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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

This study is not yet open for participant recruitment.
See Contacts and Locations
Verified March 2017 by Bioverativ Therapeutics Inc.
Sponsor:
Information provided by (Responsible Party):
Bioverativ Therapeutics Inc.
ClinicalTrials.gov Identifier:
NCT03093480
First received: March 10, 2017
Last updated: March 22, 2017
Last verified: March 2017
  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 Biological: rFVIIIFc Phase 4

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: No masking
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 ]
    Tolerization is defined as inhibitor titer less than (<) 0.6 Bethesda units per milliliter (BU/mL).


Secondary Outcome Measures:
  • Number of Participants With Immune Tolerance Induction (ITI) Success [ Time Frame: Up to 48 Weeks ]
    ITI Success will be defined as negative titer for inhibitor less than (<) 0.6 BU/mL by the Nijmegen-modified Bethesda assay.

  • 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
Anticipated Study Start Date: May 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.

  Eligibility

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
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: NCT03093480

Contacts
Contact: Bioverativ Therapeutics Inc, Waltham, MA, USA clinicaltrials@bioverativ.com

Sponsors and Collaborators
Bioverativ Therapeutics Inc.
  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. )
Study First Received: March 10, 2017
Last Updated: March 22, 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

ClinicalTrials.gov processed this record on July 26, 2017