Efficacy and Safety of Thalidomide in Treating Pediatric PFAPA Syndrome: A Multicenter Randomized Controlled Trial
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 106
- 主要终点
- Proportion of Participants Achieving Complete Remission at 6 Months
研究概览
简要总结
The goal of this clinical trial is to evaluate the efficacy and safety of thalidomide in the treatment of children with Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Adenitis (PFAPA) syndrome. The study focuses on children diagnosed with PFAPA syndrome. The main questions it aims to answer are:
Can thalidomide significantly reduce the frequency of febrile episodes in children with PFAPA syndrome? What is the safety profile and tolerability of thalidomide in this pediatric population? Researchers will compare the thalidomide group to a colchicine group to see if thalidomide is more effective in controlling recurrent fever and associated symptoms.
Participants will:
Take the assigned medication (thalidomide or colchicine) daily for a duration of 6 months.
Attend follow-up visits every 4 weeks at the clinic. Maintain a diary to record the frequency of fever episodes and any other clinical symptoms.
Undergo safety assessments and physical examinations during each scheduled visit.
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
- None
入排标准
- 年龄范围
- 3 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Meet the 2019 Eurofever or 2020 CARRA diagnostic criteria for PFAPA syndrome.
- •Aged 3 to 18 years (inclusive) at the time of screening.
- •Have experienced at least 3 febrile episodes within the past six months.
- •History of responsiveness to glucocorticoid treatment during at least 3 previous episodes, but with continued recurrence. (Responsiveness is defined as normalization of body temperature within 24 hours after a maximum dose of 2 mg/kg [up to 60 mg] administered as a single or two divided doses).
排除标准
- •1.Diagnosis of monogenic or other polygenic periodic fever syndromes. 2.Presence of immunodeficiency or neoplastic diseases. 3.Active bacterial, fungal, or viral infection during the screening period. 4.Prior treatment with immunosuppressive agents. 5.Prior use of thalidomide or colchicine. 6.Laboratory parameters at screening that meet any of the following (based on the most recent test result at the study hospital prior to the first dose):
- •White Blood Cell (WBC) count < 4 × 10⁹/L, Hemoglobin (HGB) < 100 g/L, or Platelet (PLT) count < 100 × 10⁹/L.
- •Serum Alanine Aminotransferase (ALT) > 2 times the Upper Limit of Normal (ULN).
- •Glomerular Filtration Rate (GFR/CCR) < 60 mL/min/1.73m².
研究组 & 干预措施
Thalidomide Group
Patients in this group will receive oral thalidomide treatment for 12 months.
干预措施: Thalidomide (50mg) (Drug)
Colchicine Group
Patients in this group will receive oral colchicine treatment for 12 months.
干预措施: Colchicine (Drug)
结局指标
主要结局
Proportion of Participants Achieving Complete Remission at 6 Months
时间窗: 6 months
Complete remission is defined as the total absence of febrile episodes (zero attacks) during the treatment period.
次要结局
- Complete Remission Rate at Multiple Time Points(3 months)
- Complete Remission Rate at Multiple Time Points(9 months)
- Complete Remission Rate at Multiple Time Points(12 months)
- Partial Remission Rate(3 months)
- Recurrence Rate Post-discontinuation(6 months post-treatment)
- Recurrence Rate Post-discontinuation(12 months post-treatment)
- Change in Growth Parameters (Z-scores)(6 months)
- Change in Growth Parameters (Z-scores)(12 months)
- Change in Inflammatory Markers(6 months)
研究者
Wenjie Zheng
Chief Physician
Second Affiliated Hospital of Wenzhou Medical University