Pharmacokinetics and Safety of rFVIIIFc Manufactured at 15,000 L (15K) Scale
Information source: Biogen
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Severe Hemophilia A
Intervention: rFVIIIFc (Biological)
Phase: Phase 3
Status: Not yet recruiting
Sponsored by: Biogen Official(s) and/or principal investigator(s): Medical Director, Study Director, Affiliation: Biogen
Overall contact: Biogen, Email: clinicaltrials@biogen.com
Summary
The primary objective of the study is to compare the pharmacokinetic (PK) of recombinant
coagulation factor VIII Fc fusion protein (rFVIIIFc) manufactured at the current scale of
2000 L (2K) to the PK of rFVIIIFc manufactured at the 15,000 L (15K) scale in previously
treated subjects with severe hemophilia A. The secondary objectives are: to characterize
the PK of rFVIIIFc manufactured at the 15K scale at the 15K baseline and after 13 weeks of
treatment; to characterize the PK of rFVIIIFc manufactured at the 15K scale at 1000 IU/vial
and a higher strength vial; and to evaluate the safety of rFVIIIFc manufactured at the 15K
scale.
Clinical Details
Official title: A Randomized, Open-Label Study to Evaluate the Pharmacokinetics and Safety of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc; BIIB031) Manufactured at 15K Scale and at Different Vial Strengths in Previously Treated Subjects With Severe Hemophilia A
Study design: Allocation: Randomized, Endpoint Classification: Pharmacokinetics Study, Intervention Model: Parallel Assignment, Masking: Open Label
Primary outcome: Area under the concentration time curve from time zero to infinity (AUCinf) for PK1 and PK2Incremental recovery (IR) for PK1 and PK2
Secondary outcome: area under the concentration-time curve from time zero to infinity (AUCinf) for PK2 and PK3incremental recovery (IR) for PK2 and PK3 the maximum rFVIIIFc activity (Cmax) for PK2 and PK3 half-life (t½) for PK2 and PK3 clearance (CL) for PK2 and PK3 volume of distribution at steady state (Vss) at PK2 and PK3 mean residence time (MRT) at PK2 and PK3 AUCinf of rFVIIIFc manufactured at the 15K scale at different vial strengths IR of rFVIIIFc manufactured at the 15K scale at different vial strengths Cmax of rFVIIIFc manufactured at the 15K scale at different vial strengths t½ of rFVIIIFc manufactured at the 15K scale at different vial strengths CL of rFVIIIFc manufactured at the 15K scale at different vial strengths Vss of rFVIIIFc manufactured at the 15K scale at different vial strengths MRT of rFVIIIFc manufactured at the 15K scale at different vial strengths Cmax at PK1 and Pk2 t½ at PK1 and Pk2 CL at PK1 and Pk2 Vss at PK1 and Pk2 MRT at PK1 and Pk2 Development of inhibitors as measured by the Nijmegen-modified Bethesda assay Number of participants that experience adverse events (AEs) and serious adverse events (SAEs)
Detailed description:
PK assessments are in 3 phases: PK1: PK assessments following single injection of rFVIIIFc
manufactured at the 2K scale. PK2: PK assessments are made following a single injection of
rFVIIIFc manufactured at the 15K scale where participants are randomized to the 1000 IU vial
or a higher strength vial. PK3: PK assessments are made following 13 weeks of rFVIIIFc
treatment manufactured at the 15K scale where participants are randomized to the 1000 IU
vial or a higher strength vial. After study completion, in countries where rFVIIIFc is not
commercially available, eligible subjects will be offered enrollment into a long-term safety
and efficacy extension study (8HA01EXT [NCT01454739]).
Eligibility
Minimum age: 12 Years.
Maximum age: N/A.
Gender(s): Male.
Criteria:
Key Inclusion Criteria:
- Have severe hemophilia A, defined as <1 IU/dL (<1%) endogenous FVIII as determined by
one-stage clotting assay from the central laboratory at Screening.
- Previously treated subject, defined as having at least 150 documented prior exposure
days (EDs) to any recombinant and/or plasma-derived FVIII and/or cryoprecipitate
products at Day 1. Fresh frozen plasma treatment must not be considered in the count
for documented exposure days.
- No history of a positive inhibitor test or clinical signs of decreased response to
FVIII administrations. Family history of inhibitors will not exclude the subject.
- No measurable inhibitor activity using the Nijmegen-modified Bethesda assay (≥0. 6
BU/mL is considered positive) at Screening.
Key Exclusion Criteria:
- Current enrollment in any interventional clinical study in which an investigational
drug or approved therapy for investigational use is administered within 30 days prior
to the Baseline Visit.
- Previous participation in this study.
- Any concurrent clinically significant major disease that, in the opinion of the
Investigator or Biogen, makes the subject unsuitable for participation in the study.
- Other coagulation disorder(s) in addition to hemophilia A.
- History of hypersensitivity or anaphylaxis associated with FVIII or IV immunoglobulin
administration.
NOTE: Other protocol-defined inclusion/exclusion criteria may apply.
Locations and Contacts
Biogen, Email: clinicaltrials@biogen.com Additional Information
Starting date: September 2015
Last updated: July 16, 2015
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