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临床试验/NCT03304288
NCT03304288Unknown2 期

The Combination of Low-dose Rituximab and All-trans Retinoic Acid as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia: a Multicenter, Randomized, Open-label Trial

Peking University People's Hospital4 个研究点 分布在 1 个国家目标入组 168 人开始时间: 2017年10月11日最近更新:
适应症
干预措施
相关药物

试验速览

阶段
2 期
入组人数
168
试验地点
4
主要终点
overall response

研究概览

简要总结

Randomized, open-label, multicentre study to assess the efficacy and safety of the combination of low-dose rituximab and ATRA in patients with steroid-resistant/relapsed ITP.

详细描述

Immune thrombocytopenia (ITP) is a severe bleeding disorder. Approximately 2/3 of patients achieve remission from first-line therapies. However, the underlying mechanism of steroid-resistant or relapsed ITP is not well understood; thus, treatment remains a great challenge. Rituximab has been shown to partly improve the complete remission rate of ITP. All-trans retinoic acid (ATRA) has an immunomodulatory effect on haematopoiesis, making it a possible treatment option.

A multicentre prospective study was performed in non-splenectomized ITP patients who were either resistant to a standard dose of corticosteroids or had relapsed. Patients were randomized to the low-dose rituximab+ATRA and the low-dose rituximab monotherapy groups. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Interim analysis was scheduled at 50% through recruitment. Adverse events are also recorded throughout the study, in order to assess the efficacy and safety of the combination of low-dose rituximab and ATRA in patients with steroid-resistant/relapsed ITP.

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
18 Years 至 —(Adult, Older Adult)
性别
All
接受健康志愿者

入选标准

  • ITP confirmed by excluding other supervened causes of thrombocytopenia;
  • Platelet count of less than 30×10^9/L at enrollment;
  • Patients who did not achieve a sustained response to treatment with full dose corticosteroids for a minimum duration of 4 weeks or who relapsed during steroid-tapering or after its discontinuation;

排除标准

  • Secondary immune thrombocytopenia (e.g., patients with HIV, HCV, Helicobacter pylori infection or patients with systemic lupus erythematosus)
  • Congestive heart failure
  • Severe arrhythmia
  • Nursing or pregnant women
  • Aspartate aminotransferase and alanine transaminase levels ≥ 3×the upper limit of the normal threshold criteria
  • Creatinine or serum bilirubin levels each 1•5 times or more than the normal range
  • Active or previous malignancy
  • Patients with other diseases were undergoing treatment with immunosuppressants
  • Patients with ITP had received rituximab

研究组 & 干预措施

low-dose rituximab & ATRA

Experimental

rituximab 100mg once weekly for 6 weeks and oral all-trance retinoid acid 20mg/m^2 qd for 12 weeks.

干预措施: Rituximab (Drug)

low-dose rituximab & ATRA

Experimental

rituximab 100mg once weekly for 6 weeks and oral all-trance retinoid acid 20mg/m^2 qd for 12 weeks.

干预措施: All-trans retinoic acid (Drug)

low-dose rituximab

Active Comparator

rituximab 100mg once weekly for 6 weeks

干预措施: Rituximab (Drug)

结局指标

主要结局

overall response

时间窗: From the start of study treatment (Day 1) up to the end of Year 1

The number of participants (responders) with platelet count \>=30x10\^9/L and at least a 2-fold increase in the baseline count (PR) or a platelet count \>=100x10\^9/L (CR) and the absence of bleeding, without rescue medication at 1-year follow-up. Interim analysis was scheduled at 50% through recruitment.

sustained response

时间窗: From the start of study treatment (Day 1) up to the end of Year 1

The number of participants that can maintain the platelet count \> 30 x 109/L, an absence of bleeding events, and without requirement for any other ITP-specific treatment for 6 consecutive months after achievement of response. Interim analysis was scheduled at 50% through recruitment.

次要结局

  • time to response(From the start of study treatment (Day 1) up to the end of Year 1)
  • incidence of adverse events(From the start of study treatment (Day 1) up to the end of Year 1)
  • Initial response(From the start of study treatment (Day 1) up to the end of Week 4)
  • complete response(From the start of study treatment (Day 1) up to the end of Year 1)
  • duration of response(From the start of study treatment (Day 1) up to the end of Year 1)

研究者

申办方类型
Other
责任方
Principal Investigator
主要研究者

Xiao-hui Zhang

Professor

Peking University People's Hospital

研究点 (4)

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