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)

Bioverativ

Status and phase: Completed; Phase 4

Conditions: Hemophilia A With Inhibitors

Treatments: Biological: rFVIIIFc

Study type: Interventional

Funder types: Industry

Identifiers

NCT03093480

2017-000373-36 (EudraCT Number)

LPS16473

997HA402 (Other Identifier)

Details and patient eligibility

About

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.

Enrollment: 16 patients

Sex: Male

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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16 participants in 1 patient group

Recombinant coagulation factor VIII Fc (rFVIIIFc)

Experimental group

Description:

Participants were to 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 met the criteria for immune tolerance induction (ITI) success entered the tapering period and received rFVIIIFc at a dose adjusted according to Investigator judgment based on the FVIII activity levels and with the aim of tapering the rFVIIIFc dose to reach a prophylactic dosing regimen within 16 weeks (4 months). Follow-Up was for 32 weeks under an adjusted prophylactic regimen according to Investigator judgment.

Treatment:

Biological: rFVIIIFc