Brief Title
Pharmacokinetics of rFVIIIFc at Two Vial Strengths
Official Title
A Randomized, Open-Label, Crossover Study to Evaluate the Pharmacokinetics of 2 Vial Strengths of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc; BIIB031) in Previously Treated Subjects With Severe Hemophilia A
Brief Summary
The primary objective of the study is to characterize the pharmacokinetics (PK) of rFVIIIFc
administered at vial strengths of 1000 and 3000 IU in subjects with severe hemophilia A. The
secondary objective of the study is to evaluate the safety of rFVIIIFc beyond the PK
assessment for up to 6 months for a continued treatment period.
Detailed Description
This is a randomized, open-label, crossover study during which each participant receives a
single injection of rFVIIIFc from 2 different vial concentrations (PK assessment). After the
PK assessment, participants are provided with rFVIIIFc for either prophylactic or episodic
(on-demand) treatment for up to 6 months.
Study Phase
Phase 1
Study Type
Interventional
Primary Outcome
Area Under the Concentration-time Curve From Time Zero to Infinity (AUCinf) as Measured by Activated Partial Thromboplastin Time (aPTT) Clotting Assay
Secondary Outcome
Maximum Activity (Cmax) as Measured by the aPTT Clotting Assay
Condition
Severe Hemophilia A
Intervention
rFVIIIFc
Study Arms / Comparison Groups
rFVIIIFc 1000 / 3000 PK Assessment
Description: A single intravenous (IV) injection of rFVIIIFc 50 IU/kg at a strength of 1000 IU/vial followed by a single IV injection of rFVIIIFc 50 IU/kg at a strength of 3000 IU/vial. Following the PK assessment, participants will receive either an episodic (on-demand) regimen with doses between 20 and 50 IU/kg based on the severity of the bleeding episode, or 1 of 2 prophylactic regimens: 50 IU/kg every 3 to 5 days or 65 IU/kg weekly. Participants will be allowed to switch from one regimen to another if approved by the Investigator.
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
19
Start Date
March 2014
Completion Date
May 2015
Primary Completion Date
October 2014
Eligibility Criteria
Key Inclusion Criteria:
- Have severe hemophilia A
- Previously treated subject, defined as having at least 150 documented prior exposure
days to any recombinant and/or plasma-derived FVIII and/or cryoprecipitate products
(other than any use of rFVIIIFc- study drug or commercial product) 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 subjects.
- No measurable inhibitor activity using the Nijmegen-modified Bethesda assay at
Screening.
- Platelet count ≥100,000 platelets/μL at screening
- CD4 lymphocytes >200 mm3 if known as HIV antibody positive at screening.
- Viral load of <400 copies/mL if known HIV antibody positive at screening.
Key Exclusion Criteria:
- Subject is at high risk of bleeding during the 5-day period between the first and
second injections for PK analyses, as per Investigator discretion.
- Previous treatment with rFVIIIFc as study drug or commercial product.
- Other coagulation disorder(s) in addition to hemophilia A.
- History of hypersensitivity or anaphylaxis associated with any FVIII or IV
immunoglobulin administration.
- Currently taking (or likely to require during the study) acetylsalicylic acid (ASA),
except for low-dose ASA as prophylaxis (other nonsteroidal anti-inflammatory drugs are
permitted).
- Concurrent systemic treatment with immunosuppressive drugs within 12 weeks prior to
Day 1. Exceptions to this include: ribavirin for treatment of hepatitis C virus (HCV),
and/or systemic steroids (a total of 2 courses of pulse treatments lasting no more
than 7 days at a dose of ≤1 mg/kg within 12 weeks prior to Day 1) and/or inhaled
steroids.
NOTE: Other protocol-defined inclusion/exclusion Criteria May Apply
Gender
Male
Ages
12 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Medical Director, ,
Location Countries
Australia
Location Countries
Australia
Administrative Informations
NCT ID
NCT02083965
Organization ID
997HA307
Secondary IDs
2013-003013-18
Responsible Party
Sponsor
Study Sponsor
Bioverativ Therapeutics Inc.
Study Sponsor
Medical Director, Study Director, Bioverativ Therapeutics Inc.
Verification Date
May 2017