Immune Tolerance Induction in Haemophilia A Patients Using Wilate or Nuwiq - A Canadian Study
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- Octapharma
- 入组人数
- 14
- 试验地点
- 3
- 主要终点
- Number of Moderate and Severe Haemophilia A Patients With Inhibitors Achieving Complete or Partial Immune Tolerance Induction (ITI) Success
研究概览
简要总结
Uncontrolled, multi-centre, non-interventional study with a prospective and a retrospective cohort, to evaluate the efficacy of Wilate or Nuwiq in achieving complete or partial immune tolerance induction (ITI) success in severe and moderate haemophilia A patients with inhibitors
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male patients of any age with moderate or severe haemophilia A.
- •Patients with a first occurrence of inhibitors, inhibitors refractory to previous ITI attempt(s), or relapsed inhibitors to FVIII, with an inhibitor titre of ≥0.6 BU measured on 2 separate occasions at least 2 weeks apart.
- •Informed written consent from the patient and/or the patient's parent(s) or legal guardian(s)
- •For patients in the prospective cohort:
- •Patients who are currently on Wilate or Nuwiq ITI, have just initiated ITI, or are planned to initiate ITI treatment with Wilate or Nuwiq.
- •For patients in the retrospective cohort:
- •Patients having received Wilate or Nuwiq ITI before entry into this study. Retrospective data will be collected for a maximum of 3 years before enrolment into the study. To be eligible, the following information is needed:
- •Wilate or Nuwiq treatment details (start date, dose, treatment frequency, and dose change).
- •Reliably documented bleeding frequency.
- •FVIII inhibitor titres.
- •FVIII half-life.
- •FVIII IVR.
排除标准
- •Patients who meet any of the following criteria are not eligible for the study:
- •Congenital or acquired bleeding disorders other than haemophilia A.
- •A history of hypersensitivity to blood products and/or plasma-derived FVIII concentrates.
- •Inability to speak/read English or French well enough to provide consent and adhere to the study.
- •People who are receiving other non-factor therapies, e.g. concizumab
研究组 & 干预措施
Wilate or Nuwiq prospective cohort
Evaluable haemophilia A patients with an inhibitor against FVIII enrolled prospectively
干预措施: Wilate or Nuwiq (Drug)
Wilate or Nuwiq retrospective cohort
Evaluable haemophilia A patients with an inhibitor against FVIII enrolled retrospectively
干预措施: Wilate or Nuwiq (Drug)
结局指标
主要结局
Number of Moderate and Severe Haemophilia A Patients With Inhibitors Achieving Complete or Partial Immune Tolerance Induction (ITI) Success
时间窗: From ITI start until termination of study, a maximum of 2 years
ITI success will be determined using predefined success criteria to analyze the proportion of patients achieving complete or partial ITI success. Complete success is defined by achieving all of the following variables: 1) Inhibitor titre \<0.6BU (at least 2 separate blood samplings) assessed using the modified Bethesda assay; 2) Incremental in vivo recovery (IVR) of FVIII in the normal range (≥66% of normal); 3) FVIII half-life ≥6 hours Wilate infusion. Partial success is defined as two of the three criteria being met, whilst partial response is defined as one of the three criteria being met. Partial failure is defined as none of the three criteria are met, but the inhibitor titre has decreased to \<5 BU; complete failure is defined as none of the three criteria are met, and the inhibitor titre is still ≥5 BU.
次要结局
- Time Necessary to Achieve Complete or Partial ITI Success(A maximum period of 5 years from ITI start)
- In Case of Complete or Partial ITI Success, Duration of Immune Tolerance(A maximum period of 5 years from ITI start)
- Time to Relapse Following Complete or Partial Successful ITI Using Wilate or Nuwiq(A maximum period of 5 years from ITI start)
- Adherence With the ITI Regimen(A maximum period of 5 years from ITI start)
- Bleeding Frequency While on Wilate or Nuwiq ITI Treatment(A maximum period of 5 years from ITI start)
- Association of Inhibitor Titres With the Probability of ITI Success(A maximum period of 5 years from ITI start)
- Use of Bypassing Agents Before and During ITI Treatment With Wilate or Nuwiq(12 months before the start of ITI with Wilate or Nuwiq to a maximum of 5 years from starting ITI with Wilate or Nuwiq)
- Use of Emicizumab (Hemlibra) During ITI Treatment With Wilate or Nuwiq(A maximum period of 5 years from ITI start)
- Relapse Rate Following Complete or Partial Successful ITI Using Wilate or Nuwiq(A maximum period of 5 years from ITI start)