1. Garadacimab significantly reduced the number of monthly attacks in patients with hereditary angioedema compared to placebo.
2. No serious adverse events were observed in the treatment group with garadacimab.
Evidence Rating Level: 1 (Excellent)
Study Rundown: Although relatively rare, hereditary angioedema is a debilitating disorder that leads to recurrent attacks of severe swelling in various parts of the body. A new potential prophylactic treatment for angioedema attacks is garadacimab, a monoclonal IgG antibody targeting activated FXII. In this randomized controlled trial, 32 patients were assigned to receive either placebo or 75 mg, 200 mg, or 600 mg of subcutaneous garadacimab. In a 12-week treatment period, the number of monthly attacks was greatly reduced in the garadacimab group compared to placebo with a 100% reduction in the 200 mg treatment group. Of note, garadacimab was well-tolerated with no serious adverse events. Limitations of this study include the small sample size, likely given the rareness of the disorder. Nonetheless, this study provides a potential option for prophylactic management of hereditary angioedema attacks.
In-Depth [randomized controlled trial]: This phase 2 trial took place across 12 research centers in four countries (Canada, Germany, Israel, and the USA). Inclusion criteria included patients with hereditary angioedema (HAE-C1-INH) aged 18-65 years. Patients enrolled were randomized 1:1:1:1 to either placebo, 75 mg, 200 mg, or 600 mg of subcutaneous garadacimab. The primary measured outcome was number of attacks per month experienced by patients. A total of 32 patients were randomized with a median age of 39.5 years with 56% female. During the 12-week treatment period, the rate of attacks was significantly reduced in the garadacimab groups compared to placebo. The median number of attacks was 4.6 in the placebo group, 0.0 with 75 mg, 0.0 with 200 mg, and 0.3 with 600 mg garadacimab. This translated to a 93% reduction in the 600 mg group (p=0.0003) and a 100% reduction in the 200 mg group (p=0.0002). There were no serious adverse events in either group, including no events of anaphylaxis, VTE, or bleeding.
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