A Phase 3, Open-label, Multicenter, Pharmacokinetics, Efficacy, and Safety Study of a Recombinant Single-chain Factor VIII (rVIII-SingleChain) in Chinese Previously Treated Patients (PTPs) With Hemophilia A
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- CSL Behring
- 入组人数
- 60
- 试验地点
- 37
- 主要终点
- Incremental Recovery (IR) of rVIII-SingleChain
研究概览
简要总结
For bridging the available global clinical data of rVIII-SingleChain, with the Chinese population, the aim of this study in China is to investigate the pharmacokinetics (PK) of rVIII-SingleChain after an initial and repeat dose and to assess efficacy and safety during 2 to 3 times weekly prophylaxis treatment with rVIII-SingleChain in male Chinese PTPs with severe hemophilia A (FVIII activity less than [<] 1%).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 年龄范围
- 0 Days 至 65 Years（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •• Male Chinese participants <= 65 years of age.
- •• Participants with severe hemophilia A (FVIII activity < 1%).
- •• Participants who have received FVIII products for >= 150 EDs (>= 6 years of age) or >= 50 EDs (< 6 years of age).
排除标准
- •• Known hypersensitivity (allergic reaction or anaphylaxis) to any FVIII product or hamster protein.
- •• Known congenital or acquired coagulation disorder other than congenital FVIII deficiency.
- •• Currently receiving intravenous (IV) immunomodulating agents such as immunoglobulin or chronic systemic corticosteroid treatment.
- •• Receiving any cryoprecipitate, whole blood, or plasma within 30 days before administration of rVIII-SingleChain.
- •• Use of traditional or herbal Chinese medicine(s) with an impact on hemophilia, including coagulation, within 28 days before Day 1 and / or refusal to abstain from these during the study until the end of the participant's participation in the study.
研究组 & 干预措施
rVIII-SingleChain
Participants will receive rVIII-SingleChain as an intravenous (IV) infusion for a minimum of 50 exposure days (EDs).
干预措施: Recombinant single-chain factor VIII (rVIII-SingleChain) (Biological)
结局指标
主要结局
Incremental Recovery (IR) of rVIII-SingleChain
时间窗: Before, and at 30 minutes after the end of, rVIII-SingleChain administration on Day 1
Maximum Concentration (Cmax) of rVIII-SingleChain
时间窗: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
Area Under the Plasma Concentration Time Curve from Time Zero to the Last Measurable Concentration (AUC0-last) of rVIII-SingleChain
时间窗: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
Area Under the Plasma Concentration Time Curve from Time Zero to Infinity (AUC0-inf) of rVIII-SingleChain
时间窗: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
Half-life (t1/2) of rVIII-SingleChain
时间窗: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
Clearance (Cl) of rVIII-SingleChain
时间窗: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
Annualized Spontaneous Bleeding Rate (AsBR)
时间窗: Up to 29 weeks after rVIII-SingleChain administration
AsBR for treated bleeding episodes
Number of participants who develop FVIII inhibitors
时间窗: During routine rVIII-SingleChain prophylaxis dosing, up to 29 weeks after rVIII-SingleChain administration.
次要结局
- Time to reach maximum concentration (Tmax) of rVIII-SingleChain(Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1)
- Last Concentration (Clast) of rVIII-SingleChain(Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1)
- IR (participants ≥ 12 years of age) of rVIII-SingleChain (Repeat pharmacokinetic [PK])(Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28)
- Cmax (participants ≥ 12 years of age) of rVIII-SingleChain (Repeat PK)(Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28)
- AUC0-last of rVIII-SingleChain (Repeat PK)(Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28)
- AUC0-inf of rVIII-SingleChain (Repeat PK)(Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28)
- t1/2 of rVIII-SingleChain (participants ≥ 12 years of age) (Repeat PK)(Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28)
- Cl of rVIII-SingleChain (participants ≥ 12 years of age) (Repeat PK)(Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28)
- Annualized Bleeding Rate (ABR)(Up to 29 weeks after rVIII-SingleChain administration)
- Hemostatic Efficacy for Major and Nonmajor Bleeding Episodes(Up to 29 weeks after rVIII-SingleChain administration)
- Consumption of rVIII-SingleChain - number of infusions (doses)(Up to 29 weeks after rVIII-SingleChain administration)
- Consumption of rVIII-SingleChain - IU/kg per participant per month(Up to 29 weeks after rVIII-SingleChain administration)
- Consumption of rVIII-SingleChain - IU/kg per participant per year(Up to 29 weeks after rVIII-SingleChain administration)
- Number of bleeding episodes requiring rVIII-SingleChain to achieve hemostasis(Up to 29 weeks after rVIII-SingleChain administration)
- Percentage of bleeding episodes requiring rVIII-SingleChain to achieve hemostasis(Up to 29 weeks after rVIII-SingleChain administration)
- Number of participants who develop noninhibitory antibodies against rVIII-SingleChain(Before, and up to 29 weeks after, rVIII-SingleChain administration)
- Number of participants who develop antibodies against Chinese hamster ovary host cell protein(Before, and up to 29 weeks after, rVIII-SingleChain administration)
- Number of participants with Treatment-emergent Adverse Events (TEAEs), including related TEAEs, and serious adverse events (SAEs)(Up to 33 weeks after rVIII-SingleChain administration)
- Percentage of participants with TEAEs, including related TEAEs, and serious adverse events (SAEs)(Up to 33 weeks after rVIII-SingleChain administration)
- Number of TEAEs (events)(Up to 33 weeks after rVIII-SingleChain administration)