Oral Deucrictibant for the Prophylactic and Acute Treatment in Patients With Bradykinin Mediated Angioedema With Normal C1 Inhibitor (BK-AE-nC1INH)
To assess the efficacy of prophylactic treatment with deucrictibant extended release (XR) tablet versus placebo in preventing angioedema attacks, and to also assess the efficacy of deucrictibant soft capsules as on-demand treatment versus placebo in achieving angioedema symptom relief during acute attacks.
• Provision of written informed consent.
• Male or female, aged ≥18 at the time of provision of informed consent.
• Diagnosis of bradykinin-mediated angioedema based upon all of the following:
‣ Clinical history consistent with angioedema (subcutaneous or mucosal, nonpruritic swelling without accompanying urticaria), not responsive to treatments of anti-histamine, corticosteroid, and/or omalizumab.
⁃ Tried and failed at least 2 weeks of cetirizine 20 mg twice a day (or its equivalent alternative antihistamines, such as fexofenadine, loratadine, desloratadine or levocetirizine, etc.).
⁃ Total blood BK peptide levels following 3 days cold activation is above the diagnostic value in non-attack and/or attack period\*.
‣ \*The attack period is defined as within 24 hours after an attack.
⁃ Documented diagnostic testing results: C1INH antigen concentration and functional activity within normal range; C4 antigen concentration within normal range.
• Documented history of at least 2 angioedema attacks in the previous 2 months.
• Reliable access and experience to use standard of care medication to effectively manage acute angioedema attacks.