Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE

NCT06669754 | Active Not Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: PHA022121-C305
• Study Type: interventional
• Target: 81
• Locations: 24 countries
• Sites: 56

Brief Summary

This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of once-daily orally administered deucrictibant extended-release tablet compared to placebo for prophylaxis to prevent angioedema attacks in participants aged ≥ 12 years with hereditary angioedema.

Conditions

Hereditary Angioedema (HAE)

Keywords

HAE; Oral Treatment; Bradykinin B2 Receptor Antagonists; PHVS719; PHA121; Deucrictibant; Prophylaxis

Outcome Measures

Primary Outcomes (1)

• Time-normalized (per 4 weeks) number of Investigator-confirmed HAE attacks during the 24-week Treatment Period

  24 weeks

Secondary Outcomes (15)

• Time-normalized number of Investigator-confirmed HAE attacks treated with on-demand medication during the 24-week Treatment Period

  24 weeks

• Time-normalized number of Investigator-confirmed moderate or severe HAE attacks during the 24-week Treatment Period

  24 weeks

• Time-normalized number of Investigator-confirmed severe HAE attacks during the 24-week Treatment Period

  24 weeks

• Proportion of participants achieving ≥50% reduction in HAE attack rate relative to baseline during the 24-week Treatment Period

  24 weeks

• Proportion of participants achieving ≥70% reduction in HAE attack rate relative to baseline during the 24-week Treatment Period

  24 weeks

Study Arms (2)

Active

EXPERIMENTAL

Deucrictibant 40mg extended-release tablet by mouth once daily

Drug: Deucrictibant

Placebo

EXPERIMENTAL

Placebo 1 tablet by mouth once daily

Drug: Placebo

Interventions

Placebo DRUG

Placebo

Placebo

Deucrictibant DRUG

Deucrictibant 40mg extended-release tablet for once daily oral use

Active

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Provision of written informed consent/assent.
• Male or female, aged ≥12 years at the time of providing written informed consent/assent.
• Diagnosis of hereditary angioedema (HAE)
• History of at least 3 HAE attacks within the 3 consecutive months prior to Screening Visit
• Predefined number of attacks during the Screening Period
• Reliable access and ability to use standard of care on-demand treatments to effectively manage acute HAE attacks.
• Willing and able to adhere to all protocol requirements, including eDiary and ePRO data recording.
• Female participants of childbearing potential must agree to the protocol specified pregnancy testing and contraception methods.

You may not qualify if:

• Any diagnosis of angioedema other than HAE
• Participation in a clinical study with any other investigational drug within the last 30 days or within 5 half-lives of the investigational drug at Screening (whichever is longer)
• Has received prior prophylactic treatment with deucrictibant
• Exposure to ACE inhibitors or any estrogen-containing medications with systemic absorption within 4 weeks of Screening
• Prior gene therapy for any indication at any time
• Use of prophylactic treatment for HAE within 2 weeks of Screening for C1INH, oral kallikrein inhibitors, or anti-fibrinolytics; within 4 weeks of Screening for attenuated androgens; within 5 half-lives of Screening for monoclonal antibodies, or within 7 days of Screening for short-term prophylaxis
• Any females who are pregnant, plan to become pregnant, or are currently breast-feeding
• Abnormal hepatic function
• Moderate or severe renal impairment
• Any clinically significant comorbidity or systemic dysfunction that would interfere with the participant's safety or ability to participate in the study.
• History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse
• Use of medications that are moderate and strong inhibitors or strong inducers of CYP3A4 within the last 30 days or within 5 half-lives (whichever is longer) of the time of randomization
• Known hypersensitivity to deucrictibant or any of the excipients of the study drug

Sponsors & Collaborators

• Pharvaris Netherlands B.V.: lead

Study Sites (56)

Study Site

Little Rock, Arkansas, 72205, United States

Location

Study Site

San Diego, California, 92122, United States

Location

Study site

Santa Monica, California, 90404, United States

Location

Study site

Walnut Creek, California, 94598, United States

Location

Study Site

Chevy Chase, Maryland, 20915, United States

Location

Study Site

St Louis, Missouri, 63141, United States

Location

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Corrientes, Argentina

Location

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San Martín, Argentina

Location

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Santo André, Brazil

Location

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Sofia, 1431, Bulgaria

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Sofia, 1680, Bulgaria

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Edmonton, Canada

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Ottawa, Canada

Location

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Grenoble, France

Location

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Lille, 59037, France

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Berlin, Germany

Location

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Frankfurt, Germany

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Hanover, Germany

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Hong Kong, Hong Kong

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Budapest, Hungary

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Dublin, Ireland

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Milan, 20062, Italy

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Padua, Italy

Location

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Kawasaki, Japan

Location

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Tokyo, 113-0033, Japan

Location

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Tokyo, 1130033, Japan

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Auckland, New Zealand

Location

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Krakow, Poland

Location

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San Juan, Puerto Rico

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Sângeorgiu de Mureş, Romania

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Singapore, Singapore

Location

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Martin, Slovakia

Location

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Cape Town, South Africa

Location

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Daegu, South Korea

Location

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Seoul, 06351, South Korea

Location

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Seoul, South Korea

Location

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Suwon, South Korea

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Barcelona, 08013, Spain

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Barcelona, 08907, Spain

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Seville, Spain

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Basel, Switzerland

Location

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Ankara, Turkey (Türkiye)

Location

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Istanbul, Turkey (Türkiye)

Location

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Izmir, Turkey (Türkiye)

Location

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London, England, E1 1FR, United Kingdom

Location

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Birmingham, B18 7QH, United Kingdom

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Birmingham, B9 5SS, United Kingdom

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Bristol, BS10 5NB, United Kingdom

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Cambridge, CB2 0QQ, United Kingdom

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Frimley, GU16 7UJ, United Kingdom

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Leeds, LS9 7TF, United Kingdom

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London, NW3 2QG, United Kingdom

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Oxford, United Kingdom

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Plymouth, PL6 5FP, United Kingdom

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Southampton, United Kingdom

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Stoke, United Kingdom

Location

MeSH Terms

Conditions

Angioedemas, Hereditary

Condition Hierarchy (Ancestors)

Angioedema; Vascular Diseases; Cardiovascular Diseases; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Immunologic Deficiency Syndromes

Study Officials

• Study Director, Pharvaris

  Pharvaris Netherlands B.V.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 24, 2024
• First Posted: November 1, 2024
• Study Start: December 1, 2024
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): August 1, 2026
• Last Updated: February 13, 2026

Record last verified: 2026-02

Locations

BR (1); ES (3); HK (1); JP (3); CA (2); PR (1); RO (1); SG (1); SL (1); KR (4); CH (1); ZA (1); NZ (1); PL (1); AR (2); BU (2); TU (3); FR (2); DE (3); IT (2); IE (1); HU (1); GB (12); US (6)