The efficacy of HEMLIBRA was studied in 2 clinical trials—HAVEN 1 and HAVEN 2—in adults, adolescents, and children with hemophilia A with factor VIII inhibitors. HAVEN 2 is an ongoing study, so the information shown reflects interim results. The primary goal of each study was to determine how well HEMLIBRA worked in reducing the number of treated bleeds. Treated bleeds were defined as any bleed that received treatment with hemophilia medicine as determined by the individual, their caregiver, or their healthcare provider. The studies also evaluated the safety of HEMLIBRA, the effectiveness of HEMLIBRA compared to previous bypassing agent use, how well the medicine worked in reducing all bleeds (whether treated or not), and the number of treated bleeding episodes, including joint bleeds, spontaneous bleeds, and target joint bleeds.
Overview of HAVEN 1
- HEMLIBRA was studied in 109 adult and adolescent males with hemophilia A (12 to 75 years and weighing more than 40 kg) with factor VIII inhibitors. All had previously used bypassing agents, either as on-demand therapy or prophylaxis. The primary goal was to compare reduction in bleeds on HEMLIBRA to no prophylaxis
- The study was randomized, multicentered, and open-label. This means that participants were randomly assigned to separate groups for comparison across several different study locations and each participant and his healthcare provider knew what therapy the participant was taking
- Information from 3 treatment groups was used to evaluate how
well HEMLIBRA reduced treated bleeds. Each group (or arm) was
identified by a letter—A, B, or C. The results from these three
- Participants in Groups A and C took HEMLIBRA prophylaxis at a dose of 3 mg/kg once weekly for the first 4 weeks, followed by 1.5 mg/kg once weekly for the rest of the 24-week trial
- Participants in Group B received no
prophylaxis throughout the 24-week trial
Prior to HAVEN 1, Genentech conducted an observational study called the “Non-Interventional Study” or NIS. The NIS was conducted to collect detailed real-world information on bleeds and safety outcomes in people with hemophilia A. Treatment decisions during the study were made according to local routine clinical practice.
Eligible NIS participants could later enroll in HAVEN 1, allowing for a direct comparison of HEMLIBRA to prior bypassing agent use. This is called an “intra-patient comparison.”
- Twenty-four NIS participants who previously used bypassing agent prophylaxis switched to HEMLIBRA prophylaxis (Group C)
- Seven NIS participants were unable to enter groups A, B, or C because they were full. These individuals were enrolled in a separate group to receive HEMLIBRA prophylaxis. Their data was only included in the safety results
Overview of HAVEN 2
HEMLIBRA prophylaxis is being evaluated in a single-arm, multicenter, open-label study in children (younger than 12 or 12–17 years who weigh less than 40 kg) with hemophilia A with factor VIII inhibitors. (Single-arm means that everyone received the same therapy; in this case, HEMLIBRA.)
Whether previously taking bypassing agents prophylactically or on demand, all participants received HEMLIBRA prophylaxis at 3 mg/kg once weekly for the first 4 weeks followed by 1.5 mg/kg once weekly during the trial.
An interim analysis was done, meaning that information was collected while the trial was ongoing. The information does not reflect the final findings because the trial is not yet complete.
At the time of the interim analysis:
- 23 male participants who
were younger than 12 years old and had 12 or more weeks in the trial
were eligible for the efficacy analysis
- This included 19 participants who were between 6 and 12 years old and four participants who were between 2 and 6 years old
- The median observation time was approximately 38 weeks (ranging from 12–41 weeks)
The intra-patient analysis results are from 13 participants after at least 12 weeks of treatment. Twelve of the participants received previous bypassing agent (BPA) prophylaxis and one received on-demand BPA treatment. All 13 participants were previously enrolled in the NIS study.
To see the HAVEN 1 and HAVEN 2 clinical results, click here.