Brief Title
BAX 855 Continuation
Official Title
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
Brief Summary
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.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Number of Participants With Inhibitory Antibodies to Factor VIII (FVIII)
Secondary Outcome
Total Annualized Bleed Rate (ABR)
Condition
Hemophilia A
Intervention
BAX855
Study Arms / Comparison Groups
Fixed BAX855 prophylaxis
Description: 45-80 IU/kg twice weekly to once per week.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
218
Start Date
October 15, 2013
Completion Date
March 2, 2018
Primary Completion Date
March 2, 2018
Eligibility Criteria
INCLUSION CRITERIA
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:
1. Participant has completed a previous BAX 855 study and is willing to immediately
transition into this continuation study.
2. Participant is ≤75 years of age at screening of the previous BAX 855 study.
3. Participant continues to have a Karnofsky (for participants aged ≥ 16 years) or
Lansky (for participants aged < 16 years) performance score of ≥ 60.
4. 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.
5. 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.
6. 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.
7. 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:
1. Participant is ≤75 years of age at screening.
2. Participant is naïve to BAX 855.
3. Participant has severe hemophilia A (FVIII clotting activity < 1%) as confirmed by
central laboratory at screening after at least a 72-hour washout period.
4. Participant aged ≥ 6 years has documented previous treatment with plasma-derived FVIII
or rFVIII for ≥ 150 exposure days (EDs).
5. Participant aged < 6 years has documented previous treatment with plasma-derived FVIII
concentrates or rFVIII for ≥ 50 EDs.
6. Participant is currently receiving prophylaxis or on-demand therapy with FVIII.
7. Participant has a Karnofsky (for participants aged ≥ 16 years) or Lansky (for
participants aged < 16 years) performance score of ≥ 60.
8. Participant is HIV-; or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm^3, as
confirmed by central laboratory at screening.
9. 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.
10. 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.
11. 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:
1. 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.
2. 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).
3. 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.
4. Participant has severe chronic hepatic dysfunction (eg, ≥ 5 times upper limit of
normal alanine aminotransferase [ALT], as confirmed by central laboratory at
screening).
5. Participant has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed
by central laboratory at screening.
6. Participant experienced a life-threatening or gastrointestinal bleeding episode within
3 months prior to study entry.
7. Participant is scheduled to use other PEGylated drugs during study participation.
8. 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.
9. 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.
10. 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:
1. Participant has detectable FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen
modification of the Bethesda assay) as confirmed by central laboratory at screening.
2. 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.
3. Participant has been diagnosed with an inherited or acquired hemostatic defect other
than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease).
4. Participant has known hypersensitivity towards mouse or hamster proteins, polyethylene
glycol (PEG), or Tween 80.
5. Participant has severe chronic hepatic dysfunction eg, ≥ 5 times upper limit of normal
ALT, as confirmed by central laboratory at screening).
6. Participant has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed
by central laboratory at screening.
7. Participant experienced a life-threatening or gastrointestinal bleeding episode within
3 months prior to study entry.
8. 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.
9. 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.
10. 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.
11. Participant is a family member or employee of the investigator.
Gender
All
Ages
N/A - 75 Years
Accepts Healthy Volunteers
No
Contacts
Study Director, ,
Location Countries
Australia
Location Countries
Australia
Administrative Informations
NCT ID
NCT01945593
Organization ID
261302
Secondary IDs
2013-002236-24
Responsible Party
Sponsor
Study Sponsor
Baxalta now part of Shire
Collaborators
Baxalta Innovations GmbH, now part of Shire
Study Sponsor
Study Director, Study Director, Takeda
Verification Date
April 2021