Actively Recruiting
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
Led by Runhui WU · Updated on 2025-03-07
110
Participants Needed
1
Research Sites
16 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This research focuses on boys with Hemophilia A who have developed inhibitors against factor VIII (FVIII) treatment. Hemophilia A is a genetic bleeding disorder caused by deficient FVIII, leading to frequent bleeding episodes that can cause joint damage and long-term health problems. Inhibitors are antibodies that neutralize FVIII and make standard replacement therapy ineffective, representing a major treatment challenge. The study aims to evaluate the safety and effectiveness of various dosing regimens of immune tolerance induction (ITI) therapy using FVIII to eradicate these inhibitors and improve patient outcomes. Participants receive different doses and frequencies of ADVATE4, a recombinant FVIII product, to induce immune tolerance. The study includes multiple groups based on inhibitor levels and treatment history: low inhibitor titer groups receive either non-daily or daily doses of 50 or 100 IU/kg; high inhibitor titer groups receive low, medium, or high doses of ADVATE, some combined with immunosuppressive agents for those with poor prognosis. This multicenter, open-label trial is designed to explore the impact of dose and frequency on ITI success and to examine related biomarkers. During the study, boys up to 18 years old with severe Hemophilia A and inhibitors will be closely monitored over 24 months to assess treatment success. Researchers will track inhibitor levels, immune markers, bleeding episodes, and safety outcomes. Patients undergo regular infusions of ADVATE according to assigned dosing schedules, with clinical assessments and laboratory tests to evaluate immune tolerance induction. The study also aims to identify predictors of ITI outcomes and relapse risks to improve personalized treatment strategies for children with Hemophilia A and inhibitors.
CONDITIONS
Brief Title
Assessing Different FVIII Doses and Frequencies in Immune Tolerance Induction (ITI) with ADVATE Among Hemophilia a Boys with Inhibitor (INITIATE Study)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Severe hemophilia A with factor VIII activity less than 2%
- Positive for factor VIII inhibitors
- No allergic reactions to factor VIII concentrates
You will not qualify if you...
- Presence of other coagulation-related diseases
- Hematological disorders
- Autoimmune diseases
- Malignancies
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 24 months
Participants receive immune tolerance induction treatment with different doses and frequencies of FVIII (ADVATE) to clear inhibitors and restore factor VIII activity.
Regular visits according to treatment schedule
Duration - After treatment ends until study completion
Participants are monitored for inhibitor recurrence and long-term tolerance after completion of treatment.
Follow-up visits to monitor inhibitor status
Trial Site Locations
Total: 1 location
1
Beijing Children's hospitial
Beijing, Beijing Municipality, China, 100045
Actively Recruiting
Research Team
D
Dr. Wu
+
+86 010-59616623
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
5
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