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.
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.).
Any diagnosis of angioedema other than BK-AE-nC1INH.
Participation in a clinical study with any other investigational drug within the previous 30 days or within 5 half-lives of the investigational drug at Screening (whichever was longer).
Exposure to angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications with systemic absorption (such as oral contraceptives or hormonal replacement therapy) within 4 weeks of Screening.
Any females who are pregnant, plan to become pregnant, or are currently breast-feeding.
Crossover
Deucrictibant XR tablet
DrugDeucrictibant 40 mg XR tablet for prophylaxis
Placebo comparator to XR tablet
DrugPlacebo comparator to deucrictibant 40 mg XR tablet, prophylaxis
Deucrictibant 20 mg capsule
DrugDeucrictibant active drug for on-demand treatment of angioedma attacks
Placebo comparator to 20 mg capsule
DrugPlacebo comparator to Deucrictibant 20 mg capsule for on-demand treatment of angioedema attacks
Part-1: Incidence and quality of HAE attacks comparing active versus placebo arms.
Part-1: Time-normalized number of Investigator-confirmed moderate or severe angioedema attacks during the Treatment Period. Time-normalized number of Investigator-confirmed angioedema attacks treated with on-demand medication during the Treatment Period.
Part-2: Efficacy of active on-demand treatment versus placebo in achieving angioedema symptom relief during acute attacks
Part-2: Time to onset of symptom relief of active drug versus placebo, defined as Patient Global Impression of Change (PGI-C) rating of at least "a little better" for 2 consecutive timepoints within 12 hours post-treatment. Time to complete symptom resolution, defined as achieving PGI-S rating of "none" within 48 hours post-treatment.
Safety assessment of oral deucrictibant extended release (XR) tablet (Part 1), and deucrictibant oral on-demand tablets (Part 2).
Adverse event severity, expectedness, and relatedness of the active drug, classified based on their severity, whether they were expected, and their potential relationship to the study intervention (Parts 1 and 2).
Part-1: Change in Health-Related Quality of Life (HRQoL) Scores Between Active Drug and Placebo Periods
Assessment of quality of life will be conducted using a validated disease-specific instrument, such as the Hereditary Angioedema Quality of Life Questionnaire (HAE-QoL), and supplemented by a daily patient diary Daily diary entries will record the presence or absence of attacks, their severity, duration, body location, treatment used, time to resolution, and the impact on daily living (missed work/school, physical limitations, mood, and social activities).documenting physical, emotional, and functional impacts of each attack.
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