Assessing Different FVIII Dose and Frequency in Immune Tolerance Induction (ITI) with ADVATE Among Hemophilia a Boys with Inhibitor (INITIATE Study): a Multicenter Open-label Non-randomized Clinical Trial

Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 4
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).

Eligibility
Participation Requirements
Sex: Male
Maximum Age: 18
Healthy Volunteers: f
View:

• Severe hemophilia A (FⅧ:C \<2%);

• Positive for FVIII inhibitors;

• No allergic reactions to FVIII concentrates.

Locations
Other Locations
China
Beijing Children's hospitial
RECRUITING
Beijing
Contact Information
Primary
Dr. Wu
runhuiwu@hotmail.com
+86 010-59616623
Backup
+86 010-59616623
zekunlee@outlook.com
+86 010-59616623
Time Frame
Start Date: 2025-03-01
Estimated Completion Date: 2028-06-21
Participants
Target number of participants: 110
Treatments
Experimental: Low titer inhibitor - Non-daily dose arm
Peak inhibitor titer \< 5 BU of historical and during ITI treatment Using FVIII (Advate) 50IU/kg.QoD or 100IU/kg.QoD
Experimental: Low titer inhibitor - daily dose arm
Peak inhibitor titer \< 5 BU of historical and during ITI treatment Using FVIII (Advate) 50IU/kg.QD or 100IU/kg.QD
Experimental: High titer inhibitor - low dose arm
Peak inhibitor titer ≥5BU and \<200BU of historical and during ITI treatment Using FVIII (Advate) 50IU/kg.QD or 100IU/kg.QD
Experimental: High titer inhibitor - medium or high dose arm
Peak inhibitor titer ≥5BU and \<200BU of historical and during ITI treatment Usiing FVIII (Advate) ≥ 100IU/kg.QD
Experimental: High titer inhibitor with poor prognosis
Peak inhibitor ≥200BU of historical and during ITI treatment FVIII (Advate) ≥ 100IU/kg.QD with immunosuppression agent(s)
Related Therapeutic Areas
Sponsors
Leads: Runhui WU

This content was sourced from clinicaltrials.gov