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Trial record 1 of 13 for:    rFVIIIFc
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A Study to Evaluate Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Participants With Inhibitors Undergoing the First ITI Treatment (verITI-8 Study)

This study is currently recruiting participants.
Verified October 2017 by Bioverativ Therapeutics Inc.
Sponsor:
ClinicalTrials.gov Identifier:
NCT03093480
First Posted: March 28, 2017
Last Update Posted: October 16, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
Swedish Orphan Biovitrum
Information provided by (Responsible Party):
Bioverativ Therapeutics Inc.
  Purpose
The primary purpose of this study is to describe the time to tolerization with rFVIIIFc in participants within a maximum of 12 months of ITI treatment.

Condition Intervention Phase
Hemophilia A With Inhibitors Biological: rFVIIIFc Phase 4

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Non-controlled, Open-Label, Multicenter, Study of Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Subjects With Inhibitors Undergoing the First ITI Treatment

Resource links provided by NLM:


Further study details as provided by Bioverativ Therapeutics Inc.:

Primary Outcome Measures:
  • Time to Tolerization With rFVIIIFc [ Time Frame: Up to 12 Months ]
    Time required for participants to achieve ITI success where ITI success is defined as achieving all 3 of the following criteria: Negative titer for inhibitor (less than [<] 0.6 Bethesda units/milliliter [mL] by the Nijmegen-modified Bethesda assay) at 2 consecutive visits; FVIII incremental recovery (IR) >66 percent (%) of the expected IR at 2 consecutive visits; FVIII half-life (t½) greater than or equal to (>=) 7 hours.


Secondary Outcome Measures:
  • Number of Participants With Immune Tolerance Induction (ITI) Success [ Time Frame: Up to 48 Weeks ]
    Number of participants who achieve ITI success where ITI success is defined as achieving all 3 of the following criteria: Negative titer for inhibitor (<0.6 Bethesda units/mL by the Nijmegen-modified Bethesda assay) at 2 consecutive visits; FVIII incremental recovery (IR) >66% of the expected IR at 2 consecutive visits; FVIII half-life (t½) >=7 hours.

  • Number of Participants Who Experience Relapse [ Time Frame: Up to 48 Weeks ]
    Number of Participants with ITI success who reaches the criteria for relapse (defined as inhibitor titer > 0.6 BU/mL or abnormal recovery after tolerance is achieved) will be evaluated.

  • Number of Bleeding Episodes During ITI and During the 48-week Period After Successful ITI performed with rFVIIIFc [ Time Frame: Up to Week 104 ]
    A bleeding episode started from the first sign of a bleed and ended no more than 72 hours after the last treatment for the bleed, within which any symptoms of bleeding at the same location or injections less than or equal to 72 hours apart were considered the same bleeding episode.

  • Number of Participants With Treatment-emergent Adverse Events (AEs) and Treatment-emergent Serious Adverse Events (SAEs) as a Measure of Safety and Tolerability [ Time Frame: Approximately 2 Years ]
    An AE is any untoward medical occurrence that does not necessarily have a causal relationship with this treatment. An SAE is any untoward medical occurrence that at any dose: results in death; in the view of the Investigator, places the participant at immediate risk of death (a life-threatening event); requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect; any other medically important event that, in the opinion of the Investigator, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed in the definition.

  • Number of Days Away From Work or School [ Time Frame: Up to Week 104 ]
    Number of days missed from school or work will be summarized descriptively.

  • Number of Hospitalization Days [ Time Frame: Up to Week 104 ]
    Number of hospitalization days will be summarized descriptively.

  • Adherence to Treatment Regimen [ Time Frame: Up to Week 104 ]
    Defined as percentage of administered doses versus planned doses.

  • Consumption of rFVIIIFc [ Time Frame: Up to Week 104 ]
    Consumption will be assessed based on amount of administered study treatment.


Estimated Enrollment: 30
Actual Study Start Date: June 30, 2017
Estimated Study Completion Date: December 2020
Estimated Primary Completion Date: December 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Recombinant coagulation factor VIII Fc (rFVIIIFc)
Participants will receive rFVIIIFc at a dose of 200 international units (IU)/kilogram (kg) as once daily injections or divided on several injections per day at the discretion of the Investigator, starting at baseline visit up to maximum of 48 Weeks in ITI Period. Participants who meet the criteria for immune tolerance induction (ITI) success will enter the tapering period and will receive rFVIIIFc at a dose adjusted according to Investigator judgement (50 or 100 IU/kg) once a day from Week 1 to 6 and every other day thereafter through Week 16.
Biological: rFVIIIFc
rFVIIIFc 200 IU/kg/day in ITI Period and 50 or 100 IU/kg (adjusted according to Investigator judgement) in tapering Period as powder for injection administered intravenously.
Other Name: ELOCTATE/ELOCTA

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Ability of the participant or his legally authorized representative (e.g., 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 in accordance with national and local participant privacy regulations
  • Male participants of any age diagnosed with severe hemophilia A (as confirmed from the medical record)
  • Diagnosed with high titer inhibitors (historical peak greater than or equal to (>=) 5 Bethesda units per milliliter (BU/mL), according to medical records)
  • Previously treated with any plasma-derived or recombinant conventional or Extended Half-Life FVIII

Exclusion Criteria:

  • Other coagulation disorder(s) in addition to hemophilia A
  • Previous immune tolerance induction (ITI)
  • History of hypersensitivity or anaphylaxis associated with any recombinant coagulation factor VIII Fc (rFVIIIFc) administration
  • Planned major surgery to be deferred after study completion (minor surgery such as tooth extraction or insertion-replacement of central venous access device is allowed)
  • Abnormal renal function (serum creatinine greater than [>] 2.0 milligram per deciliter [mg/dL]) as assessed by local laboratory
  • Serum alanine aminotransferase or aspartate aminotransferase > 5 × upper limit of normal (ULN) as assessed by local laboratory
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03093480


Contacts
Contact: Bioverativ Therapeutics Inc, Waltham, MA, USA 1-888-794-1415 clinicaltrials@bioverativ.com

Locations
United States, California
Center for Inherited Blood Disorders Recruiting
Orange, California, United States, 92868
United States, Mississippi
Mississippi Center for Advanced Medicine (MCAM) Recruiting
Madison, Mississippi, United States, 39110
Contact: Coordinator         
United States, Ohio
Dayton Children's Hospital Recruiting
Dayton, Ohio, United States, 45404
Sponsors and Collaborators
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum
  More Information

Responsible Party: Bioverativ Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT03093480     History of Changes
Other Study ID Numbers: 997HA402
2017-000373-36 ( EudraCT Number )
997HA402 ( Other Identifier: Bioverativ Therapeutics Inc. )
First Submitted: March 10, 2017
First Posted: March 28, 2017
Last Update Posted: October 16, 2017
Last Verified: October 2017

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No

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