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临床试验/NCT01945593
NCT01945593已完成3 期

A Phase 3b Continuation Study of the Safety and Efficacy of PEGylated Recombinant Factor VIII (PEG-rFVIII; BAX 855) in Prophylaxis of Bleeding in Previously Treated Patients With Severe Hemophilia A

Baxalta now part of Shire177 个研究点 分布在 10 个国家目标入组 218 人开始时间: 2013年10月15日最近更新:
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适应症

试验速览

阶段
3 期
状态
已完成
发起方
入组人数
218
试验地点
177
主要终点
Annualized Bleed Rate (ABR) - Spontaneous Bleeds
研究概览研究设计入排标准结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

To continue the evaluation of the safety and efficacy of BAX 855 for prophylaxis and treatment of bleeding episodes in adult and pediatric previously treated patients (PTPs) aged ≤ 75 years of age with severe hemophilia A.

研究设计

研究类型
Interventional
分配方式
Non Randomized
干预模型
Parallel
主要目的
Prevention
盲法
None

入排标准

年龄范围
— 至 75 Years（Child, Adult, Older Adult）
性别
All
接受健康志愿者
否

入选标准

  • •Participants Transitioning from Other BAX 855 Studies:
  • •Participants transitioning from other BAX 855 studies can be provided with the continuation study informed consent form (ICF) prior to the end of study visit to review and consider participation in this continuation study. These participants will complete any additional screening assessments within 2 weeks of the previous study's end of study visit and will return to the study site within 6 (± 1) weeks of the previous study end of study visit to confirm eligibility for this continuation study.
  • •Participants transitioning from other BAX 855 studies who meet ALL of the following criteria are eligible for this study:
  • •Participant has completed a previous BAX 855 study and is willing to immediately transition into this continuation study.
  • •Participant is ≤75 years of age at screening of the previous BAX 855 study.
  • •Participant continues to have a Karnofsky (for participants aged ≥ 16 years) or Lansky (for participants aged < 16 years) performance score of ≥
  • •Participant is human immunodeficiency virus negative (HIV-); or HIV+ with stable disease and CD4+ count ≥ 200 cells/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, antibody titer will be confirmed by PCR), as confirmed by central laboratory at screening; or HCV+ with chronic stable hepatitis.
  • •If female of childbearing potential, participant presents with a negative urine pregnancy test and agrees to employ adequate birth control measures for the duration of the study.
  • •Participant and/or legally authorized representative is willing and able to comply with the requirements of the protocol.
  • •BAX 855 Naïve Participants:
  • •BAX 855 naïve participants who are ≥ 12 years of age can only be enrolled in this continuation study after enrollment in the phase 2/3 pivotal study is closed. BAX 855 naïve participants who are < 12 years of age can only be enrolled in this continuation study after enrollment in the pediatric previously treated patient (PTP) study is closed.
  • •- Enrolment of BAX 855 naïve participants will only start once the sponsor has notified the study sites accordingly.
  • •BAX 855 naïve participants who meet ALL of the following criteria are eligible for this study:
  • •Participant is ≤75 years of age at screening.
  • •Participant is naïve to BAX
  • •Participant has severe hemophilia A (FVIII clotting activity < 1%) as confirmed by central laboratory at screening after at least a 72-hour washout period.
  • •Participant aged ≥ 6 years has documented previous treatment with plasma-derived FVIII or rFVIII for ≥ 150 exposure days (EDs).
  • •Participant aged < 6 years has documented previous treatment with plasma-derived FVIII concentrates or rFVIII for ≥ 50 EDs.
  • •Participant is currently receiving prophylaxis or on-demand therapy with FVIII.
  • •Participant has a Karnofsky (for participants aged ≥ 16 years) or Lansky (for participants aged < 16 years) performance score of ≥
  • •Participant is HIV-; or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm^3, as confirmed by central laboratory at screening.
  • •Participant is HCV- by antibody or PCR testing (if positive, antibody titer will be confirmed by PCR), as confirmed by central laboratory at screening; or HCV+ with chronic stable hepatitis.
  • •If female of childbearing potential, participant presents with a negative urine pregnancy test and agrees to employ adequate birth control measures for the duration of the study.
  • •Participant and/or legally authorized representative is willing and able to comply with the requirements of the protocol.
  • •EXCLUSION CRITERA
  • •- Participants Transitioning from Other BAX 855 Studies:
  • •Participants transitioning from other BAX 855 studies who meet ANY of the following criteria are not eligible for this study:
  • •Participant had detectable factor VIII (FVIII) inhibitory antibodies (≥ 0.6 Bethesda unit (BU) using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening.
  • •Participant has developed FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay as determined at central laboratory in a previous BAX 855 study).
  • •Participant has acquired a hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease) in a previous BAX 855 study.
  • •Participant has severe chronic hepatic dysfunction (eg, ≥ 5 times upper limit of normal alanine aminotransferase [ALT], as confirmed by central laboratory at screening).
  • •Participant has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screening.
  • •Participant experienced a life-threatening or gastrointestinal bleeding episode within 3 months prior to study entry.
  • •Participant is scheduled to use other PEGylated drugs during study participation.
  • •Participant is planning to take part in any other clinical study during the course of the continuation study, with the exception of any other parallel BAX 855 study.
  • •Participant has medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance.
  • •Participant is a family member or employee of the investigator.
  • •BAX 855 Naïve Participants:
  • •BAX 855 naïve participants who meet ANY of the following criteria are not eligible for this study:
  • •Participant has detectable FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening.
  • •Participant has history of FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay or the Bethesda assay) at any time prior to screening.
  • •Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease).
  • •Participant has known hypersensitivity towards mouse or hamster proteins, polyethylene glycol (PEG), or Tween
  • •Participant has severe chronic hepatic dysfunction eg, ≥ 5 times upper limit of normal ALT, as confirmed by central laboratory at screening).
  • •Participant has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screening.
  • •Participant experienced a life-threatening or gastrointestinal bleeding episode within 3 months prior to study entry.
  • •Participant has current or recent (< 30 days) use of other PEGylated drugs prior to study participation or scheduled use of such drugs during study participation.
  • •Participant has participated in another clinical study involving an IP other than BAX 855 or device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an investigational product (IP) or investigational device during the course of this study.
  • •Participant has medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance.
  • 另有 1 项未显示

排除标准

  • 未提供

结局指标

主要结局

Annualized Bleed Rate (ABR) - Spontaneous Bleeds

时间窗: Baseline through end of study (53 months)

The ABR was assessed based upon each individual bleeding episode. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII. The ABR of spontaneous bleeds was reported separately for twice weekly, PK-t R, each of the every 5 days and every 7 days treatment regimens at the time of bleed.

Number of Participants With Inhibitory Antibodies to Factor VIII (FVIII)

时间窗: Baseline through end of study (53 months)

Inhibitory antibodies to Factor VIII were measured by the Nijmegen modification of the Bethesda assay. Inhibitors had to be confirmed by 2 separate assessments within a 2 to 4 week period from the central laboratory.

次要结局

  • Total Annualized Bleed Rate (ABR)(Baseline through end of study (53 months))
  • Overall Hemostatic Efficacy Rating of BAX 855 for Treatment of Breakthrough Bleeding Episodes(Baseline through end of study (53 months))
  • Change From Baseline in Pain Severity(Baseline, end of study (53 months))
  • Total Time Intervals Between Bleeding Episodes(Baseline through end of study (53 months))
  • Average Dose of BAX 855 Per Prophylactic Infusion(Baseline through end of study (53 months))
  • Change From Baseline in Blood Pressure(Baseline, end of study (53 months))
  • Change From Baseline in Bleed Severity(Baseline, end of study (53 months))
  • BAX 855 Infusions Needed to Treat Bleeding Episodes(Baseline through end of study (53 months))
  • Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)(Baseline through end of study (53 months))
  • Change From Baseline in Body Temperature(Baseline, end of study (53 months))
  • Change From Baseline in Respiratory Rate(Baseline, end of study (53 months))
  • Change From Baseline in Pulse Rate(Baseline, end of study (53 months))
  • Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)(Baseline, end of study (53 months))
  • Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.(Baseline through end of study (53 months))
  • Number of Participants With Shifts in Hematology Laboratory Assessments(Baseline through end of study (53 months))
  • Number of Participants With Shifts in Lipid Panel Assessments(Baseline through end of study (53 months))
  • Number of Participants With Binding Antibodies(Baseline through end of study (53 months))
  • Number of Participants With Anti-Chinese Hamster Ovary (CHO) Antibodies(Baseline through end of study (53 months))
  • Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Pediatrics Quality of Life (PedsQL) Questionnaire(Baseline, end of study (53 months))

研究者

发起方
Baxalta now part of Shire
申办方类型
Industry
责任方
Sponsor

研究点 (177)

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