Pharmacokinetics and Safety of rFVIIIFc Manufactured at 15,000 L (15K) Scale

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

24 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-08-31

Study Completion Date

2017-06-30

Brief Summary

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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 participants 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 6000 IU/vial strengths; and to evaluate the safety of rFVIIIFc manufactured at the 15K scale.

Detailed Description

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PK assessments are in 3 phases: Pharmacokinetic Assessment 1(PK1): PK assessments following single injection of rFVIIIFc manufactured at the 2K scale. Pharmacokinetic Assessment 2 (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 6000 IU/vial strengths. Pharmacokinetic Assessment 3 (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 6000 IU/vial strengths. After study completion, in countries where rFVIIIFc is not commercially available, eligible participants will be offered enrollment into a long-term safety and efficacy extension study (8HA01EXT \[NCT01454739\]).

Conditions

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Severe Hemophilia A

Keywords

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rFVIIIFc Eloctate Hemophilia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

OTHER

Blinding Strategy

NONE

Study Groups

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rFVIIIFc (15K scale) 1000 IU vial

Single injection of rFVIIIFc (current 2K scale) followed by 2 single injections of rFVIIIFc (15K scale) 1000 IU vial at PK2 and PK3 timepoints. Participants will be on prophylaxis regimen along with treatment for bleeding episodes for 26 weeks of treatment period using the rFVIIIFc (15K scale) 1000 IU vial.

Group Type EXPERIMENTAL

rFVIIIFc

Intervention Type BIOLOGICAL

As per arm description

rFVIIIFc (15K scale) 6000 IU vial

Single injection of rFVIIIFc (current 2K scale) followed by 2 single injections of rFVIIIFc high strength vial (15K scale) at PK2 and PK3 timepoints. Participants will be on prophylaxis regimen along with treatment for bleeding episodes for 26 weeks of treatment period using the rFVIIIFc (15K scale) 6000 IU vial.

Group Type EXPERIMENTAL

rFVIIIFc

Intervention Type BIOLOGICAL

As per arm description

Interventions

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rFVIIIFc

As per arm description

Intervention Type BIOLOGICAL

Other Intervention Names

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Eloctate; BIIB031; efmoroctocog alfa; recombinant coagulation factor VIII Fc fusion protein; antihemophilic factor [recombinant] Fc fusion protein

Eligibility Criteria

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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 Bethesda Unit per milliliter \[BU/mL\] is considered positive) at Screening.

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 OR prior participation in any of the following Biogen studies: 998HA101 (NCT01027377), 997HA301 (NCT01181128), 8HA02PED (NCT01458106), 997HA307 (NCT02083965), and 8HA01EXT (NCT01454739).
* 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 intravenous (IV) immunoglobulin administration.

Minimum Eligible Age

12 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Bioverativ Therapeutics Inc.

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United States Australia New Zealand

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2014-003895-21

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

997HA309

Identifier Type: -

Identifier Source: org_study_id

Pharmacokinetics and Safety of rFVIIIFc Manufactured at 15,000 L (15K) Scale

Study Results

Basic Information

Brief Summary

Detailed Description

Conditions

Keywords

Study Design

Study Groups

rFVIIIFc (15K scale) 1000 IU vial

rFVIIIFc

rFVIIIFc (15K scale) 6000 IU vial

rFVIIIFc

Interventions

rFVIIIFc

Other Intervention Names

Eligibility Criteria

Inclusion Criteria

Exclusion Criteria

Sponsors

Swedish Orphan Biovitrum

Bioverativ Therapeutics Inc.

Responsible Party

Principal Investigators

Medical Director

Locations

Research Site

Research Site

Research Site

Research Site

Research Site

Research Site

Research Site

Research Site

Research Site

Research Site

Research Site

Research Site

Research Site

Research Site

Research Site

Countries

Provided Documents

Document Type: Study Protocol

Document Type: Statistical Analysis Plan

Other Identifiers

2014-003895-21

997HA309