HAVEN 1 and HAVEN 2 study design

2 multicenter, open-label pivotal studies of patients with hemophilia A with FVIII inhibitors

HEMLIBRA dosing: 3 mg/kg once weekly for the first 4 weeks followed by 1.5 mg/kg once weekly

*An earlier prospective, observational, non-interventional study was conducted to collect detailed, high-quality, real-world data on bleeding events and safety outcomes in patients with hemophilia A treated according to local routine clinical practice. Patients who previously participated in a non-interventional study and then entered the HAVEN 1 or HAVEN 2 trials allowed for the intra-patient comparison of bleed rate with HEMLIBRA prophylaxis vs prior prophylactic treatment with bypassing agents. 

7 patients who previously participated in the non-interventional study and were unable to enter HAVEN 1 prior to enrollment closure were enrolled in a separate arm to receive HEMLIBRA prophylaxis.

HAVEN 2 also included 3 patients aged 12-17 years weighing under 40 kg.

§2 patients aged under 2 years enrolled after data cutoff in HAVEN 2 and were not included in the interim analysis.

At the time of the interim analysis, 57 patients were included in the safety analysis: 55 (29%) were children (2 years to under 12 years), and two (1%) were infants (1 month to under 2 years). At the time of the interim analysis, efficacy was evaluated in 23 patients with at least 12 weeks on treatment, including 19 patients aged 6 to under 12 years and 4 patients aged 2 to under 6 years. There were 13 pediatric patients who participated in the NIS-evaluated intra-patient analysis.

HAVEN 1 and HAVEN 2 study endpoints

  •  Treated bleeds were defined as bleeds treated with bypassing agents based on an adaptation of standard criteria defined by the International Society on Thrombosis and Haemostasis (ISTH) 

Evaluated over time (minimum of 24 weeks or date of discontinuation). Median observation time was 24 (3–47.9) weeks, with an open-label extension following 24 weeks.
#The median observation time for these patients was 38.1 (12.7–41.6) weeks.
**Patients aged under 12 years were evaluable for efficacy.


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