Brief Title
Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Pediatric Subjects With Hemophilia A
Official Title
An Open-Label, Multicenter Evaluation of Safety, Pharmacokinetics, and Efficacy of Recombinant Coagulation Factor VIII Fc Fusion Protein, BIIB031, in the Prevention and Treatment of Bleeding Episodes in Pediatric Subjects With Hemophilia A
Brief Summary
The primary objective of the study is to evaluate the safety of Recombinant Coagulation
Factor VIII Fc Fusion Protein (rFVIIIFc) in previously treated pediatric subjects with
hemophilia A. Secondary objectives of this study in this study population are as follows: to
evaluate the efficacy of rFVIIIFc for prevention and treatment of bleeding episodes; to
evaluate and assess the pharmacokinetics (PK) of rFVIIIFc; and to evaluate rFVIIIFc
consumption for prevention and treatment of bleeding episodes.
Detailed Description
Previously treated pediatric participants will be treated with a prophylactic regimen of
rFVIIIFc. PK analysis of pre-study factor VIII (FVIII) and rFVIIIFc will be performed in a
sub-group of the study participants prior to commencement of prophylactic treatment. After
these PK results are available, remaining participants have the option of proceeding directly
to prophylactic treatment. After completing the end of study assessments, eligible
participants would be able to continue treatment in Study 8HA01EXT.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Occurrence of FVIII Inhibitor Development
Secondary Outcome
Annualized Bleeding Rate
Condition
Hemophilia A
Intervention
BIIB031 (rFVIIIFc)
Study Arms / Comparison Groups
All participants
Description: PK subgroup: After a Washout Period of ≥72 hours, at the Baseline Visit (28±7 days prior to Day 1), participants receive a single IV injection of prestudy FVIII over 5 (±2) minutes at a dose of 50 IU/kg, rounded up to the nearest 250 IU increment, for a PK assessment. Following a second Washout Period of ≥72 hours, participants receive a single IV injection of rFVIIIFc over 5 (±2) minutes at a dose of 50 IU/kg for PK assessment. The first prophylactic dose of rFVIIIFc is administered at a starting dose of 25 IU/kg IV injection on Day 1 and 50 IU/kg on Day 4. Dose increases to a maximum of 80 IU/kg, and frequency of administration to a minimum interval of once every 2 days, are allowed as indicated. Non-PK subgroup: On Day 1, a first prophylactic dose of rFVIIIFc of 25 IU/kg IV injection is given, followed by a dose of 50 IU/kg on Day 4. Dose increases to a maximum of 80 IU/kg, and frequency of administration to a minimum interval of once every 2 days, are allowed as indicated.
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
Drug
Estimated Enrollment
71
Start Date
November 2012
Completion Date
December 2013
Primary Completion Date
December 2013
Eligibility Criteria
Key Inclusion Criteria:
- Severe hemophilia A defined as <1 IU/dL (<1%) endogenous FVIII
- Male <12 years of age and weight ≥13 kg
- History of at least 50 documented prior exposure days to FVIII
- No current, or history of, inhibitor development to FVIII
Key Exclusion Criteria:
- Other coagulation disorders in addition to Hemophilia A
- History of anaphylaxis associated with any FVIII or IV immunoglobulin administration
NOTE: Other protocol-defined Inclusion/Exclusion criteria may apply.
Gender
Male
Ages
N/A - 11 Years
Accepts Healthy Volunteers
No
Contacts
Medical Director, ,
Location Countries
Australia
Location Countries
Australia
Administrative Informations
NCT ID
NCT01458106
Organization ID
8HA02PED
Secondary IDs
2011-003073-28
Responsible Party
Sponsor
Study Sponsor
Bioverativ Therapeutics Inc.
Collaborators
Swedish Orphan Biovitrum
Study Sponsor
Medical Director, Study Director, Bioverativ Therapeutics Inc.
Verification Date
August 2018