hemoglobin <9.0 g/dL)
- •Thrombocytopenia (platelet count <100*10^12/L)
- •Positive Hepatitis B virus serological indicators (Hepatitis B surface antigen or / and Hepatitis B virus e antigen or / and Hepatitis B core antibody), Hepatitis C virus-positive or patients with abnormal liver function (2 or more times of alamine aminotransferase or total bilirubin was exceeded the normal value, and continued to rise for 2 weeks)
- •There are chronic active infections such as Epstein-Barrvirus, cytomegalovirus or Mycobacterium tuberculosis, and the usage of steroids and immunosuppressive agents may aggravate the state of an illness
- •Secondary nephrotic syndrome (such as purpuric nephritis, lupus nephritis, etc.)
- •Those who with hematological or endocrine system diseases as well as serious organs illness such as heart, liver or kidney
- •Those who with other autoimmune diseases or primary immunodeficiencies or tumors
- •Those who have participated in other clinical trials within three months prior to the enrollment
- •Those who was not suitable for participating this study judged by investigator
研究组 & 干预措施
Telitacicept group
Weekly administration (administration time can be within 1 week + 3 days). Body weight and dosage: for subjects with body weight greater than 10kg and less than or equal to 20kg, the dose of Telitacicept is 40mg; for subjects with body weight greater than 20kg and less than or equal to 40kg, the dose of Telitacicept is 80mg; for subjects with body weight greater than 40kg and less than or equal to 60kg, the dose of Telitacicept is 120mg; for subjects with body weight greater than or equal to 60kg, the dose of Telitacicept is 160mg. Treatment duration: 52 weeks.
干预措施: Telitacicept (Drug)
结局指标
主要结局
1-year relapse-free survival rate
时间窗: 1-year period after enrollment
The rate of no relapse within 1 year
次要结局
- Relapse of nephrotic syndrome during 12 months after enrollment(1-year period after enrollment)
- Number of relapses during 12 months follow up(1-year period after enrollment)
- The first time to relapse(1-year period after enrollment)
- Cumulative prednisone dosage (milligrams per kilogram per year)(1-year period after enrollment)
- Change in hemoglobin of the patients(1-year period after enrollment)
- Change in blood albumin of the patients(1-year period after enrollment)
- Change in renal function of the patients(1-year period after enrollment)
- Change in mass index (BMI) during 12-month period after enrollment(1-year period after enrollment)
研究者
Mao Jianhua
Professor
The Children's Hospital of Zhejiang University School of Medicine