Hereditary Angioedema (HAE)Phase 3CHAPTER-4NCT06679881
Long-Term, Open-label Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE
A Phase 3 study testing Deucrictibant in people with Hereditary Angioedema (HAE), run by Pharvaris Netherlands B.V. at 67 sites in 23 countries.
Trial 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 Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hereditary Angioedema (HAE)
- What is being tested?
- Deucrictibant
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 170 participants
- Where
- 67 sitesin 23 countries
- Study dates
- 1 Feb 2025to Dec 2028
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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 the participant being capable of and compliant with data recording into an eDiary.
- Female participants of childbearing age must agree to the protocol specified pregnancy testing and contraception methods.
Who cannot join
- 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 angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications with systemic absorption (such as oral contraceptives or hormonal replacement therapy) 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
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- 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 enrollment.
- Known hypersensitivity to deucrictibant or any of the excipients of the study drug
Trial locations and contacts
67 sites in 23 countries. Contact the study team or a site near you to ask about taking part.
67 sites in 23 countries
United States13 sites
Study Site
Birmingham, Alabama, 35209
Study Site
Colorado Springs, Colorado, 80907
Study Site
Dallas, Texas, 75231
Study Site
Hershey, Pennsylvania, 17033
Study Site
Las Vegas, Nevada, 89128-0450
Study Site
Little Rock, Arkansas, 72205
Study Site
Milwaukee, Wisconsin, 53226-3067
Study Site
San Diego, California, 92122
Study Site
Santa Monica, California, 90404
Study Site
Scottsdale, Arizona, 85251
Study Site
St Louis, Missouri, 63141
Study Site
Walnut Creek, California, 94598
Study Site
Wheaton, Illinois, 20902
United Kingdom11 sites
Study Site
Birmingham, B18 7QH
Study Site
Birmingham, B9 5SS
Study Site
Brighton
Study Site
Bristol
Study Site
Cambridge
Study Site
Frimley
Study Site
London, E1 1FR
Study Site
London
Study Site
Oxford
Study Site
Plymouth
Study Site
Southampton
Brazil5 sites
Study Site
Ribeirão Preto
Study Site
Salvador
Study Site
Santo André
Study Site
São Paulo, 01454010
Study Site
São Paulo, 14051-140
Japan5 sites
Study Site
Fukuoka
Study Site
Hiroshima
Study Site
Kanagawa
Study Site
Osaka
Study Site
Tokyo
Australia4 sites
Study Site
Box Hill, 3128
Study Site
Parkville
Study Site
Perth
Study Site
Sydney
Turkey (Türkiye)3 sites
Study Site
Ankara
Study Site
Istanbul
Study Site
Izmir
About this study
Official title: 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
Read the 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 240 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, and also complete questionnaires at predefined timepoints during the study.
- Conditions
- Hereditary Angioedema (HAE)
- Being tested
- Deucrictibant
- Keywords
- HAE, Oral Treatment, Bradykinin B2 Receptor Antagonists, PHVS719, PHA121, Deucrictibant, Prophylaxis
- Medical subject headings
- Angioedemas, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DeucrictibantDrug
Deucrictibant extended-release tablet for once daily oral use
| Study group | Role | What they receive |
|---|---|---|
| Deucrictibant | Experimental | Deucrictibant Receives: Drug: Deucrictibant |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs), adverse events of special interest (AESIs), and TEAEs leading to study drug discontinuation
Measured: Up to Week 240
Change in heart rate
Measured: Up to Week 240
Change in blood pressure
Measured: Up to Week 240
Change in body temperature
Measured: Up to Week 240
Number of participants with clinically significant changes in Hematology
Blood samples will be collected for the analysis of hematology parameters.
Measured: Up to Week 240
Number of participants with clinically significant changes in Clinical Chemistry
Blood samples will be collected for the analysis of clinical chemistry parameters.
Measured: Up to Week 240
Number of participants with clinically significant changes in Urinalysis
Urine samples will be collected for the analysis of urinalysis parameters.
Measured: Up to Week 240
Change from Baseline electrocardiograms (ECGs)
Digital triplicate 12-lead ECG. Descriptive in nature, no formal statistical hypothesis testing will be performed.
Measured: Up to Week 240
Secondary outcomes (12)
Time-normalized number of Investigator-confirmed HAE attacks during the Treatment Period
Measured: Up to Week 240
Time-normalized number of Investigator-confirmed HAE attacks treated with on-demand medication during the Treatment Period
Measured: Up to Week 240
Time-normalized number of Investigator-confirmed moderate or severe HAE attacks during Treatment Period
Measured: Up to Week 240
Time-normalized number of Investigator-confirmed severe HAE attacks during the Treatment Period
Measured: Up to Week 240
Proportion of time without angioedema symptoms during the Treatment Period
Measured: Up to Week 240
Patient reported outcome: Angioedema Quality of Life (AE-QoL) questionnaire
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.
Measured: Up to Week 132
Patient reported outcome: Patient Global Assessment of Change (PGA-Change)
PGA-Change assesses on a 5-point scale how the participant's QoL has been impacted by HAE since start taking the study drug.
Measured: Up to Week 132
Patient reported outcome: Angioedema Control Test 4-week version (AECT-4wk)
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).
Measured: Up to Week 132
Patient reported outcome: Work Productivity and Activity Impairment Questionnaire - Specific Health Problem (WPAI-SHP)
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.
Measured: Up to Week 132
Patient reported outcome: Abbreviated Treatment Satisfaction Questionnaire for Medication (TSQM-9)
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.
Measured: Up to Week 132
Pharmacokinetics [PK]: Pre-dose plasma concentration of deucrictibant and deucrictibant metabolites (Ctrough)
Measured: Up to Week 96
Pharmacokinetics [PK]: Urine concentration of deucrictibant and deucrictibant metabolites
Measured: Up to Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Nov 2024
- Study start1 Feb 2025
- Primary completionDec 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Angioedema (HAE)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 170 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Pharvaris Netherlands B.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Pharvaris, Study director, Pharvaris Netherlands B.V.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06679881
- Sponsor's protocol ID
- PHA022121-C307
- Acronym
- CHAPTER-4
- Status last verified
- Sep 2026
- First submitted
- 24 Oct 2024
- Last updated
- 22 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06679881
- Acronym / sponsor ID
- CHAPTER-4 / PHA022121-C307
- Conditions
- Hereditary Angioedema (HAE)
- Keywords
- HAE, Oral Treatment, Bradykinin B2 Receptor Antagonists, PHVS719, PHA121, Deucrictibant, Prophylaxis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 170 participants (estimated)
- Lead sponsor
- Pharvaris Netherlands B.V.Industry (pharma, biotech, devices)
- Study start
- 1 Feb 2025 (actual)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 8 Nov 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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 240 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, and also complete questionnaires at predefined timepoints during the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Deucrictibant | Experimental | Drug: DeucrictibantDeucrictibant |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (13 listed)
- 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 angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications with systemic absorption (such as oral contraceptives or hormonal replacement therapy) within 4 weeks of Screening
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DeucrictibantDeucrictibant extended-release tablet for once daily oral use | Drug | Deucrictibant |
Primary outcome
Number of participants with 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: Up to Week 240
Change in heart rate
Time frame: Up to Week 240
Change in blood pressure
Time frame: Up to Week 240
Change in body temperature
Time frame: Up to Week 240
Number of participants with clinically significant changes in Hematology
Time frame: Up to Week 240
Blood samples will be collected for the analysis of hematology parameters.
Number of participants with clinically significant changes in Clinical Chemistry
Time frame: Up to Week 240
Blood samples will be collected for the analysis of clinical chemistry parameters.
Number of participants with clinically significant changes in Urinalysis
Time frame: Up to Week 240
Urine samples will be collected for the analysis of urinalysis parameters.
Change from Baseline electrocardiograms (ECGs)
Time frame: Up to Week 240
Digital triplicate 12-lead ECG. Descriptive in nature, no formal statistical hypothesis testing will be performed.
12 secondary outcomes
Time-normalized number of Investigator-confirmed HAE attacks during the Treatment Period
Time frame: Up to Week 240
Time-normalized number of Investigator-confirmed HAE attacks treated with on-demand medication during the Treatment Period
Time frame: Up to Week 240
Time-normalized number of Investigator-confirmed moderate or severe HAE attacks during Treatment Period
Time frame: Up to Week 240
Time-normalized number of Investigator-confirmed severe HAE attacks during the Treatment Period
Time frame: Up to Week 240
Proportion of time without angioedema symptoms during the Treatment Period
Time frame: Up to Week 240
Patient reported outcome: Angioedema Quality of Life (AE-QoL) questionnaire
Time frame: Up to Week 132
Patient reported outcome: Patient Global Assessment of Change (PGA-Change)
Time frame: Up to Week 132
Patient reported outcome: Angioedema Control Test 4-week version (AECT-4wk)
Time frame: Up to Week 132
Patient reported outcome: Work Productivity and Activity Impairment Questionnaire - Specific Health Problem (WPAI-SHP)
Time frame: Up to Week 132
Patient reported outcome: Abbreviated Treatment Satisfaction Questionnaire for Medication (TSQM-9)
Time frame: Up to Week 132
Pharmacokinetics [PK]: Pre-dose plasma concentration of deucrictibant and deucrictibant metabolites (Ctrough)
Time frame: Up to Week 96
Pharmacokinetics [PK]: Urine concentration of deucrictibant and deucrictibant metabolites
Time frame: Up to Week 48
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Sep 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Study Director, Pharvaris study director
Pharvaris Netherlands B.V.
67 sites in 23 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Deucrictibant in people with Hereditary Angioedema (HAE), run by Pharvaris Netherlands B.V. at 67 sites in 23 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 22 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 67 sites in United States, United Kingdom, Brazil, Japan, Australia and 18 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with 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: Up to Week 240). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Sep 2026.