Treatment of Hemophilia A Patients With FVIII Inhibitors
- Conditions
- Hemophilia A
- Registration Number
- NCT04023019
- Lead Sponsor
- Emory University
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Male
- Target Recruitment
- Not specified
Inclusion Criteria:<br><br> - Male persons with haemophilia A, of any severity, who have a historical inhibitor<br> titer = 0.6 BU/mL, including those who have failed previous immune tolerance<br> induction (ITI) attempt(s)<br><br> - Persons undergoing ITI with Nuwiq, octanate, or wilateor undergoing ITI with Nuwiq®,<br> octanate® or wilate® and receiving prophylactic therapy with emicizumab, activated<br> prothrombin complex concentrate (aPCC), or activated recombinant factor VII (rFVIIa)<br><br> - Participants or participants' parent(s)/legal guardian(s) must be capable of giving<br> signed informed consent and be able to understand the trial documents<br><br>Exclusion Criteria:<br><br> - Participants are excluded from the trial if any coagulation disorder other than<br> haemophilia A is diagnosed<br><br> - Partly retrospective patients will be excluded if detailed documentation on<br> treatment, all bleeding episodes, inhibitor titers, and FVIII levels is not<br> available for the retrospective period
Not provided
Study & Design
- Study Type
- Observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Proportion of participants achieving inhibitor titer < 0.6 Bethesda units (BU)/mL L for at least 2 consecutive measurements;Proportion of participants achieving FVIII recovery = 66% of the predefined reference value of 1.5% IU/kg body weight (Groups 1 and 2);Proportion of participants achieving FVIII half-life = 6 h (Groups 1 and 2);Annualized bleeding rate
- Secondary Outcome Measures
Name Time Method Time to achieve immune tolerance induction outcome;Frequency of emicizumab, aPCC, and rFVIIa use during immune tolerance induction;Rate of FVIII inhibitor relapse;Frequency of bleeding episodes;Severity of bleeding episodes;Number of infusions required to control bleeding episodes;Frequency of bleeding with surgical procedures;Severity of bleeding with surgical procedures;Proportion of participants experiencing adverse drug reactions;Number of thrombotic events;Treatment costs