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Assessing Different FVIII Doses and Frequencies in Immune Tolerance Induction (ITI) with ADVATE Among Hemophilia a Boys with Inhibitor (INITIATE Study)

NCT06864975 · Status: RECRUITING · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 110

Last updated 2025-03-07

No results posted yet for this study

Summary

Hemophilia A is a blood coagulation disorder caused by deficient or dysfunctional clotting factor VIII (FVIII) leading to incomplete haemostasis. Patients with severe Hemophilia A are predisposed to recurrent bleeding episodes (BEs) in joints and soft tissues that culminate in debiltating arthropathy and long-term morbidity. Prophylaxis with plasma-derived or recombinant FVIII concentrates effectively restores FVIII levels in patients with Hemophilia A, and significantly reduces the risk of bleeding. A critical concern for patients receiving FVIII replacement therapy is the development of neutralising antibodies (inhibitors) against the treatment. Inhibitors develop in up to 40% of patients with severe Hemophilia A when first exposed to FVIII treatment, typically within the first 20-30 exposure days (EDs) although a residual risk remains until after 75 EDs. Inhibitors preclude the use of FVIII replacement therapy for prevention and treatment of bleeding.

Eradication of inhibitors therefore remains an important objective for Hemophilia A patients with inhibitors. Immune tolerance induction (ITI) therapy is the only clinically proven strategy for inhibitor eradication, and at least one attempt should be offered to patients with inhibitors. However, while ITI is well-studied and has a 60- 80% success rate, treatment regimens can be expensive and burdensome to patients.

There are limited data on the use of different dose regimen of FVIII ITI in China. The INITIATE Study was designed to observe treatment strategies in patients with hemophilia A with inhibitors, with a focus on evaluating the safety and effectiveness of different dose regimens of ITI. The INITIATE Study includes multiple groups to explore factors that may affect ITI outcomes, and to explore the effects of different treatment methods on patient ITI biomarkers (genomics, transcriptomics, proteins (antibodies).

Conditions

  • Hemophilia a with Inhibitor

Interventions

DRUG

Advate®

Non-daily dose FVIII 50IU/kg.QoD or 100IU/kg.QoD

DRUG

Advate®

Daily dose FVIII 50IU/kg.QD or 100IU/kg.QD

DRUG

Advate®

Low dose FVIII 50IU/kg.QD or 100IU/kg.QoD

DRUG

Advate®

Medium and high dose FVIII ≥100IU/kg.QD

DRUG

Advate®

Medium and high dose FVIII ≥100IU/kg.QD with immunosuppressant agent(s)

Sponsors & Collaborators

  • Runhui WU

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
0 Years
Max Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-03-01
Primary Completion
2028-03-01
Completion
2028-06-21

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06864975 on ClinicalTrials.gov