Trial record 1 of 6 for:    rFVIIIFc
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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 not yet open for participant recruitment. (see Contacts and Locations)
Verified September 2014 by Biogen Idec
Sponsor:
Collaborators:
Biogen Idec Australia Pty Ltd
Swedish Orphan Biovitrum
Information provided by (Responsible Party):
Biogen Idec
ClinicalTrials.gov Identifier:
NCT02234323
First received: August 29, 2014
Last updated: September 5, 2014
Last verified: September 2014
  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: BIIB031 (rFVIIIFc)
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
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 Biogen Idec:

Primary Outcome Measures:
  • Number of participants with inhibitor development [ Time Frame: For the duration of study participation, approximately 3 years ] [ Designated as safety issue: Yes ]
    Participants will be tested for development of inhibitors at timepoints 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 ] [ Designated as safety issue: No ]
  • Annualized number of spontaneous joint bleeding episodes per participant [ Time Frame: For the duration of study participation, approximately 3 years ] [ Designated as safety issue: No ]
  • Number of injections required to resolve a bleeding episode [ Time Frame: For the duration of study participation, approximately 3 years ] [ Designated as safety issue: No ]
  • Dose per injection of rFVIIIFc required to resolve a bleeding episode [ Time Frame: For the duration of study participation, approximately 3 years ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • Number of EDs per participant per year [ Time Frame: For the duration of study participation, approximately 3 years ] [ Designated as safety issue: No ]
  • Annualized rFVIIIFc consumption per participant for the prevention and treatment of bleeding episodes [ Time Frame: For the duration of study participation, approximately 3 years ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]

Estimated Enrollment: 125
Study Start Date: December 2014
Estimated Study Completion Date: September 2019
Estimated Primary Completion Date: September 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: BIIB031 (rFVIIIFc)
Doses for on demand or prophylaxis may be adjusted by the investigator within the range of 20 to 80 IU/kg
Biological: BIIB031 (rFVIIIFc)
Administered as specified in the treatment arm
Other Names:
  • rFVIIIFc
  • Recombinant coagulation factor VIII Fc fusion protein

  Eligibility

Ages Eligible for Study:   up to 17 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Ability of the subject or his parent or legal guardian to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations. Subjects may provide assent in addition to the parental/guardian consent, if appropriate.
  • Weight ≥3.5 kg at the time of informed consent.
  • Severe hemophilia A defined as <1 IU/dL (<1%) endogenous coagulation factor VIII (FVIII) documented in the medical record or as tested during the Screening Period. Any subject who is enrolled based on results of the local laboratory must be withdrawn if the central laboratory screening results indicate a baseline FVIII activity level ≥1% of normal.

Key Exclusion Criteria:

  • Prior history of inhibitor as defined by the reporting laboratory. The historical positive inhibitor test is defined as per local laboratory Bethesda value for a positive inhibitor test (i.e., equal to or above lower level of detection).
  • Measurable inhibitor activity at the Screening Visit, measured using the Nijmegen-modified Bethesda assay performed at the central laboratory.
  • History of hypersensitivity reactions associated with any IV immunoglobulin administration.
  • Injection with any FVIII replacement product or any blood component prior to confirmation of eligibility.
  • Injection with rFVIIIFc prior to confirmation of eligibility.
  • Other coagulation disorder(s) in addition to hemophilia A.

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 Idec clinicaltrials@biogenidec.com

Sponsors and Collaborators
Biogen Idec
Biogen Idec Australia Pty Ltd
Swedish Orphan Biovitrum
Investigators
Study Director: Medical Director Biogen Idec
  More Information

No publications provided

Responsible Party: Biogen Idec
ClinicalTrials.gov Identifier: NCT02234323     History of Changes
Other Study ID Numbers: 997HA306, 2013-005512-10
Study First Received: August 29, 2014
Last Updated: September 5, 2014
Health Authority: Ireland: Irish Medicines Board
Australia: Department of Health and Ageing Therapeutic Goods Administration
Italy: The Italian Medicines Agency
Spain: Spanish Agency of Medicines
New Zealand: Medsafe
Canada: Health Canada
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
United States: Food and Drug Administration

Keywords provided by Biogen Idec:
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
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on October 19, 2014