NCT02525172Unknown4 期
Immune Modulation Therapy for ERT-naïve or ERT-treated Pompe Disease Patients
适应症
干预措施
相关药物
试验速览
- 阶段
- 4 期
- 入组人数
- 8
- 试验地点
- 1
- 主要终点
- anti-recombinant human acid α-glucosidase (anti-rhGAA) antibody titers decrease
研究概览
简要总结
The purpose of this study is to assess anti-recombinant human acid α-glucosidase (anti-rhGAA) antibody titers after treatment with immune modulation therapy in patients of Pompe disease.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The patient (and/or patient's legal guardian if patient is < 18years) must provide written informed consent prior to any study-related procedures that are performed;
- •The patient must have a confirmed diagnosis of Pompe disease defined as a documented acid α-glucosidase (GAA) enzyme deficiency from blood samples or 2 GAA gene mutations;
- •The patient (and/or legal guardian) must have ability to comply with clinical protocol;
- •Regimen A only: The patient is receiving enzyme replacement therapy, exhibits clinical decline, and has persistent high anti-recombinant human acid α-glucosidase (anti-rhGAA) antibody titers and/or tested positive for antibodies that inhibit enzymatic activity and/or uptake of Myozyme;
- •Regimen B only: The patient is cross-reactive immune material (CRIM) -negative AND have not received Myozyme infusion prior to enrollment
排除标准
- •The patient is at risk of reactivation or is a carrier of Hepatitis B or Hepatitis C;
- •The patient is at risk of reactivation of tuberculosis or has regular contact with individuals who are being actively treated for tuberculosis;
- •The patient has used any investigational product (other than alglucosidase alfa) within 30 days prior to study enrollment;
- •The patient is pregnant or lactating;
- •The patient has had or is required to have any live vaccination within one month prior to enrollment.
研究组 & 干预措施
ITT
Experimental
immune modulation therapy
干预措施: Rituximab (Drug)
ITT
Experimental
immune modulation therapy
干预措施: intravenous immune globulin (Drug)
ITT
Experimental
immune modulation therapy
干预措施: Bortezomib (Drug)
ITT
Experimental
immune modulation therapy
干预措施: Methotrexate (Drug)
结局指标
主要结局
anti-recombinant human acid α-glucosidase (anti-rhGAA) antibody titers decrease
时间窗: 6 months
次要结局
未报告次要终点
研究者
研究点 (1)
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