EUCTR2016-001094-33-BE进行中（未招募）1 期
A MULTICENTER, OPEN-LABEL, PHASE III STUDY TO EVALUATE THE EFFICACY, SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF EMICIZUMAB GIVEN EVERY 4 WEEKS (Q4W) IN PATIENTS WITH HEMOPHILIA A
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 48
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
- •Aged >=12 years
- •Body weight >= 40 kg at screening
- •Have severe congenital hemophilia A or hemophilia A with FVIII inhibitors
- •Patients using rFVIIa or willing to switch to rFVIIa as primary bypassing agent for the treatment of breakthrough bleeds
- •FVIII inhibitor test during screening with titer results available prior to first administration of study drug
- •Patients without FVIII inhibitors (< 0.6 Bethesda Units [BU]/milliliter [mL], < 1.0 BU/mL only for laboratories with an historical sensitivity cutoff for inhibitor detection of 1.0 BU/mL) who completed successful immune tolerance induction (ITI) must have done so at least 5 years before screening and must have no evidence of inhibitor recurrence (permanent or temporary) indicated by detection of an inhibitor > 0.6 BU/mL (> 1.0 BU/mL only for laboratories with an historical sensitivity cutoff for inhibitor detection of 1.0 BU/mL) since ITI
- •For patients to be enrolled into PK run-in cohort: Current episodic treatment (FVIII or bypassing agents) at the time of entry into this study and documentation of details of episodic treatment for at least 24 weeks prior to entry into this study
- •For patients to be enrolled into the expansion cohort: Documentation of details of prophylactic or episodic treatment and the number of bleeding episodes for at least 24 weeks prior to entry into this study. For patients on an episodic regimen, >= 5 bleeds in the prior 24 weeks, regardless of inhibitor status
- •For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use highly effective contraceptive methods that result in a failure rate of < 1percent per year during the treatment period and for at least 5 elimination half-lives (24 weeks) after the last dose of study drug
- •Adequate values of hematologic, hepatic, and renal function tests
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 4
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 41
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 4
排除标准
- •Inherited or acquired bleeding disorder other than hemophilia A
- •Ongoing or planned ITI therapy; patient in whom ITI has failed will be eligible with a 72-hour washout period prior to the first emicizumab administration
- •History of illicit drug or alcohol abuse within 48 weeks prior to screening
- •Patients who are at high risk for thrombotic microangiopathy (TMA) (e.g., have a previous medical or family history of TMA), in the investigator’s judgment
- •Previous (within the last 12 months) or current treatment for thromboembolic disease or signs of thromboembolic disease
- •Other conditions (e.g., certain autoimmune diseases) that may currently increase the risk of bleeding or thrombosis
- •History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
- •Known HIV infection with cluster of differentiation 4 (CD4) counts < 200 cells/microliter (µL) (CD4 counts >= 200 cell/µL will be permitted)
- •Use of systemic immunomodulators (e.g., interferon) at enrollment or planned use during the study, with the exception of anti-retroviral therapy
- •Concomitant disease, condition, significant abnormality on screening evaluations or laboratory tests, or treatment that could interfere with the conduct of the study or pose an additional unacceptable risk in administering study drug to the patient
- •Women with a positive serum pregnancy test or pregnant/lactating women
- •Receipt of any of the following: emicizumab in a prior investigational study, an investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration, a non hemophilia related investigational drug within last 30 days or 5 half-lives, whichever is shorter, and any other investigational drug currently being administered or planned to be administered
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