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Completed

NCT Number: NCT03093480

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)

The primary purpose of this study was to describe the time to tolerization (i.e., ITI success) with rFVIIIFc in participants within a maximum of 48 weeks (12 months) of ITI treatment.

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Key information

Conditions

Hemophilia A With Inhibitors

Sex eligibility

Male

Study type

Interventional

Phase

Phase 4

Primary location

Cliniques Universitaires Saint-Luc, Brussels, Belgium

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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)
  • Currently 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 factor VIII (FVIII) administration
  • Planned major surgery scheduled during the study unless deferred until after study completion (minor surgery such as tooth extraction or insertion/replacement of central venous access device is allowed)
  • Abnormal renal function (serum creatinine >1.5 milligram per deciliter (mg/dL) or 2 × upper limit of normal (ULN) for participant age based on local laboratory range) as assessed by local laboratory
  • Serum alanine aminotransferase or aspartate aminotransferase > 5 × upper limit of normal (ULN) as assessed by local laboratory

Treatment and study plan

rFVIIIFc

Biological

rFVIIIFc 200 IU/kg/day in ITI Period, 50 or 100 IU/kg (adjusted according to Investigator judgement) in tapering Period, and prophylactic regimen in Follow-Up period as powder for injection administered intravenously.

Other names: ELOCTATE/ELOCTA; BIIB031; efmoroctocog alfa; antihemophilic factor [recombinant], Fc fusion protein

Primary outcomes

  1. Time to Tolerization With rFVIIIFc

    Time frame: Up to 48 Weeks

    Time required for participants to achieve immune tolerance induction (ITI) success where ITI success is defined as achieving all 3 of the following criteria: confirmed negative titers consisting of 2 consecutive negative inhibitor assessments within 2 weeks (less than [<] 0.6 Bethesda units/milliliter [mL] by the Nijmegen-modified Bethesda assay); incremental recovery (IR) greater than or equal to (>=) 66 percent (%) of the expected IR in 2 consecutive assessments; half-life (t½) >= 7 hours.

Secondary outcomes

  1. 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: confirmed negative titers consisting of 2 consecutive negative inhibitor assessments within 2 weeks (<0.6 Bethesda units/mL by the Nijmegen-modified Bethesda assay); incremental recovery (IR) >= 66% of the expected IR at 2 consecutive assessments; half-life (t½) >=7 hours.

  2. Number of Participants Who Experienced Relapse

    Time frame: Up to 48 weeks (16 weeks Tapering period and 32 weeks follow-up period)

    Number of Participants with ITI success who reaches the criteria for relapse (defined as confirmed positive inhibitor titer >= 0.6 BU/mL or abnormal recovery after tolerance is achieved, and t½ less than [<] 7 hours) evaluated during the Tapering or Follow-Up Periods

  3. Annualized Bleeding Rates During ITI Period

    Time frame: Up to 48 weeks

    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. Annualized bleeding rate for a patient during the ITI period is defined as the number of bleeding episodes divided by the length of the ITI period in days* 365.25.

  4. Annualized Bleeding Rates After ITI Period

    Time frame: Up to 48 weeks (16 weeks Tapering period and 32 weeks follow-up period)

    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. Annualized bleeding rate for a patient after the ITT period (for tapering and follow-up period) is defined as the number of bleeding episodes divided by the length of the period after the ITI period in days* 365.25.

  5. Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (TESAEs) as a Measure of Safety and Tolerability

    Time frame: Up to 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.

  6. Average Number of Days Missed From Work or School Per Month During ITI Period

    Time frame: Up to 48 weeks

    Average number of days missed from school or work per month for a period (counting in non-missing diary days) is defined as number of the missing school/work days in the period divided by number of days with data entry in the period.

    Number of days per month missed from school or work is reported for those who attend school or have a job.

  7. Average Number of Days Missed From Work or School Per Month After ITI Period

    Time frame: Up to 48 weeks (16 weeks Tapering period & 32 weeks Follow-up period)

    Average number of days missed from school or work per month for a period (counting in non-missing diary days) is defined as number of the missing school/work days in the period divided by number of days with data entry in the period.

    Number of days per month missed from school or work is reported for those who attend school or have a job.

  8. Annualized Number of Hospitalization Days During ITI Period

    Time frame: Up to 48 weeks

    Annualized number of hospitalization days during a period for a patient is defined as the number of hospitalization days divided by the length of the period in days * 365.25.

  9. Annualized Number of Hospitalization Days After ITI Period

    Time frame: Up to 48 weeks (16 weeks Tapering period & 32 weeks Follow-up period)

    Annualized number of hospitalization days during a period for a patient is defined as the number of hospitalization days divided by the length of the period in days * 365.25.

  10. Adherence to Treatment Regimen Overall Study Period

    Time frame: Up to 2 Years

    Adherence to treatment is based on prescribed daily dose for the overall study period which is defined as the percentage of administered doses versus the prescribed doses to a patient for the entire study duration.

  11. Annualized rFVIIIFc Consumption for Overall Study Period

    Time frame: Up to 2 Years

    Annualized rFVIIIFc consumption for a treatment period is the total nominal rFVIIIFc (IU/kg) / length of period in days * 365.25.

Sponsors and collaborators

Lead sponsor

Bioverativ, a Sanofi company

Industry

Collaborators

  • Swedish Orphan Biovitrum

Registry information

Official study 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

Important dates

Study start
2017
Primary completion
2020
Study completion
2021
First posted
Mar 28, 2017
Registry last updated
Mar 28, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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