A Phase III Study on the Safety, Pharmacokinetics, and Efficacy of Coagulation Factor VIIa (Recombinant) in Congenital Hemophilia A or B Pediatric Patients From Birth to <12 Years Old With Inhibitors to Factor VIII or IX: PerSept 2
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 25
- 试验地点
- 12
- 主要终点
- Proportion of Successfully Treated Mild/Moderate Bleeding Episodes Per FDA Requirement.
研究概览
简要总结
The purpose of the study is to assess the safety, efficacy and pharmacokinetics of 2 separate dose regimens (75µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or IX in 12 patients ( birth to <6 years old), and 12 patients (≥6 years old to <12 years old).
详细描述
A Phase III Study on the Safety, Pharmacokinetics, and Efficacy of Coagulation Factor VIIa (Recombinant) in Congenital Hemophilia A or B Pediatric Patients from birth to <12 years old with Inhibitors to Factor VIII or IX: PerSept 2
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 11 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •be male with a diagnosis of congenital hemophilia A or B of any severity
- •have one of the following:
- •a positive inhibitor test BU ≥5, OR
- •a Bethesda Unit (BU) <5 but expected to have a high anamnestic response to FVIII or FIX, as demonstrated from the patient's medical history, precluding the use of factor VIII or IX products to treat bleeding episodes, OR
- •a BU <5 but expected to be refractory to increased dosing of FVIII or FIX, as demonstrated from the patient's medical history, precluding the use of factor VIII or IX products to treat bleeding episodes
- •be aged from birth to <12 years old
- •have experienced at least 3 bleeding episodes of any severity in the past 6 months
- •parents or legal guardians must be capable of understanding and be willing to comply with the conditions of the protocol
- •parents or legal guardians must have read, understood, and provided written informed consent
排除标准
- •have any coagulation disorder other than hemophilia A or B
- •be immunosuppressed (i.e., the patient may not be receiving systemic immunosuppressive medication; cluster of differentiation 4 (CD4) counts at screening must be >200/µL)
- •have a known allergy or hypersensitivity to rabbits
- •have platelet count <100,000/mL
- •have had a major surgical procedure (e.g. orthopedic, abdominal) within 1 month prior to first administration of study drug
- •have received an investigational drug within 30 days of first study drug administration, or be expected to receive such drug during participation in this study
结局指标
主要结局
Proportion of Successfully Treated Mild/Moderate Bleeding Episodes Per FDA Requirement.
时间窗: 12 hours after first administration of study drug
For the primary efficacy endpoint, successful treatment of mild/moderate bleeding episode was defined as meeting all of the following: * "Good" or "excellent" response noted by the patient/parent/legal guardian or other caregiver, depending on patient's age and maturity * Study drug treatment: No further treatment with LR769 beyond timepoint where a "good" or "excellent" response for this bleeding episode was noted * No other hemostatic treatment needed for this bleeding episode * No administration of blood products that would indicate continuation of bleeding beyond timepoint where a "good" or "excellent" response for this bleeding episode was noted * No increase of pain beyond timepoint where a "good" or "excellent" response for this bleeding episode was noted that could not be explained other than as continuation of bleeding
Proportion of Successfully Treated Bleeding Episodes (Mild/Moderate/Severe) Per EMA Definition
时间窗: 12 hours after first administration of study drug
* "Good" or "excellent" response noted by the patient/caregiver for mild/moderate bleeding episodes; * "Good" or "excellent" response noted by the physician for severe bleeding episodes.
次要结局
- Total Amount of Study Drug Administered Per Mild/Moderate Bleeding Episode(Within 24 hours of Bleeding Episode)
- Patient-Reported "Good" or "Excellent" Response for Mild/Moderate Bleeding Episodes(12 hour after first administration of study drug)
- Number of Administrations of Study Drug Per Mild/Moderate Bleeding Episode(Within 24 hours of Bleeding Episode)
- Time to Patient Assessment of a "Good" or "Excellent" Response for Mild/Moderate Bleeding Episodes(Within 24 hours of Bleeding Episode)