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

Completed · Phase 2 · Has a placebo group

Conditions studied: Hereditary Angioedema, Hereditary Angioedema Type I, Hereditary Angioedema Type II, Hereditary Angioedema Types I and II, Hereditary Angioedema Attack, Hereditary Angioedema With C1 Esterase Inhibitor Deficiency, Hereditary Angioedema - Type 1, Hereditary Angioedema - Type 2, C1 Esterase Inhibitor Deficiency, C1 Inhibitor Deficiency

In brief

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 consists of 2 parts, with patients completing participation in Part 1 prior to initiation of treatment in Part 2. Part 1 of the study has 3 parallel arms and approximately 30 patients will be equally randomized to one of two dose regimens of PHA-022121 or matching placebo. Patients will continue to the single open-label arm in Part 2 of the study after completion of Part 1. The screening period is up to 8 weeks and the treatment periods are 12 weeks (Part 1) and 30 months (Part 2) in duration.

Key facts

Study ID

NCT05047185

Run by

Pharvaris Netherlands B.V.

People needed

34

Starts

2022-04-19

Expected to finish

2025-06-30

Last updated by the study team

2025-07-30

Who can join

Age: 18 and older, up to 75. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Study site — Birmingham, Alabama, United States
• Study site — Paradise Valley, Arizona, United States
• Study site — St Louis, Missouri, United States
• Study Site — Vienna, Austria
• Study site — Sofia, Bulgaria
• Study site — Ottawa, Ontario, Canada
• Study site — Montreal, Quebec, Canada
• Study site — Berlin, Germany
• Study site — Frankfurt, Germany
• Study site — Dublin, Ireland
• Study site — Padua, PD, Italy
• Study site — Milan, Italy
• Study site — Palermo, Italy
• Study site — Krakow, Poland
• Study site — Brighton, England, United Kingdom
• Study site — Bristol, England, United Kingdom
• Study site — Cambridge, England, United Kingdom
• Study site — London, England, United Kingdom
• Study site — Southampton, England, United Kingdom

Full record on ClinicalTrials.gov

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