Long-Term, Open-label Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE

NCT06679881 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: PHA022121-C307
• Study Type: interventional
• Target: 170
• Locations: 21 countries
• Sites: 61

Brief Summary

This is a Phase 3, multicenter, long-term, open-label study to evaluate the safety and efficacy of once-daily orally administered deucrictibant extended-release tablet 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 (6)

• Treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs), adverse events of special interest (AESIs), and TEAEs leading to study drug discontinuation

  130 weeks

• Change in heart rate

  130 weeks

• Change in blood pressure

  130 weeks

• Change in body temperature

  130 weeks

• Change in clinical laboratory tests from baseline

  Hematology, blood chemistry, and urinalysis. Descriptive in nature, no formal statistical hypothesis testing will be performed.

  130 weeks

• Change in electrocardiograms (ECGs) from baseline

  Digital triplicate 12-lead ECG. Descriptive in nature, no formal statistical hypothesis testing will be performed.

  130 weeks

Secondary Outcomes (12)

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

  130 weeks

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

  130 weeks

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

  130 weeks

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

  130 weeks

• Proportion of time without angioedema symptoms during the Treatment Period

  130 weeks

Study Arms (1)

Deucrictibant

EXPERIMENTAL

Deucrictibant

Drug: Deucrictibant

Interventions

Deucrictibant DRUG

Deucrictibant extended-release tablet for once daily oral use

Deucrictibant

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 the signed ICF by the participant and/or legally designated representative.
• Male or female, aged ≥12 years at the time of providing written informed consent/assent.
• Diagnosis of hereditary angioedema (HAE)
• For participants that did not participate in a previous deucrictibant prophylactic study: history of at least 1 attack in the last 3 consecutive months prior to Screening
• 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 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 ICF signature (whichever is longer)
• Prior gene therapy for any indication at any time
• Participants who discontinued from previous studies with deucrictibant prophylactic and/or on-demand treatment due to safety reasons or compliance issues that, in the opinion of the Investigator, would interfere with the participant's safety or compliance to participate in the study
• Exposure to ACE inhibitors or any estrogen-containing medications with systemic absorption within 4 weeks of Screening
• 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 (62)

Study Site

Birmingham, Alabama, 35209, United States

RECRUITING

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Scottsdale, Arizona, 85251, United States

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Little Rock, Arkansas, 72205, United States

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San Diego, California, 92122, United States

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Santa Monica, California, 90404, United States

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Walnut Creek, California, 94598, United States

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Wheaton, Maryland, 20902, United States

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St Louis, Missouri, 63141, United States

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Las Vegas, Nevada, 89128-0450, United States

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Hershey, Pennsylvania, 17033, United States

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Dallas, Texas, 75231, United States

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Milwaukee, Wisconsin, 53226-3067, United States

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Corrientes, Argentina

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Pilar, Argentina

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Salta, Argentina

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Melbourne, 3052, Australia

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Melbourne, Australia

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Perth, Australia

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Sydney, Australia

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Vienna, Austria

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Ribeirão Preto, Brazil

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Salvador, Brazil

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Santo André, Brazil

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São Paulo, 01454010, Brazil

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São Paulo, 14051-140, Brazil

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Sofia, Bulgaria

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Edmonton, Canada

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Montreal, Canada

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Beijing, China

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Grenoble, France

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Berlin, Germany

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Frankfurt, Germany

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Munich, 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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Padova, Italy

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Palermo, Italy

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Roma, Italy

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Hiroshima, Japan

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Kanagawa, Japan

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Osaka, Japan

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Tokyo, Japan

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Krakow, Poland

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Martin, Slovakia

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Cape Town, South Africa

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Daegu, South Korea

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Seoul, South Korea

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Barcelona, 08907, Spain

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Ankara, Turkey (Türkiye)

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Istanbul, Turkey (Türkiye)

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Izmir, Turkey (Türkiye)

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Birmingham, B18 7QH, United Kingdom

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Birmingham, B9 5SS, United Kingdom

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Brighton, United Kingdom

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Bristol, United Kingdom

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Cambridge, United Kingdom

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London, E1 1FR, United Kingdom

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London, United Kingdom

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Oxford, United Kingdom

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Plymouth, United Kingdom

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Southampton, United Kingdom

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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

Central Study Contacts

Pharvaris Clinical Team

CONTACT

0031 712-036-410; clinicaltrials@pharvaris.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 24, 2024
• First Posted: November 8, 2024
• Study Start: February 1, 2025
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2028
• Last Updated: May 7, 2026

Record last verified: 2026-05

Locations

BU (1); HK (1); IE (1); CA (2); PL (1); AU (4); US (12); IT (3); ES (1); KR (2); DE (3); GB (10); ZA (1); SL (1); HU (1); FR (1); JP (4); TU (3); AR (3); BR (5); CN (1)