Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema

The study consists of a Screening Phase during which eligibility is confirmed, a Treatment Phase in which participants will be randomized and receive double blinded study drug to treat 2 qualifying HAE attacks (i.e., 2 Treatment Periods within the Treatment Phase), and an End-of-Study Follow-up Phase after the second attack treated with study drug. In addition, for adolescent participants (age ≥12 to <18 years), PK samples are collected after administration of deucrictibant at Day 1 in a non-attack state.

Inclusion criteria

• Provision of written informed consent/assent.
• Male or female, aged ≥12 to ≤75 years at the time of providing written informed consent/assent.
• Diagnosis of HAE-1/2/3.
• History of at least 2 HAE attacks in the last 3 months before screening.
• Experience with using standard-of-care treatment to effectively manage on-demand treatment for HAE attacks.
• Participants on long-term prophylactic therapy with plasma-derived C1-INH (danazol, anti-fibrinolytics, berotralstat, or lanadelumab) must be on a stable dose and regimen and intend to remain on the same dose for 6 months before screening and the duration of the study. OR, Participant has stopped using plasma-derived C1-INH (danazol, anti-fibrinolytics, berotralstat) at least 2 weeks or lanadelumab at least 10 weeks before screening.
• Capable of recording, without assistance, electronic HAE diary and ePRO data using an electronic device.
• For adolescent participants aged ≥12 and <18 years of age: body weight ≥40 kg.
• Female participants of childbearing potential must agree to the protocol specified pregnancy testing and contraception methods.

Exclusion criteria

• Any female who is pregnant, plans to become pregnant, or is breastfeeding.
• Any diagnosis of angioedema other than HAE.
• Any clinically significant comorbidity or systemic dysfunction that would interfere with the participant's safety or ability to participate in the study.
• Use of attenuated androgens for short-term prophylaxis within 2 weeks before screening.
• Abnormal hepatic function.
• Abnormal renal function (eGFR <60 ml/min/1.73 m2).
• History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse.
• Has received prior on-demand HAE treatment with deucrictibant.
• Currently participating in any other investigational drug study or receiving other investigational treatment within the last 30 days, or within 5 half-lives (whichever is longer) of the time of randomization.
• Prior gene therapy for any indication at any time.
• Use of concomitant medications with systemic absorption that are strong inhibitors of CYP3A4 or strong inducers of CYP3A4 within the last 30 days, or within 5 half-lives (whichever is longer) of the time of randomization.
• Known hypersensitivity to study drug or any of the excipients of study drug.