A study of the Safety and Effectiveness of Pegylated Recombinant Factor VIII (BAX 855) in Prevention of Bleeding in Patients with Severe Hemophilia A (a blood clotting disorder) using two different dosing schedules to target different levels of BAX855 in the blood.
- Conditions
- Severe hemophilia A (FVIII <1%)MedDRA version: 20.0Level: LLTClassification code 10060612Term: Hemophilia ASystem Organ Class: 100000011915Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2014-005477-37-AT
- Lead Sponsor
- Baxalta Innovations GmbH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 116
Inclusion Criteria for Subjects Transitioning from Another BAX 855 study:
1. Subject has completed the end of study visit of a BAX 855 study or is transitioning from the ongoing Continuation Study 261302
2. Subject is either receiving on-demand or prophylactic treatment with BAX 855 and had an ABR of = 2 documented and treated during the past 12 months
3. Subject is human immunodeficiency virus negative (HIV-); or HIV+ with stable disease and CD4+ count = 200 cells/mm3, as confirmed by central laboratory
4. Subject is willing and able to comply with the requirements of the protocol
Inclusion Criteria for Newly Recruited Subjects:
1. Subject is 12 to 65 years old at the time of screening
2. Subject has severe hemophilia A (FVIII clotting activity < 1%) as confirmed by central laboratory OR by historically documented FVIII clotting activity performed by a certified clinical laboratory, optionally supported by a FVIII gene mutation consistent with severe hemophilia A
3. Subject has been previously treated with plasma-derived FVIII concentrates or recombinant FVIII for = 150 documented EDs.
4. Subject is either receiving on-demand treatment or prophylactic treatment and had an annual bleeding rate of = 2 documented and treated during the past 12 months.
5. Subject has a Karnofsky performance score of = 60 at screening
6. Subject is HIV-; or HIV+ with stable disease and CD4+ count = 200 cells/mm3, as confirmed by central laboratory at screening
7. Subject is hepatitis C virus negative (HCV-) by antibody (if positive, additional PCR testing will be performed), as confirmed by central laboratory at screening; or HCV+ with chronic stable hepatitis
8. If female of childbearing potential, subject presents with a negative urine pregnancy test and agrees to employ adequate birth control measures for the duration of the study
9. Subject is willing and able to comply with the requirements of the protocol.
Are the trial subjects under 18? yes
Number of subjects for this age range: 58
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 58
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
Exclusion Criteria for Subjects Transitioning from Another BAX 855 study:
1. Subject has developed a confirmed inhibitory antibody to FVIII with a titer of = 0.6 Bethesda Units (BU) using the Nijmegen modification of the Bethesda assay as determined at the central laboratory during the course of the previous BAX 855 study
2. Subject has been diagnosed with an acquired hemostatic defect other than hemophilia A
3. The subject’s weight is < 35 kg or > 100 kg
4. Subject’s platelet count is < 100,000/mL
5. Subject has an abnormal renal function (serum creatinine > 1.5 times the upper limit of normal)
6. Subject has active hepatic disease with alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) levels = 5 times the upper limit of normal
7. Subject is scheduled to receive systemic immunomodulating drug (e.g., corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day, or a-interferon) other than anti-retroviral chemotherapy during the study
8. Subject has a clinically significant medical, psychiatric, or cognitive illness, or
recreational drug/alcohol use that, in the opinion of the investigator, would affect subject’s safety or compliance
9. Subject is planning to take part in any other clinical study during the course of the study.
10. Subject is a member of the team conducting this study or is in a dependent relationship with one of the study team members. Dependent relationships include close relatives (i.e., children, partner/spouse, siblings, parents) as well as employees of the investigator or site personnel conducting the study
Exclusion Criteria for Newly Recruited Subjects
1. Subject has detectable FVIII inhibitory antibodies (= 0.6 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening
2. Subject has a history of confirmed FVIII inhibitors with a titer = 0.6 BU (as determined by the Nijmegen modification of the Bethesda assay or the assay employed with the respective cut-off in the local laboratory) at any time prior to screening
3. Subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand’s disease)
4. The subject’s weight is < 35 kg or > 100 kg.
5. Subject’s platelet count is < 100,000/mL.
6. Subject has known hypersensitivity towards mouse or hamster proteins, PEG or Tween 80
7. Subject has severe chronic hepatic dysfunction [eg, = 5 times upper limit of normal ALT and/ or AST, as confirmed by central laboratory at screening, or a documented INR > 1.5).
8. Subject has severe renal impairment (serum creatinine > 1.5 times the upper limit of normal)
9. Subject has current or recent (< 30 days) use of other pegylated drugs prior to study participation or is scheduled to use such drugs during study participation
10. Subject is scheduled to receive during the course of the study, a systemic immunomodulating drug (eg, corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day, or a- interferon) other than anti-retroviral chemotherapy.
11. Subject has participated in another clinical study involving an 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
12. Subject has a medical, psychiatric, or cognitive illness or recreational
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method