Long-Term, Open-label 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 (only for participants not rolling over within 28 days from a previous deucrictibant prophylactic study), a Treatment Period in which participants will receive open-label deucrictibant extended-release tablet once daily for approximately 130 weeks, followed by an End of Study visit after maximum 4 weeks. Participants will undergo regular safety (e.g. lab draws) and efficacy assessments, will complete an electronic diary daily, and also complete questionnaires at predefined timepoints during the study.

Inclusion criteria

• Provision of the signed ICF by the participant and/or legally designated representative.
• Male or female, aged ≥12 years at the time of providing written informed consent/assent.
• Diagnosis of hereditary angioedema (HAE)
• For participants that did not participate in a previous deucrictibant prophylactic study: history of at least 1 attack in the last 3 consecutive months prior to Screening
• 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 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 ICF signature (whichever is longer)
• Prior gene therapy for any indication at any time
• Participants who discontinued from previous studies with deucrictibant prophylactic and/or on-demand treatment due to safety reasons or compliance issues that, in the opinion of the Investigator, would interfere with the participant's safety or compliance to participate in the study
• Exposure to ACE inhibitors or any estrogen-containing medications with systemic absorption within 4 weeks of Screening
• 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