Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema

NCT ID: NCT06343779

Last Updated: 2025-11-21

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 134 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-02-26
• Study Completion Date: 2025-10-17

Brief Summary

This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled, 2-period, 2-treatment cross-over study to evaluate the efficacy and safety of orally administered deucrictibant compared to placebo for the on-demand treatment of HAE attacks, including non-severe laryngeal attacks, in participants ≥12 to ≤75 years of age with HAE type 1, type 2, or type 3, a proportion of whom are using long-term prophylactic medication for HAE.

Detailed Description

The study consists of a Screening Phase during which eligibility is confirmed, a Treatment Phase in which participants will be randomized and receive double blinded study drug to treat 2 qualifying HAE attacks (i.e., 2 Treatment Periods within the Treatment Phase), and an End-of-Study Follow-up Phase after the second attack treated with study drug. In addition, for adolescent participants (age ≥12 to <18 years), PK samples are collected after administration of deucrictibant at Day 1 in a non-attack state.

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 [C1-INH] Deficiency; C1 Esterase Inhibitor Deficiency; C1 Esterase Inhibitor, Deficiency of; C1 Inhibitor Deficiency; Hereditary Angioedema - Type 3; Hereditary Angioedema Type III

Study Design

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

Study Groups

Arm 1

Deucrictibant administered for first HAE attack, placebo administered for second HAE attack.

Group Type: EXPERIMENTAL

Deucrictibant, Placebo

Intervention Type: DRUG

Deucrictibant Soft Capsules for Oral Use

Arm 2

Placebo administered for first HAE attack, deucrictibant administered for second HAE attack.

Group Type: EXPERIMENTAL

Deucrictibant, Placebo

Intervention Type: DRUG

Deucrictibant Soft Capsules for Oral Use

Interventions

Deucrictibant, Placebo

Deucrictibant Soft Capsules for Oral Use

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Provision of written informed consent/assent.

2. Male or female, aged ≥12 to ≤75 years at the time of providing written informed consent/assent.

3. Diagnosis of HAE-1/2/3.

4. History of at least 2 HAE attacks in the last 3 months before screening.

5. Experience with using standard-of-care treatment to effectively manage on-demand treatment for HAE attacks.

6. Participants on long-term prophylactic therapy with plasma-derived C1-INH (danazol, anti-fibrinolytics, berotralstat, or lanadelumab) must be on a stable dose and regimen and intend to remain on the same dose for 6 months before screening and the duration of the study. OR, Participant has stopped using plasma-derived C1-INH (danazol, anti-fibrinolytics, berotralstat) at least 2 weeks or lanadelumab at least 10 weeks before screening.

7. Capable of recording, without assistance, electronic HAE diary and ePRO data using an electronic device.

8. For adolescent participants aged ≥12 and <18 years of age: body weight ≥40 kg.

9. Female participants of childbearing potential must agree to the protocol specified pregnancy testing and contraception methods.

Exclusion Criteria

1. Any female who is pregnant, plans to become pregnant, or is breastfeeding.

2. Any diagnosis of angioedema other than HAE.

3. Any clinically significant comorbidity or systemic dysfunction that would interfere with the participant's safety or ability to participate in the study.

4. Use of attenuated androgens for short-term prophylaxis within 2 weeks before screening.

5. Abnormal hepatic function.

6. Abnormal renal function (eGFR <60 ml/min/1.73 m2).

7. History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse.

8. Has received prior on-demand HAE treatment with deucrictibant.

9. Currently participating in any other investigational drug study or receiving other investigational treatment within the last 30 days, or within 5 half-lives (whichever is longer) of the time of randomization.

10. Prior gene therapy for any indication at any time.

11. Use of concomitant medications with systemic absorption that are strong inhibitors of CYP3A4 or strong inducers of CYP3A4 within the last 30 days, or within 5 half-lives (whichever is longer) of the time of randomization.

12. Known hypersensitivity to study drug or any of the excipients of study drug.

• Minimum Eligible Age: 12 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

Study Director, Pharvaris

Role: STUDY_DIRECTOR

Pharvaris Netherlands B.V.

Locations

Study Site

Birmingham, Alabama, United States

Study Site

Paradise Valley, Arizona, United States

Study Site

Little Rock, Arkansas, 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

Colorado Springs, Colorado, United States

Study Site

Chevy Chase, Maryland, United States

Study Site

Boston, Massachusetts, United States

Study Site

Detroit, Michigan, United States

Study Site

St Louis, Missouri, United States

Study Site

Hershey, Pennsylvania, United States

Study Site

Dallas, Texas, United States

Study Site

Buenos Aires, , Argentina

Study Site

Salta, , Argentina

Study Site

Campbelltown, New South Wales, Australia

Study Site

Box Hill, , Australia

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Graz, , Austria

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Linz, , Austria

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Vienna, , Austria

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Paraná, , Brazil

Study Site

Ribeirão Preto, , Brazil

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Salvador, , Brazil

Study Site

Santo André, , Brazil

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São Paulo, , Brazil

Study Site

Sofia, , Bulgaria

Study Site

Sofia, , Bulgaria

Study Site

Edmonton, Alberta, Canada

Study Site

Bogotá, , Colombia

Study Site

Bogotá, , Colombia

Study Site

Bogotá, , Colombia

Study Site

Medellín, , Colombia

Study Site

Brno, , Czechia

Study Site

Lille, , France

Study Site

Paris, , France

Study Site

Berlin, , Germany

Study Site

Frankfurt am Main, , Germany

Study Site

Frankfurt am Main, , Germany

Study Site

Lübeck, , Germany

Study Site

Hong Kong, , Hong Kong

Study Site

Budapest, , Hungary

Study Site

Dublin, , Ireland

Study Site

Catania, , Italy

Study Site

Milan, , Italy

Study Site

Milan, , Italy

Study Site

Padua, , Italy

Study Site

Palermo, , Italy

Study Site

Roma, , Italy

Study Site

Chiba, , Japan

Study Site

Hiroshima, , Japan

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Kanagawa, , Japan

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Osaka, , Japan

Study Site

Tokyo, , Japan

Study Site

Amsterdam, , Netherlands

Study Site

Skopje, , North Macedonia

Study Site

Krakow, , Poland

Study Site

San Juan, , Puerto Rico

Study Site

San Juan, , Puerto Rico

Study Site

Sângeorgiu de Mureş, , Romania

Study Site

Riyadh, , Saudi Arabia

Study Site

Singapore, , Singapore

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Cape Town, , South Africa

Study Site

Daegu, , South Korea

Study Site

Seoul, , South Korea

Study Site

Barcelona, , Spain

Study Site

Barcelona, , Spain

Study Site

Lund, , Sweden

Study Site

Ankara, , Turkey (Türkiye)

Study Site

Istanbul, , Turkey (Türkiye)

Study Site

Izmir, , Turkey (Türkiye)

Study Site

Bristol, , United Kingdom

Study Site

Camberley, , United Kingdom

Study Site

Cambridge, , United Kingdom

Study Site

Leeds, , United Kingdom

Study Site

London, , United Kingdom

Study Site

Plymouth, , United Kingdom

Countries

United States; Argentina; Australia; Austria; Brazil; Bulgaria; Canada; Colombia; Czechia; France; Germany; Hong Kong; Hungary; Ireland; Italy; Japan; Netherlands; North Macedonia; Poland; Puerto Rico; Romania; Saudi Arabia; Singapore; South Africa; South Korea; Spain; Sweden; Turkey (Türkiye); United Kingdom

Other Identifiers

PHA022121-C306

Identifier Type: -

Identifier Source: org_study_id