EUCTR2015-000583-34-Outside-EU/EEA进行中（未招募）不适用
An Exploratory Study of the Safety and Efficacy of Immune Tolerance Induction (ITI) in Patients with Pompe Disease Who Have Previously Received Myozyme
Genzyme Corporation Inc0 个研究点目标入组 9 人开始时间: 2015年4月15日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 9
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
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 a-glucosidase (GAA) enzyme deficiency from any tissue source or 2 GAA gene mutations;
- •The patient (and/or legal guardian) must have ability to comply with clinical protocol;
- •If the patient is Cross-reacting immunologic material (CRIM)-positive, he/she must have received at least 6 consecutive months of Myozyme infusions (20mg/kg qow)
- •If the patient is CRIM-negative, he/she must have received at least 1 Myozyme infusion prior to enrollment
- •Regimen A only: The patient exhibits clinical decline; The patient has persistent high anti-recombinant human acid a-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 CRIM-negative AND The patient does not exhibit clinical decline; OR ALL OF THE FOLLOWING: The patient is CRIM-negative AND The patient exhibits clinical decline AND The patient does NOT exhibit high anti-rhGAA antibody titers and has NOT tested positive for antibodies that inhibit enzymatic activity and/or uptake of Myozyme.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 4
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •The patient has a clinical condition unrelated to Pompe disease that would interfere with program assessments;
- •The patient is at risk of reactivation or is a carrier of Hepatitis B or Hepatitis C;
- •The patient is at risk of reactivation or has positive serology suggestive of active infection for cytomegalovirus, Herpes simplex, JC virus, Parvovirus or Epstein Barr virus;
- •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 low serum albumin;
- •The patient has a major congenital abnormality;
- •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.
研究者
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