Dose-ranging Study of Oral PHA-022121 for Prophylaxis Against Angioedema Attacks in Patients With Hereditary Angioedema Type I or Type II

• Status: Recruiting
• Trial end date: 2022-12-01
• Target enrollment: 0
• Participant gender: All

Summary

This study evaluates the safety and efficacy of PHA-022121 administered orally for prophylaxis against angioedema attacks in patients with hereditary angioedema (HAE). The study will have 3 parallel arms and approximately 30 patients will be equally randomized to one of two dose regimens of PHA-022121 or matching placebo. The screening period is up to 8 weeks and the treatment period is 12 weeks in duration. The study will assess the efficacy of PHA-022121 as prophylaxis against HAE attacks, and safety of each dose of PHA-022121 compared with placebo.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Pharvaris Netherlands B.V.

Criteria

Inclusion Criteria:

• Signed and dated informed consent form

• Diagnosis of HAE type I or II

• Documented history of at least 3 HAE attacks within the last 3 consecutive months prior to screening, or a minimum of 2 HAE attacks during the screening period

• Reliable access and experience to use standard of care acute attack medications

Exclusion Criteria:

• Pregnancy or breast-feeding

• Clinically significant abnormal electrocardiogram

• Any other systemic disease or significant disease or disorder that would interfere with the patient's safety or ability to participate in the study

• Use of C1-esterase inhibitor, oral kallikrein inhibitors, attenuated androgens, anti-fibrinolytics, or monoclonal HAE therapy within a defined period prior to enrolment

• Abnormal hepatic function

• Abnormal renal function

• History of alcohol or drug abuse within defined period, or current evidence of substance dependence or abuse

• Participation in any other investigational drug study within defined period