Hereditary AngioedemaPhase 2/3RAPIDe-2NCT05396105
Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema
A Phase 2/3 study testing deucrictibant in people with Hereditary Angioedema and Hereditary Angioedema Type I, run by Pharvaris Netherlands B.V. at 60 sites in 24 countries.
Trial summary
This study evaluates the safety and efficacy of long-term on-demand treatment with orally administered deucrictibant for acute hereditary angioedema (HAE) attacks, including laryngeal attacks. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hereditary Angioedema, Hereditary Angioedema Type I, Hereditary Angioedema Type II
- 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
- 150 participants
- Where
- 60 sitesin 24 countries
- Study dates
- 28 Dec 2022to Jun 2027
- Phase
- Phase 2/3
- 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 informed consent form by the participant and/ or legally designated representative. If the participant is a minor (i.e., <18 years of age or as determined by local law), consent will be obtained from the participant's parent/legally designated representative/guardian and signed assent will be obtained from the participant, per country regulations.
- For participants from Study C201, received at least one dose of study drug (including the non-attack visit) in Study C201. For participants from Study C306, participant was randomized (and for adolescent participants ≥12 to <18 years received a dose of study drug in a non-attack state at Visit 1) and completed Study C306, with 2 attacks treated, or after closure of that study by the Sponsor. Enrollment of adolescents (≥12 to <18 years or age of adulthood as defined locally) from these studies is with consideration of local age requirements.
- Female participants of childbearing potential (or who become of childbearing potential during the study) must agree to the protocol-specified pregnancy testing and to be abstinent from heterosexual intercourse or to use an acceptable contraception method as defined in the protocol and as available locally from enrollment until 30 days after the last study drug administration.
- In the opinion of the Investigator, the participant (and parent/caregiver for adolescent participants) is willing and able to comply with the protocol.
- Adult participants with HAE type III (HAE-nC1INH) who are deucrictibant-treatment naïve, must meet all of the following:
- Recurrent angioedema attacks with diagnostic testing results obtained during screening to confirm C1INH function ≥50% of normal and C4 level not below the lower level of the normal range performed by the central laboratory.
Show all 13Show fewer
- Must either have:
- Documented genetic mutation associated with HAE-nC1INH as listed in the Hereditary Angioedema Association (HAEA) and World Allergy Organization (WAO)/European Academy of Allergy and Clinical Immunology (EAACI) Guidelines.
- OR
- If no documented mutation: clinical diagnosis with family history of HAE-nC1INH and documented elevations of bradykinin levels in blood. (US, UK and Canada only).
- Attacks not responding to treatments with high-dose antihistamine (cetirizine 40 mg/day or equivalent high-dose second-generation antihistamine medication) and no clinical attack symptoms relief if treated with corticosteroid, montelukast, or omalizumab.
- Documented effective attack symptom relief with on-demand icatibant treatment.
- A history of at least 1 HAE attack in the last 3 months prior to Screening
Who cannot join
- Any female who is pregnant, plans to become pregnant, or is breast-feeding.
- Any other systemic disease (e.g., cardiovascular, gastrointestinal, renal, respiratory, neurological) or significant disease or disorder that, in the opinion of the Investigator, would interfere with the participant's safety or ability to participate in the study.
- Use of lanadelumab for long-term HAE prophylactic therapy within 12 weeks prior to enrollment in Part A.
- Participants who have recently used short or long-term HAE prophylaxis or on-demand HAE treatment will not be excluded from the study provided the following washout period is observed (i.e., study screening or enrollment/rollover should be delayed allowing for washout):
- For Part A:
- 2-week washout period before enrollment should be respected for participants who have used any C1-INH product, oral kallikrein inhibitors, attenuated androgens, or anti-fibrinolytics for long-term prophylactic HAE therapy.
Show all 14Show fewer
- 1-week washout period before enrollment should be respected for participants who have used plasma derived C1-INH concentrates (Berinert, Cinryze, Haegarda) for on-demand treatment or short-term prophylaxis.
- 24-hour washout period before enrollment should be respected for participants who have used recombinant C1-INH (Ruconest) for on-demand treatment or short-term prophylaxis.
- For Part B:
- If a participant is receiving long-term prophylactic therapy with a medication indicated for HAE:, eg, plasma-derived C1INH, danazol at less than or equal to 200 mg/day, antifibrinolytics, berotralstat, or lanadelumab, they must be on a stable dose and regimen for at least 3 months before screening and intends to remain on the same dose for the duration of the study.
- History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse
- Participation in any other investigational drug study within (except with deucrictibant) currently, within the last 30 days prior to the first deucrictibant dose or within 5 half-lives of study drug at enrollment, whichever is longer.
- Discontinued from parent study after enrollment for any study drug-related safety reason or non-compliance including significant protocol deviation.
- Use of concomitant medications that are strong CYP3A4 inhibitors (e.g., clarithromycin, erythromycin, itraconazole, ketoconazole, ritonavir) or strong CYP3A4 inducers (e.g., carbamazepine and phenytoin).
Trial locations and contacts
60 sites in 24 countries. Contact the study team or a site near you to ask about taking part.
60 sites in 24 countries
United States12 sites
Study site
Birmingham, Alabama, 35209
Study site
Boston, Massachusetts, 02115
Study site
Chevy Chase, Maryland, 20815
Study site
Colorado Springs, Colorado, 80907
Study site
Dallas, Texas, 75231
Study site
Detroit, Michigan, 48202
Study site
Hershey, Pennsylvania, 17033
Study site
Little Rock, Arkansas, 72205
Study site
San Diego, California, 92122
Study site
Santa Monica, California, 90404
Study site
St Louis, Missouri, 63141
Study site
Walnut Creek, California, 94598
Italy7 sites
Study site
Catania, 95124
Study site
Milan, 20097
Study site
Milan, 20138
Study site
Naples, 80131
Study site
Padova, 35128
Study site
Palermo, 90146
Study site
Roma, 00133
Japan5 sites
Study site
Hiroshima, 730-8518
Study site
Kanagawa, 216-8511
Study site
Osaka, 569-8686
Study site
Tokyo, 113-8431
Study site
Tokyo, 133-8431
Brazil4 sites
Study site
Ribeirão Preto, 14048-900
Study site
Salvador, Estado de Bahia, 41950-640
Study site
Santo André, 09060-870
Study site
São Paulo, 05403-000
Germany4 sites
Study site
Berlin, 12203
Study site
Frankfurt am Main, 60590
Study site
Frankfurt am Main, 60596
Study site
Lübeck, 23538
Turkey (Türkiye)2 sites
Study site
Ankara, 06230
Study site
Istanbul, 34093
About this study
Official title: A Phase II/III, Extension Study of Orally Administered PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema
This study evaluates the safety and efficacy of long-term on-demand treatment with orally administered deucrictibant for acute hereditary angioedema (HAE) attacks, including laryngeal attacks. The study will enroll participants from Study PHA022121-C201 (NCT04618211), Study PHA022121-C306 (NCT06343779) and deucrictibant treatment naïve HAE-nC1INH adult participants who elect to participate in this extension study and meet the eligibility requirements.
Read the detailed description
Part A of the study will enroll adult participants from Study PHA022121-C201. The double-blind treatment assignment from Study PHA022121-C201 will be maintained.
Part B is open-label treatment and includes participants rolling over from Part A, participants from Study PHA022121-C201 who did not participate in Part A, participants aged 12 years and older with HAE type I, type II, or HAE-nC1INH rolling over from Study PHA022121-C306, and deucrictibant treatment naïve adult participants with HAE-nC1INH who elect to participate in this extension study and meet the eligibility requirements.
- 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
- Being tested
- deucrictibant
- Keywords
- HAE, HAE Type I, HAE Type II, Oral Treatment, Bradykinin B2 Receptor Antagonists, On-Demand, PHA121, PHVS416, PHA-022121, Deucrictibant, HAE Type III, HAE-nC1INH
- Medical subject headings
- Angioedemas, Hereditary, Hereditary Angioedema Types I and II, Hereditary Angioedema Type III
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
deucrictibantDrug
Also called PHVS416, PHA121, PHA-022121
3 capsules of deucrictibant or matching placebo will be administered orally for each HAE attack
deucrictibantDrug
Also called PHVS416, PHA121, PHA-022121
deucrictibant soft capsules will be administered orally for each HAE attack
| Study group | Role | What they receive |
|---|---|---|
| Part A: Deucrictibant, blinded dose | Experimental | Participants will receive the dose of deucrictibant they were randomized to in the PHA022121-C201 study (low, medium, or high dose, each consisting of 3 capsules of deucrictibant or matching placebo) for oral use for on-demand treatment of HAE attacks. Receives: Drug: deucrictibant |
| Part B: Deucrictibant, open-label | Experimental | Participants will receive deucrictibant soft capsules for oral use for on-demand treatment of HAE attacks. Receives: Drug: deucrictibant |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment-emergent Adverse Events (TEAEs), treatment-related TEAEs, treatment-emergent serious adverse events (TESAEs), treatment-related TESAEs, and TEAEs leading to deucrictibant discontinuation
Measured: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Heart Rate
Descriptive in nature, no formal statistical hypothesis testing will be performed.
Measured: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Blood pressure
Systolic and diastolic blood pressure will be measured. Descriptive in nature, no formal statistical hypothesis testing will be performed.
Measured: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Body temperature
Descriptive in nature, no formal statistical hypothesis testing will be performed.
Measured: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Clinical laboratory tests
hematology, blood chemistry, urinalysis
Measured: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Electrocardiograms
Measured: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Physical Examination
Measured: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Secondary outcomes (8)
Time to onset of symptom relief, defined as Patient Global Impression of Change (PGI-C) rating of at least "a little better" for 2 consecutive timepoints within 12 hours post-treatment
PGI-C evaluates the change in the attack symptoms over time with a 7-point response scale.
Measured: Assessed from 1 hour to 12 hours post-treatment
Time to substantial symptom relief, defined as achieving PGI-C rating of at least "better" for 2 consecutive timepoints within 12 hours post-treatment
PGI-C evaluates the change in the attack symptoms over time with a 7-point response scale.
Measured: Assessed from 1 hour to 12 hours post-treatment
Time to substantial symptom relief by Patient Global Impression of Severity (PGI-S), defined as achieving ≥1 point reduction in PGI-S from pre-treatment for 2 consecutive timepoints within 12 hours post-treatment
PGI-S evaluates the severity of attack symptoms with a 5-point response scale.
Measured: Assessed from pre-treatment to 12 hours post-treatment
Proportion of attacks achieving symptom resolution, defined as achieving PGI-S rating of "none" at 24 hours post-treatment.
Measured: At 24 hours post-treatment
Proportion of deucrictibant-treated attacks requiring rescue medication within 24 hours post-treatment
Measured: Assessed from pre-treatment to 24 hours post-treatment
Time to onset of symptom relief, assessed by a ≥30% reduction in VAS-3/ VAS-5 (Part A) or AMRA (Part B) composite score from the pre-treatment score
VAS/AMRA scores range between 0 and 100. A larger reduction means a better outcome.
Measured: Assessed from pre-treatment to 48 hours post-treatment
Time to substantial symptom relief by VAS-3/ VAS-5 (Part A) or AMRA (Part B), defined as a ≥50% reduction in VAS-3/ VAS-5 (Part A) or AMRA (Part B) composite score from pre-treatment for 2 consecutive timepoints within 12 hours post-treatment
VAS/AMRA scores range between 0 and 100. A larger reduction means a better outcome.
Measured: Assessed from pre-treatment to 12 hours post-treatment
Proportion of study drug-treated attacks reaching almost complete or complete symptom relief by VAS-3/ VAS-5 (Part A) or AMRA (Part B), defined as all item scores in VAS-3/ VAS-5/ AMRA having a value ≤10 at 24 hours post-treatment
Almost complete or complete symptom relief is defined as all individual item scores in VAS/AMRA having a value ≤10 sustained for 2 consecutive timepoints.
Measured: At 24 hours post-treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 May 2022
- Study start28 Dec 2022
- Primary completionJun 2027 (estimated)
- Study completionJun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary AngioedemaStudies 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 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Pharvaris Netherlands B.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Pharvaris Netherlands B.V.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05396105
- Sponsor's protocol ID
- PHA022121-C303
- Other IDs
- 2023-505766-28-00, 2023
- Acronym
- RAPIDe-2
- Status last verified
- Jun 2026
- First submitted
- 28 Apr 2022
- Last updated
- 21 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II/III, Extension Study of Orally Administered PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema
- ClinicalTrials.gov ID
- NCT05396105
- Acronym / sponsor ID
- RAPIDe-2 / PHA022121-C303
- 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
- Keywords
- HAE, HAE Type I, HAE Type II, Oral Treatment, Bradykinin B2 Receptor Antagonists, On-Demand, PHA121, PHVS416
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 150 participants (estimated)
- Lead sponsor
- Pharvaris Netherlands B.V.Industry (pharma, biotech, devices)
- Study start
- 28 Dec 2022 (actual)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- Jun 2027 (estimated)
- First posted
- 31 May 2022 (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 study evaluates the safety and efficacy of long-term on-demand treatment with orally administered deucrictibant for acute hereditary angioedema (HAE) attacks, including laryngeal attacks. The study will enroll participants from Study PHA022121-C201 (NCT04618211), Study PHA022121-C306 (NCT06343779) and deucrictibant treatment naïve HAE-nC1INH adult participants who elect to participate in this extension study and meet the eligibility requirements.
Detailed description
Part A of the study will enroll adult participants from Study PHA022121-C201. The double-blind treatment assignment from Study PHA022121-C201 will be maintained.
Part B is open-label treatment and includes participants rolling over from Part A, participants from Study PHA022121-C201 who did not participate in Part A, participants aged 12 years and older with HAE type I, type II, or HAE-nC1INH rolling over from Study PHA022121-C306, and deucrictibant treatment naïve adult participants with HAE-nC1INH who elect to participate in this extension study and meet the eligibility requirements.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Part A: Deucrictibant, blinded dose | Experimental | Drug: deucrictibantParticipants will receive the dose of deucrictibant they were randomized to in the PHA022121-C201 study (low, medium, or high dose, each consisting of 3 capsules of deucrictibant or matching placebo) for oral use for on-demand treatment of HAE attacks. |
| Part B: Deucrictibant, open-label | Experimental | Drug: deucrictibantParticipants will receive deucrictibant soft capsules for oral use for on-demand treatment of HAE attacks. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (13 listed)
- Provision of the signed informed consent form by the participant and/ or legally designated representative. If the participant is a minor (i.e., <18 years of age or as determined by local law), consent will be obtained from the participant's parent/legally designated representative/guardian and signed assent will be obtained from the participant, per country regulations.
- For participants from Study C201, received at least one dose of study drug (including the non-attack visit) in Study C201. For participants from Study C306, participant was randomized (and for adolescent participants ≥12 to <18 years received a dose of study drug in a non-attack state at Visit 1) and completed Study C306, with 2 attacks treated, or after closure of that study by the Sponsor. Enrollment of adolescents (≥12 to <18 years or age of adulthood as defined locally) from these studies is with consideration of local age requirements.
- Female participants of childbearing potential (or who become of childbearing potential during the study) must agree to the protocol-specified pregnancy testing and to be abstinent from heterosexual intercourse or to use an acceptable contraception method as defined in the protocol and as available locally from enrollment until 30 days after the last study drug administration.
- In the opinion of the Investigator, the participant (and parent/caregiver for adolescent participants) is willing and able to comply with the protocol.
- Adult participants with HAE type III (HAE-nC1INH) who are deucrictibant-treatment naïve, must meet all of the following:
Key exclusion criteria (14 listed)
- Any female who is pregnant, plans to become pregnant, or is breast-feeding.
- Any other systemic disease (e.g., cardiovascular, gastrointestinal, renal, respiratory, neurological) or significant disease or disorder that, in the opinion of the Investigator, would interfere with the participant's safety or ability to participate in the study.
- Use of lanadelumab for long-term HAE prophylactic therapy within 12 weeks prior to enrollment in Part A.
- Participants who have recently used short or long-term HAE prophylaxis or on-demand HAE treatment will not be excluded from the study provided the following washout period is observed (i.e., study screening or enrollment/rollover should be delayed allowing for washout):
- History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse
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 |
|---|---|---|
| deucrictibantAlso: PHVS416, PHA121, PHA-0221213 capsules of deucrictibant or matching placebo will be administered orally for each HAE attack | Drug | Part A: Deucrictibant, blinded dose |
| deucrictibantAlso: PHVS416, PHA121, PHA-022121deucrictibant soft capsules will be administered orally for each HAE attack | Drug | Part B: Deucrictibant, open-label |
Primary outcome
Treatment-emergent Adverse Events (TEAEs), treatment-related TEAEs, treatment-emergent serious adverse events (TESAEs), treatment-related TESAEs, and TEAEs leading to deucrictibant discontinuation
Time frame: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Heart Rate
Time frame: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Descriptive in nature, no formal statistical hypothesis testing will be performed.
Blood pressure
Time frame: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Systolic and diastolic blood pressure will be measured. Descriptive in nature, no formal statistical hypothesis testing will be performed.
Body temperature
Time frame: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Descriptive in nature, no formal statistical hypothesis testing will be performed.
Clinical laboratory tests
Time frame: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
hematology, blood chemistry, urinalysis
Electrocardiograms
Time frame: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Physical Examination
Time frame: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
8 secondary outcomes
Time to onset of symptom relief, defined as Patient Global Impression of Change (PGI-C) rating of at least "a little better" for 2 consecutive timepoints within 12 hours post-treatment
Time frame: Assessed from 1 hour to 12 hours post-treatment
Time to substantial symptom relief, defined as achieving PGI-C rating of at least "better" for 2 consecutive timepoints within 12 hours post-treatment
Time frame: Assessed from 1 hour to 12 hours post-treatment
Time to substantial symptom relief by Patient Global Impression of Severity (PGI-S), defined as achieving ≥1 point reduction in PGI-S from pre-treatment for 2 consecutive timepoints within 12 hours post-treatment
Time frame: Assessed from pre-treatment to 12 hours post-treatment
Proportion of attacks achieving symptom resolution, defined as achieving PGI-S rating of "none" at 24 hours post-treatment.
Time frame: At 24 hours post-treatment
Proportion of deucrictibant-treated attacks requiring rescue medication within 24 hours post-treatment
Time frame: Assessed from pre-treatment to 24 hours post-treatment
Time to onset of symptom relief, assessed by a ≥30% reduction in VAS-3/ VAS-5 (Part A) or AMRA (Part B) composite score from the pre-treatment score
Time frame: Assessed from pre-treatment to 48 hours post-treatment
Time to substantial symptom relief by VAS-3/ VAS-5 (Part A) or AMRA (Part B), defined as a ≥50% reduction in VAS-3/ VAS-5 (Part A) or AMRA (Part B) composite score from pre-treatment for 2 consecutive timepoints within 12 hours post-treatment
Time frame: Assessed from pre-treatment to 12 hours post-treatment
Proportion of study drug-treated attacks reaching almost complete or complete symptom relief by VAS-3/ VAS-5 (Part A) or AMRA (Part B), defined as all item scores in VAS-3/ VAS-5/ AMRA having a value ≤10 at 24 hours post-treatment
Time frame: At 24 hours post-treatment
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 21 Jul 2026.
- Individual participant dataSharing planned: No
Related trials for Hereditary Angioedema
RecruitingPhase 3
Donidalorsen Treatment in Children With Hereditary Angioedema
Hereditary Angioedema (HAE)
RecruitingObservational study
A Study of Takhzyro in Teenagers and Adults With Hereditary Angioedema (HAE) in South Korea
Hereditary Angioedema (HAE)
Active, not recruitingPhase 3
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
Hereditary Angioedema
RecruitingPhase 3
Study of IV Human Plasma-derived C1 Esterase Inhibitor Concentrate in Patients With Congenital C1-INH Deficiency for Treatment and Pre-procedure Preventing of Acute Hereditary Angioedema Attacks
Acute Hereditary Angio Edema
Active, not recruitingPhase 1/2
NTLA-2002 in Adults With Hereditary Angioedema (HAE)
Hereditary Angioedema
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 study director
Pharvaris Netherlands B.V.
60 sites in 24 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing deucrictibant in people with Hereditary Angioedema and Hereditary Angioedema Type I, run by Pharvaris Netherlands B.V. at 60 sites in 24 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 13 inclusion and 14 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 21 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 60 sites in United States, Italy, Japan, Brazil, Germany and 19 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Treatment-emergent Adverse Events (TEAEs), treatment-related TEAEs, treatment-emergent serious adverse events (TESAEs), treatment-related TESAEs, and TEAEs leading to deucrictibant discontinuation (time frame: From enrollment through study completion, up to 54 months (dependent on time of enrollment).). The study lists 7 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 21 Jul 2026.