Dose-ranging Study of Oral PHA-022121 for Prophylaxis Against Angioedema Attacks in Patients With Hereditary Angioedema Type I or Type II (HAE CHAPTER-1)

Pharvaris

Status and phase: Completed; Phase 2

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

Treatments: Drug: Deucrictibant low dose; Drug: Placebo; Drug: Deucrictibant high dose

Study type: Interventional

Funder types: Industry

Identifiers

NCT05047185

PHA022121-C301

2021-000227-13 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment: 34 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Prevention

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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34 participants in 4 patient groups, including a placebo group

Part 1: Low dose

Experimental group

Description:

BID low dose of deucrictibant

Treatment:

Drug: Deucrictibant low dose

Part 1: High dose

Experimental group

Description:

BID high dose of deucrictibant

Treatment:

Drug: Deucrictibant high dose

Part 1: Placebo

Placebo Comparator group

Description:

BID placebo

Treatment:

Drug: Placebo

Part 2: Open-label

Experimental group

Description:

BID high dose of deucrictibant

Treatment:

Drug: Deucrictibant high dose