Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE

The study consists of a Screening Period during which eligibility is confirmed, a Treatment Period of 24 weeks, and a Follow-up Period of maximum 4 weeks or subjects may roll over into the open-label study PHA022121-C307 (CHAPTER-4). During the Treatment period participants will receive blinded study drug (deucrictibant or placebo randomized in a 2:1 ratio). Participants will undergo regular efficacy and safety assessments, complete an electronic diary daily, and also complete questionnaires at predefined timepoints during the study.

Inclusion criteria

• Provision of written informed consent/assent.
• Male or female, aged ≥12 years at the time of providing written informed consent/assent.
• Diagnosis of hereditary angioedema (HAE)
• History of at least 3 HAE attacks within the 3 consecutive months prior to Screening Visit
• Predefined number of attacks during the Screening Period
• Reliable access and ability to use standard of care on-demand treatments to effectively manage acute HAE attacks.
• Willing and able to adhere to all protocol requirements, including eDiary and ePRO data recording.
• Female participants of childbearing potential must agree to the protocol specified pregnancy testing and contraception methods.

Exclusion criteria

• Any diagnosis of angioedema other than HAE
• Participation in a clinical study with any other investigational drug within the last 30 days or within 5 half-lives of the investigational drug at Screening (whichever is longer)
• Has received prior prophylactic treatment with deucrictibant
• Exposure to ACE inhibitors or any estrogen-containing medications with systemic absorption within 4 weeks of Screening
• Prior gene therapy for any indication at any time
• Use of prophylactic treatment for HAE within 2 weeks of Screening for C1INH, oral kallikrein inhibitors, or anti-fibrinolytics; within 4 weeks of Screening for attenuated androgens; within 5 half-lives of Screening for monoclonal antibodies, or within 7 days of Screening for short-term prophylaxis
• Any females who are pregnant, plan to become pregnant, or are currently breast-feeding
• Abnormal hepatic function
• Moderate or severe renal impairment
• Any clinically significant comorbidity or systemic dysfunction that would interfere with the participant's safety or ability to participate in the study.
• History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse
• Use of medications that are moderate and strong inhibitors 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 deucrictibant or any of the excipients of the study drug