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

Phase 2

Active, not recruiting

• Conditions: Hereditary Angioedema Types I and II; Hereditary Angioedema With C1 Esterase Inhibitor Deficiency; Hereditary Angioedema - Type 2; Hereditary Angioedema; Hereditary Angioedema Type I; Hereditary Angioedema Type II; Hereditary Angioedema Attack; Hereditary Angioedema - Type 1; C1 Inhibitor Deficiency; C1 Esterase Inhibitor Deficiency
• Interventions: Drug: Deucrictibant high dose; Drug: Deucrictibant low dose; Drug: Placebo

• Registration Number: NCT05047185
• Lead Sponsor: Pharvaris Netherlands B.V.

Brief 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 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.

Detailed Description

Not available

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations
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Related News

Study Timeline

• Study First Submitted: 2021-09-07
• Study First Submitted QC: 2021-09-07
• Study First Posted: 2021-09-17
• Study Start: 2022-04-19
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-05
• Last Update Posted: 2024-11-07
• Primary Completion: 2025-06
• Study Completion: 2025-09

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 34

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Part 2: Open-label	Deucrictibant high dose	BID high dose of deucrictibant
Part 1: Low dose	Deucrictibant low dose	BID low dose of deucrictibant
Part 1: High dose	Deucrictibant high dose	BID high dose of deucrictibant
Part 1: Placebo	Placebo	BID placebo

Primary Outcome Measures

Name	Time	Method
Number of investigator-confirmed HAE attacks	Day 0 to Day 84

Secondary Outcome Measures

Name	Time	Method
Number of patients that are attack-free during the treatment period	Day 0 to Day 84
Number and proportion of days with angioedema symptoms during the treatment period	Day 0 to Day 84
Time to first investigator-confirmed HAE attack in the treatment period	Day 0 to Day 84
Number of investigator-confirmed angioedema attacks during the treatment period in Part 2.	Day 84 to Day 938
Number of investigator-confirmed moderate or severe angioedema attacks during the treatment period in Part 2.	Day 84 to Day 938
Number of investigator-confirmed moderate or severe HAE attacks during the treatment period	Day 0 to Day 84
Number of investigator-confirmed HAE attacks requiring acute treatment during the treatment period	Day 0 to Day 84
Number of patients achieving reduction in attack rate during the treatment period relative to baseline	Day 0 to Day 84
Number of investigator-confirmed HAE attacks resulting in a visit to the emergency department or an admission to hospital	Day 0 to Day 84
Number of investigator-confirmed angioedema attacks requiring acute treatment during the treatment period in Part 2.	Day 84 to Day 938
Incidence of HAE attacks during the treatment period in Part 2 (attack rate trend over time).	Day 84 to Day 938
Number and proportion of days with angioedema symptoms during the treatment period in Part 2.	Day 84 to Day 938

Trial Locations

Locations (2)

Study Site; 🇦🇹 Wien, Austria

Study site; 🇬🇧 Southampton, England, United Kingdom