Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema

NCT ID: NCT04618211

Last Updated: 2025-12-17

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 74 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-02-03
• Study Completion Date: 2023-03-01

Brief Summary

This study evaluates the efficacy of orally administered deucrictibant for the acute treatment of attacks in patients with hereditary angioedema (HAE). Eligible subjects are randomized to one of three single doses of deucrictibant and placebo. The study will compare symptom relief (skin pain, skin swelling, abdominal pain) during HAE attacks and safety of each dose of deucrictibant with placebo.

Detailed Description

In Part I of the study, patients in non-attack state receive the assigned active single dose of deucrictibant at the study center to assess pharmacokinetics (the way the body absorbs, distributes, and gets rid of the drug) and safety. In Part II of the study, patients self-administer blinded study drug at home to treat three HAE attacks with deucrictibant or placebo (cross-over).

Conditions

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Low dose/placebo

Single low dose of deucrictibant or placebo

Group Type: OTHER

Deucrictibant

Intervention Type: DRUG

deucrictibant soft capsules for oral use

Placebo

Intervention Type: DRUG

Matching placebo capsules for oral use

Medium dose/placebo

Single medium dose of deucrictibant or placebo

Group Type: OTHER

Deucrictibant

Intervention Type: DRUG

deucrictibant soft capsules for oral use

Placebo

Intervention Type: DRUG

Matching placebo capsules for oral use

High dose/placebo

Single high dose of deucrictibant or placebo

Group Type: OTHER

Deucrictibant

Intervention Type: DRUG

deucrictibant soft capsules for oral use

Placebo

Intervention Type: DRUG

Matching placebo capsules for oral use

Interventions

Deucrictibant

deucrictibant soft capsules for oral use

Intervention Type: DRUG

Placebo

Matching placebo capsules for oral use

Intervention Type: DRUG

Other Intervention Names

PHA-022121; PHA121; PHVS416

Eligibility Criteria

Inclusion Criteria

1. Signed and dated informed consent form

2. Diagnosis of HAE type I or II

3. Documented history of HAE attacks: at least three in the last 4 months, or at least two in the last 2 months prior to screening

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

Exclusion Criteria

1. Pregnancy or breast-feeding

2. Clinically significant abnormal electrocardiogram

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

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

5. Positive serology for HIV or active infection with hepatitis B virus or hepatitis C virus

6. Abnormal hepatic function

7. Abnormal renal function

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

9. History of documented severe hypersensitivity to any medicinal product

10. Participation in any other investigational drug study within defined period

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Pharvaris Netherlands B.V.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Marcus Maurer, Prof MD

Role: PRINCIPAL_INVESTIGATOR

Charite University, Berlin, Germany

Locations

Study site

Birmingham, Alabama, United States

Study site

Paradise Valley, Arizona, United States

Study site

San Diego, California, United States

Study site

Santa Monica, California, United States

Study site

Walnut Creek, California, United States

Study site

Chevy Chase, Maryland, United States

Study site

St Louis, Missouri, United States

Study site

Hershey, Pennsylvania, United States

Study site

Sofia, , Bulgaria

Study site

Edmonton, Alberta, Canada

Study site

Ottawa, Ontario, Canada

Study site

Toronto, Ontario, Canada

Study site

Montreal, Quebec, Canada

Study site

Québec, Quebec, Canada

Study site

Brno, , Czechia

Study site

Hradec Králové, , Czechia

Study site

Grenoble, , France

Study site

Montpellier, , France

Study site

Paris, , France

Study site

Berlin, , Germany

Study site

Dresden, , Germany

Study site

Frankfurt, , Germany

Study site

Mainz, , Germany

Study site

Ulm, , Germany

Study site

Budapest, , Hungary

Study site

Ashkelon, , Israel

Study site

Haifa, , Israel

Study site

Tel Aviv, , Israel

Study site

Monserrato, , Italy

Study site

Naples, , Italy

Study site

Amsterdam, , Netherlands

Study site

Krakow, , Poland

Study site

Barcelona, , Spain

Study site

Madrid, , Spain

Study site

Brighton, , United Kingdom

Study site

London, , United Kingdom

Countries

United States; Bulgaria; Canada; Czechia; France; Germany; Hungary; Israel; Italy; Netherlands; Poland; Spain; United Kingdom

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2020-003445-11

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

PHA022121-C201

Identifier Type: -

Identifier Source: org_study_id