NCT04985682已完成4 期
Phase 4, Multicenter, Prospective, Interventional, Post-Marketing Study in Hemophilia A Patients in India Receiving ADVATE as On-Demand or Prophylaxis Under Standard Clinical Practice
Baxalta now part of Shire10 个研究点 分布在 1 个国家目标入组 50 人开始时间: 2022年1月14日最近更新:
适应症
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 50
- 试验地点
- 10
- 主要终点
- Number of Participants With Serious Adverse Events (SAE) at Least Possibly Related to ADVATE
研究概览
简要总结
The main aim of this study is to learn more about side effects of Advate when given as standard treatment to people with hemophilia A who have already been treated.
The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during the study. Participants will need to visit the study doctor 5 times in total during the study. During these visits, study data will be collected by the study doctor.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The participant or legally authorized representative (in case of study participants less than (<) 18 years of age) gave written informed consent to participate in the study.
- •Participant of any age with hemophilia A.
- •Participant defined as a previously treated patient (PTP):
- •Participant aged greater than or equal to (>=) 6 years that has been previously treated with plasma-derived and/or recombinant FVIII concentrate(s) for a minimum of 150 exposure doses (EDs).
- •Participant aged less than <6 years that has been previously treated with plasma-derived or recombinant FVIII concentrate(s) for a minimum of 50 EDs.
- •Participant as negative history of FVIII inhibitors and negative inhibitor at screening defined as less than 0.6 Bethesda units (BU) per milliliter (Nijmegen-modified Bethesda assay).
- •Participant is human immunodeficiency virus negative (HIV-); or human immunodeficiency virus positive (HIV+) with stable disease and cluster of differentiation 4 (CD4+) count >=200 cells per cubic millimeter (mm^3), as confirmed by central laboratory at screening.
- •Participant is hepatitis C virus negative (HCV-) by antibody or polymerase chain reaction (PCR) testing (if positive, anti-body titer will be confirmed by PCR), as confirmed by central laboratory at screening; or hepatitis C virus positive (HCV+) with chronic stable hepatitis.
- •Participant is willing and able to comply with the requirements of the protocol.
排除标准
- •Participant has known hypersensitivity to mouse or hamster proteins or to any of the excipients of FVIII (factor VIII) concentrates.
- •Participant has been diagnosed with bleeding disorder(s) other than congenital hemophilia A, such as acquired hemophilia A, von Willebrand´s disease (VWD) or thrombocytopenia (platelet count <100,000 per milliliter).
- •Participant has received treatment for hemophilia A with non-FVIII products or concentrates (example, emicizumab [Hemlibra®]) in the 6 months prior to screening.
- •Participant has severe chronic hepatic dysfunction (example, >=5 times upper limit of normal alanine aminotransferase [ALT], aspartate aminotransferase [AST] or international normalized ratio [INR] >1.5 as confirmed by central laboratory at screening).
- •Participant has planned or is likely to have, surgery during the study period.
- •Participant has a clinically significant medical, psychiatric, or cognitive illness, or recreational drug or alcohol use that, in the opinion of the investigator, would affect participant's safety or compliance.
- •Participant currently receiving or is scheduled to receive during the course of the study, an immunomodulating drug (example, corticosteroid agents at a dose equivalent to hydrocortisone >10 milligram per day, or α-interferon) other than antiretroviral chemotherapy.
- •Participant has participated in another clinical study involving an investigational product (IP) or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
- •Participant is a family member or employee of the investigator.
结局指标
主要结局
Number of Participants With Serious Adverse Events (SAE) at Least Possibly Related to ADVATE
时间窗: Baseline (Day 0) up to end of study (up to 12.9 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this investigational product or medicinal product. An SAE was defined as any untoward medical occurrence that resulted in death; was life-threatening; required inpatient hospitalization or prolongation of present hospitalization; resulted in persistent or significant disability/incapacity; was a congenital anomaly/birth defect or was a medically important event. Number of participants with SAEs (including FVIII inhibitor formation) that were at least possibly related to ADVATE were reported.
次要结局
- Average Number of ADVATE Infusions Required Per Week During Prophylactic Treatment(Baseline (Day 0) up to end of study (up to 12.9 months))
- Average Number of ADVATE Infusions Required Per Month During Prophylactic Treatment of Bleeding Episode(Baseline (Day 0) up to end of study (up to 12.9 months))
- Total Annualized Bleeding Rate (ABR) With Prophylactic Treatment of ADVATE(Baseline (Day 0) up to end of study (up to 12.9 months))
- ABR With Prophylactic Treatment of ADVATE Categorized Based on Location of Bleed(Baseline (Day 0) up to end of study (up to 12.9 months))
- Total Number of ADVATE Infusions Required During Prophylactic Treatment(Baseline (Day 0) up to end of study (up to 12.9 months))
- Average Body Mass Adjusted Consumption of ADVATE Per Week During Prophylactic Treatment(Baseline (Day 0) up to end of study (up to 12.9 months))
- Number of Participants With Non-serious Adverse Events (AEs) at Least Possibly Related to ADVATE(Baseline (Day 0) up to end of study (up to 12.9 months))
- Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameters(Baseline (Day 0) up to end of study (up to 12.9 months))
- ABR With Prophylactic Treatment of ADVATE Categorized Based on Type of Bleed(Baseline (Day 0) up to end of study (up to 12.9 months))
- Total Body Mass Adjusted Consumption of ADVATE During Prophylactic Treatment(Baseline (Day 0) up to end of study (up to 12.9 months))
- Average Body Mass Adjusted Consumption of ADVATE Per Month During Prophylactic Treatment(Baseline (Day 0) up to end of study (up to 12.9 months))
- Number of ADVATE Infusions Required to Achieve Resolution of Bleeding Episodes(Baseline (Day 0) up to end of study (up to 12.9 months))
- Total Body Mass Adjusted Consumption of ADVATE Per Bleeding Episode(Baseline (Day 0) up to end of study (up to 12.9 months))
- Overall Hemostatic Efficacy Rating of ADVATE for Treatment of Bleeding Episodes(Baseline (Day 0) up to end of study (up to 12.9 months))
研究者
研究点 (10)
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