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An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Untreated Males With Severe Hemophilia A

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2017 by Bioverativ Therapeutics Inc.
Sponsor:
Collaborator:
Swedish Orphan Biovitrum
Information provided by (Responsible Party):
Bioverativ Therapeutics Inc.
ClinicalTrials.gov Identifier:
NCT02234323
First received: August 29, 2014
Last updated: February 27, 2017
Last verified: February 2017
  Purpose
The primary objective of the study is to evaluate the safety of rFVIIIFc (BIIB031) in previously untreated participants with severe hemophilia A. The secondary objectives are to evaluate the efficacy of rFVIIIFc in the prevention and treatment of bleeding episodes in previously untreated patients (PUPs), to evaluate rFVIIIFc consumption for the prevention and treatment of bleeding episodes in PUPs, and to describe experience with the use of rFVIIIFc for immune tolerance induction (ITI) in participants with inhibitors.

Condition Intervention Phase
Hemophilia A
Biological: rFVIIIFc
Phase 3

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
Official Title: An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc; BIIB031) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Severe Hemophilia A

Resource links provided by NLM:


Further study details as provided by Bioverativ Therapeutics Inc.:

Primary Outcome Measures:
  • Number of participants with inhibitor development [ Time Frame: For the duration of study participation, approximately 3 years ]
    Participants will be tested for development of inhibitors at time points throughout the study based on exposure days (ED). One ED is equivalent to a 24 hour period in which rFVIIIFc is dosed


Secondary Outcome Measures:
  • Annualized number of bleeding episodes per participant [ Time Frame: For the duration of study participation, approximately 3 years ]
  • Annualized number of spontaneous joint bleeding episodes per participant [ Time Frame: For the duration of study participation, approximately 3 years ]
  • Number of injections required to resolve a bleeding episode [ Time Frame: For the duration of study participation, approximately 3 years ]
  • Dose per injection of rFVIIIFc required to resolve a bleeding episode [ Time Frame: For the duration of study participation, approximately 3 years ]
  • Response to treatment with rFVIIIFc for bleeding episodes, using the 4-point bleeding response scale [ Time Frame: For the duration of study participation, approximately 3 years ]
    Investigators will record assessments of each participant's response to their assigned rFVIIIFc regimen using the following 4-point scale: Excellent, Effective, Partially Effective, Ineffective

  • Number of EDs per participant per year [ Time Frame: For the duration of study participation, approximately 3 years ]
  • Annualized rFVIIIFc consumption per participant for the prevention and treatment of bleeding episodes [ Time Frame: For the duration of study participation, approximately 3 years ]
  • rFVIIIFc incremental recovery (IR) as measured by the one-stage aPTT clotting assay and the two-stage chromogenic assay [ Time Frame: For the duration of study participation, approximately 3 years ]
    Samples for incremental recovery taken when the participant is in a non-bleeding state.

  • Response to immune tolerance induction (ITI) with rFVIIIFc (success, partial success, failure, early withdrawal) [ Time Frame: Until immune tolerance is achieved or up to 33 months ]

Estimated Enrollment: 125
Study Start Date: January 2015
Estimated Study Completion Date: December 2020
Estimated Primary Completion Date: December 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: rFVIIIFc
Doses for prophylaxis may be adjusted by the investigator within the range of 25 to 80 IU/kg.
Biological: rFVIIIFc
Administered as specified in the treatment arm
Other Names:
  • Eloctate; BIIB031; efmoroctocog alfa; recombinant coagulation factor VIII Fc fusion protein; antihemophilic factor [recombinant]
  • Fc fusion protein

  Eligibility

Ages Eligible for Study:   up to 5 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Ability of the participant's legally authorized representative (e.g. their parent or legal guardian) to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use confidential health information in accordance with national and local subject privacy regulations.
  • Weight ≥3.5 kg at the time of screening.
  • Severe hemophilia A defined as <1 IU/dL (<1%) endogenous FVIII documented in the medical record or as tested during the Screening Period

Key Exclusion Criteria:

  • Any exposure to blood components, factor VIII replacement products, including commercially available rFVIIIFc at any time prior to or during screening.
  • Other coagulation disorder(s) in addition to hemophilia A.
  • Any concurrent clinically significant major disease that, in the opinion of the Investigator, would make the participant unsuitable for enrollment.
  • Current systemic treatment with chemotherapy and/or other immunosuppressant drugs.

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02234323

Contacts
Contact: Biogen clinicaltrials@biogen.com

  Show 50 Study Locations
Sponsors and Collaborators
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum
Investigators
Study Director: Medical Director Biogen
  More Information

Responsible Party: Bioverativ Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT02234323     History of Changes
Other Study ID Numbers: 997HA306
2013-005512-10 ( EudraCT Number )
Study First Received: August 29, 2014
Last Updated: February 27, 2017

Keywords provided by Bioverativ Therapeutics Inc.:
prophylaxis treatment
Hemophilia A
Hemophilia
episodic treatment

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Immunoglobulin Fc Fragments
Coagulants
Immunologic Factors
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on March 24, 2017