Phase 4
Completed N=16
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)
Hemophilia A With Inhibitors
Source: ClinicalTrials.gov NCT03093480 ↗
Enrolled (actual)
16
Serious AEs
56.3%
Results posted
Jul 2021
Primary outcomePrimary: Time to Tolerization With rFVIIIFc — 11.7 weeks
◆ Published Evidence
Emerging
18citations · ~6 / year
Recombinant factor VIII Fc fusion protein for first-time immune tolerance induction: final results of the verITI-8 study.
Summary
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.
Linked Publications
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Recombinant factor VIII Fc fusion protein for first-time immune tolerance induction: final results of the verITI-8 study.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Time to Tolerization With rFVIIIFc |
11.7 | — |
| SECONDARY Number of Participants With Immune Tolerance Induction (ITI) Success |
10 | — |
| SECONDARY Number of Participants Who Experienced Relapse |
— | — |
| SECONDARY Annualized Bleeding Rates During ITI Period |
6.6 | — |
| SECONDARY Annualized Bleeding Rates After ITI Period |
1.0; 1.2 | — |
| SECONDARY Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (TESAEs) as a Measure of Safety and Tolerability |
16; 9; 0; 0 | — |
| SECONDARY Average Number of Days Missed From Work or School Per Month During ITI Period |
2.3 | — |
| SECONDARY Average Number of Days Missed From Work or School Per Month After ITI Period |
— | — |
| SECONDARY Annualized Number of Hospitalization Days During ITI Period |
15.4 | — |
| SECONDARY Annualized Number of Hospitalization Days After ITI Period |
2.7 | — |
| SECONDARY Adherence to Treatment Regimen Overall Study Period |
100.8 | — |
| SECONDARY Annualized rFVIIIFc Consumption for Overall Study Period |
42713.4 | — |
Eligibility 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)
- 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
Data sourced from ClinicalTrials.gov (NCT03093480) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.