NCT02502149

Brief 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 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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
24

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Aug 2015

Geographic Reach
3 countries

15 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 29, 2015

Completed
2 months until next milestone

First Posted

Study publicly available on registry

July 20, 2015

Completed
12 days until next milestone

Study Start

First participant enrolled

August 1, 2015

Completed
1.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2017

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2017

Completed
10 months until next milestone

Results Posted

Study results publicly available

April 6, 2018

Completed
Last Updated

December 19, 2020

Status Verified

March 1, 2018

Enrollment Period

1.7 years

First QC Date

May 29, 2015

Results QC Date

March 9, 2018

Last Update Submit

December 16, 2020

Conditions

Severe Hemophilia A

Keywords

rFVIIIFcEloctateHemophilia

Outcome Measures

Primary Outcomes (2)

  • Area Under the Concentration-time Curve From Time Zero to Infinity (AUCinf) as Measured by One-stage Activated Partial Thromboplastin Time (aPTT) Clotting Assay for Pharmacokinetic Assessment 1 (PK1) and Pharmacokinetic Assessment 2 (PK2)

    AUCinf is area under the concentration-time curve from time zero to infinity. Results were summarized overall for 15K rFVIIIFc 1000 IU/vial and 6000 IU/Vial (PK2).

    Pre-dose and post dose at: 0.5 hour (hr), 1 hr, 6 hr, 24 hr, 48 hr, 72 hr and 96 hr

  • Incremental Recovery (IR) as Measured by One-stage aPTT Clotting Assay for PK1 and PK2

    Incremental Recovery is defined as the increase in the circulating FVIII activity in international unit per deciliter (IU/dL) per unit dose administered in international unit per kilogram (IU/kg) (IU/dL per IU/kg). Results were summarized overall for 15K rFVIIIFc 1000 IU/vial and 6000 IU/Vial (PK2).

    Pre-dose and post dose at: 0.5 hr, 1 hr, 6 hr, 24 hr, 48 hr, 72 hr and 96 hr

Secondary Outcomes (57)

  • Maximum Activity (Cmax) of rFVIIIFc as Measured by One-stage aPTT Clotting Assay for PK1 and PK2

    Pre-dose and post dose at: 0.5 hr, 1 hr, 6 hr, 24 hr, 48 hr, 72 hr and 96 hr

  • Half-life (t½) of rFVIIIFc as Measured by One-stage aPTT Clotting Assay for PK1 and PK2

    Pre-dose and post dose at: 0.5 hr, 1 hr, 6 hr, 24 hr, 48 hr, 72 hr and 96 hr

  • Clearance (CL) of rFVIIIFc as Measured by One-stage aPTT Clotting Assay for PK1 and PK2

    Pre-dose and post dose at: 0.5 hr, 1 hr, 6 hr, 24 hr, 48 hr, 72 hr and 96 hr

  • Volume of Distribution at Steady State (Vss) of rFVIIIFc as Measured by One-stage aPTT Clotting Assay for PK1 and PK2

    Pre-dose and post dose at: 0.5 hr, 1 hr, 6 hr, 24 hr, 48 hr, 72 hr and 96 hr

  • Mean Residence Time (MRT) of rFVIIIFc as Measured by One-stage aPTT Clotting Assay for PK1 and PK2

    Pre-dose and post dose at: 0.5 hr, 1 hr, 6 hr, 24 hr, 48 hr, 72 hr and 96 hr

  • +52 more secondary outcomes

Study Arms (2)

rFVIIIFc (15K scale) 1000 IU vial

EXPERIMENTAL

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.

Biological: rFVIIIFc

rFVIIIFc (15K scale) 6000 IU vial

EXPERIMENTAL

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.

Biological: rFVIIIFc

Interventions

rFVIIIFcBIOLOGICAL

As per arm description

Also known as: Eloctate; BIIB031; efmoroctocog alfa; recombinant coagulation factor VIII Fc fusion protein; antihemophilic factor [recombinant], Fc fusion protein
rFVIIIFc (15K scale) 1000 IU vialrFVIIIFc (15K scale) 6000 IU vial

Eligibility Criteria

Age12 Years+
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (15)

Research Site

Los Angeles, California, 90007, United States

Location

Research Site

Chicago, Illinois, 60612, United States

Location

Research Site

Indianapolis, Indiana, 46260, United States

Location

Research Site

Louisville, Kentucky, 40202, United States

Location

Research Site

East Lansing, Michigan, 48823, United States

Location

Research Site

St Louis, Missouri, 63104, United States

Location

Research Site

Salt Lake City, Utah, 84108, United States

Location

Research Site

Seattle, Washington, 98104, United States

Location

Research Site

Camperdown, New South Wales, 2050, Australia

Location

Research Site

Melbourne, Victoria, 3004, Australia

Location

Research Site

Murdoch, Western Australia, 6150, Australia

Location

Research Site

Grafton, Auckland, 1023, New Zealand

Location

Research Site

Newtown, Wellington Region, 6021, New Zealand

Location

Research Site

Christchurch, 8011, New Zealand

Location

Research Site

Hamilton, 3200, New Zealand

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

factor VIII-Fc fusion proteinFactor VIII

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation FactorsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsProtein PrecursorsBiological Factors

Results Point of Contact

Title
Bioverativ Study Medical Director
Organization
Bioverativ
clinicaltrials@bioverativ.com
781-6631801

Study Officials

  • Medical Director

    Bioverativ Therapeutics Inc.

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
NONE
Purpose
OTHER
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 29, 2015

First Posted

July 20, 2015

Study Start

August 1, 2015

Primary Completion

April 1, 2017

Study Completion

June 1, 2017

Last Updated

December 19, 2020

Results First Posted

April 6, 2018

Record last verified: 2018-03

Locations

US(8)NZ(4)AU(3)