Overview
This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of once-daily orally administered deucrictibant extended-release tablet compared to placebo for prophylaxis to prevent angioedema attacks in participants aged ≥ 12 years with hereditary angioedema.
Description
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.
Eligibility
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