Long-Term, Open-label Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE (CHAPTER-4)

August 31, 2026 updated by: Pharvaris Netherlands B.V.

A Long-term, Open-label Study to Evaluate the Safety and Efficacy of Orally Administered Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

The study consists of a Screening Period during which eligibility is confirmed (only for participants not rolling over within 28 days from a previous deucrictibant prophylactic study), a Treatment Period in which participants will receive open-label deucrictibant extended-release tablet once daily for approximately 130 weeks, followed by an End of Study visit after maximum 4 weeks. Participants will undergo regular safety (e.g. lab draws) and efficacy assessments, will complete an electronic diary daily, and also complete questionnaires at predefined timepoints during the study.

Study Type

Interventional

Enrollment (Estimated)

170

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Argentina
      • Corrientes, Argentina
        • Recruiting
        • Study Site
      • Pilar, Argentina
        • Recruiting
        • Study Site
      • Salta, Argentina
        • Recruiting
        • Study Site
  • Australia
      • Box Hill, Australia, 3128
        • Recruiting
        • Study Site
      • Parkville, Australia
        • Recruiting
        • Study Site
      • Perth, Australia
        • Recruiting
        • Study Site
      • Sydney, Australia
        • Recruiting
        • Study Site
  • Austria
      • Vienna, Austria
        • Recruiting
        • Study Site
  • Brazil
      • Ribeirão Preto, Brazil
        • Recruiting
        • Study Site
      • Salvador, Brazil
        • Recruiting
        • Study Site
      • Santo André, Brazil
        • Recruiting
        • Study Site
      • São Paulo, Brazil, 01454010
        • Recruiting
        • Study Site
      • São Paulo, Brazil, 14051-140
        • Recruiting
        • Study Site
  • Bulgaria
      • Sofia, Bulgaria
        • Recruiting
        • Study Site
  • Canada
      • Edmonton, Canada
        • Recruiting
        • Study Site
      • Montreal, Canada
        • Recruiting
        • Study Site
  • China
      • Beijing, China
        • Recruiting
        • Study Site
  • France
      • Grenoble, France
        • Recruiting
        • Study Site
  • Germany
      • Berlin, Germany
        • Recruiting
        • Study Site
      • Frankfurt, Germany
        • Recruiting
        • Study Site
      • Munich, Germany
        • Recruiting
        • Study Site
  • Hong Kong
      • Hong Kong, Hong Kong
        • Recruiting
        • Study Site
  • Hungary
      • Budapest, Hungary
        • Recruiting
        • Study Site
  • Ireland
      • Dublin, Ireland
        • Recruiting
        • Study Site
  • Italy
      • Milan, Italy
        • Recruiting
        • Study Site
      • Padova, Italy
        • Recruiting
        • Study Site
      • Palermo, Italy
        • Recruiting
        • Study Site
      • Roma, Italy
        • Recruiting
        • Study Site
  • Japan
      • Fukuoka, Japan
        • Recruiting
        • Study Site
      • Hiroshima, Japan
        • Recruiting
        • Study Site
      • Kanagawa, Japan
        • Recruiting
        • Study Site
      • Osaka, Japan
        • Recruiting
        • Study Site
      • Tokyo, Japan
        • Recruiting
        • Study Site
  • New Zealand
      • Auckland, New Zealand
        • Recruiting
        • Study Site
  • Poland
      • Krakow, Poland
        • Recruiting
        • Study Site
  • Slovakia
      • Martin, Slovakia
        • Recruiting
        • Study Site
  • South Africa
      • Cape Town, South Africa
        • Recruiting
        • Study Site
  • South Korea
      • Daegu, South Korea
        • Recruiting
        • Study Site
      • Seoul, South Korea
        • Recruiting
        • Study Site
  • Spain
      • Barcelona, Spain, 08907
        • Recruiting
        • Study Site
  • Turkey (Türkiye)
      • Ankara, Turkey (Türkiye)
        • Recruiting
        • Study Site
      • Istanbul, Turkey (Türkiye)
        • Recruiting
        • Study Site
      • Izmir, Turkey (Türkiye)
        • Recruiting
        • Study Site
  • United Kingdom
      • Birmingham, United Kingdom, B18 7QH
        • Recruiting
        • Study Site
      • Birmingham, United Kingdom, B9 5SS
        • Recruiting
        • Study Site
      • Brighton, United Kingdom
        • Recruiting
        • Study Site
      • Bristol, United Kingdom
        • Recruiting
        • Study Site
      • Cambridge, United Kingdom
        • Recruiting
        • Study Site
      • Frimley, United Kingdom
        • Recruiting
        • Study Site
      • London, United Kingdom, E1 1FR
        • Recruiting
        • Study Site
      • London, United Kingdom
        • Recruiting
        • Study Site
      • Oxford, United Kingdom
        • Recruiting
        • Study Site
      • Plymouth, United Kingdom
        • Recruiting
        • Study Site
      • Southampton, United Kingdom
        • Recruiting
        • Study Site
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35209
        • Recruiting
        • Study Site
    • Arizona
      • Scottsdale, Arizona, United States, 85251
        • Recruiting
        • Study Site
    • Arkansas
      • Little Rock, Arkansas, United States, 72205
        • Recruiting
        • Study Site
    • California
      • San Diego, California, United States, 92122
        • Recruiting
        • Study Site
      • Santa Monica, California, United States, 90404
        • Recruiting
        • Study Site
      • Walnut Creek, California, United States, 94598
        • Recruiting
        • Study Site
    • Colorado
      • Colorado Springs, Colorado, United States, 80907
        • Recruiting
        • Study Site
    • Illinois
      • Wheaton, Illinois, United States, 20902
        • Recruiting
        • Study Site
    • Missouri
      • St Louis, Missouri, United States, 63141
        • Recruiting
        • Study Site
    • Nevada
      • Las Vegas, Nevada, United States, 89128-0450
        • Recruiting
        • Study Site
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Recruiting
        • Study Site
    • Texas
      • Dallas, Texas, United States, 75231
        • Recruiting
        • Study Site
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226-3067
        • Recruiting
        • Study Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Provision of the signed ICF by the participant and/or legally designated representative.
  2. Male or female, aged ≥12 years at the time of providing written informed consent/assent.
  3. Diagnosis of hereditary angioedema (HAE)
  4. 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
  5. Reliable access and ability to use standard of care on-demand treatments to effectively manage acute HAE attacks.
  6. Willing and able to adhere to all protocol requirements, including eDiary and ePRO data recording.
  7. Female participants of childbearing must agree to the protocol specified pregnancy testing and contraception methods.

Exclusion Criteria:

  1. Any diagnosis of angioedema other than HAE
  2. 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)
  3. Prior gene therapy for any indication at any time
  4. 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
  5. Exposure to ACE inhibitors or any estrogen-containing medications with systemic absorption within 4 weeks of Screening
  6. 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
  7. Any females who are pregnant, plan to become pregnant, or are currently breast-feeding
  8. Abnormal hepatic function
  9. Moderate or severe renal impairment
  10. Any clinically significant comorbidity or systemic dysfunction that would interfere with the participant's safety or ability to participate in the study.
  11. History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse
  12. 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
  13. Known hypersensitivity to deucrictibant or any of the excipients of the study drug

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Deucrictibant
Drug: Deucrictibant
Deucrictibant extended-release tablet for once daily oral use

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs), adverse events of special interest (AESIs), and TEAEs leading to study drug discontinuation
Time Frame: 130 weeks
130 weeks
Change in heart rate
Time Frame: 130 weeks
130 weeks
Change in blood pressure
Time Frame: 130 weeks
130 weeks
Change in body temperature
Time Frame: 130 weeks
130 weeks
Change in clinical laboratory tests from baseline
Time Frame: 130 weeks
Hematology, blood chemistry, and urinalysis. Descriptive in nature, no formal statistical hypothesis testing will be performed.
130 weeks
Change in electrocardiograms (ECGs) from baseline
Time Frame: 130 weeks
Digital triplicate 12-lead ECG. Descriptive in nature, no formal statistical hypothesis testing will be performed.
130 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Time-normalized number of Investigator-confirmed HAE attacks during the Treatment Period
Time Frame: 130 weeks
130 weeks
Time-normalized number of Investigator-confirmed HAE attacks treated with on-demand medication during the Treatment Period
Time Frame: 130 weeks
130 weeks
Time-normalized number of Investigator-confirmed moderate or severe HAE attacks during Treatment Period
Time Frame: 130 weeks
130 weeks
Time-normalized number of Investigator-confirmed severe HAE attacks during the Treatment Period
Time Frame: 130 weeks
130 weeks
Proportion of time without angioedema symptoms during the Treatment Period
Time Frame: 130 weeks
130 weeks
Patient reported outcome: Angioedema Quality of Life (AE-QoL) questionnaire
Time Frame: 130 weeks
The AE-QoL is a short 17-item questionnaire designed to retrospectively assess HRQoL, with a recall period of 4 weeks. Its results can be displayed as a total score or as 4 domain scores. The scores range from 0 to 100, after linear transformation of raw values, with higher scores indicating higher HRQoL impairment.
130 weeks
Patient reported outcome: Patient Global Assessment of Change (PGA-Change)
Time Frame: 130 weeks
PGA-Change assesses on a 5-point scale how the participant's QoL has been impacted by HAE since start taking the study drug.
130 weeks
Patient reported outcome: Angioedema Control Test 4-week version (AECT-4wk)
Time Frame: 130 weeks
AECT-4wk measures disease control retrospectively, it comprises 4 questions over a 4-week recall period. Scores for the responses in the AECT range from 0 to 16, with higher scores indicating better disease control (≤ 9 poorly controlled; ≥ 10 well controlled).
130 weeks
Patient reported outcome: Work Productivity and Activity Impairment Questionnaire - Specific Health Problem (WPAI-SHP)
Time Frame: 130 weeks
WPAI-SHP is a questionnaire assessing how a health condition impacts a person's ability to work and do regular activities and it includes 4 domains. Scores indicate the percentage of time the patient missed work or was less productive owing to HAE-related complications.
130 weeks
Patient reported outcome: Abbreviated Treatment Satisfaction Questionnaire for Medication (TSQM-9)
Time Frame: 130 weeks
TSQM-9 is a 9-item questionnaire evaluating patient treatment satisfaction and it includes 3 domains. Scoring is by domain and each domain score is computed by summing the individual TSQM items in each domain and then transforming the composite score into a value ranging from 0 to 100, with higher scores indicating higher satisfaction.
130 weeks
Pharmacokinetics [PK]: Pre-dose plasma concentration of deucrictibant and deucrictibant metabolites (Ctrough)
Time Frame: 130 weeks
130 weeks
Pharmacokinetics [PK]: Urine concentration of deucrictibant and deucrictibant metabolites
Time Frame: 130 weeks
130 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Pharvaris Netherlands B.V.

Investigators

  • Study Director: Study Director, Pharvaris, Pharvaris Netherlands B.V.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 1, 2025

Primary Completion (Estimated)

December 1, 2028

Study Completion (Estimated)

December 1, 2028

Study Registration Dates

First Submitted

October 24, 2024

First Submitted That Met QC Criteria

November 5, 2024

First Posted (Actual)

November 8, 2024

Study Record Updates

Last Update Posted (Actual)

September 1, 2026

Last Update Submitted That Met QC Criteria

August 31, 2026

Last Verified

August 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PHA022121-C307

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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