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New pill could stop dangerous swelling attacks in hereditary angioedema

NCT ID NCT05396105

Symptom relief Sponsor: Pharvaris Netherlands B.V. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only This study
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 22, 2026 · Updated 2 times

Summary

This study tests a pill called deucrictibant for quickly treating swelling attacks in people with hereditary angioedema (HAE), including life-threatening throat swelling. About 150 adults who have had HAE attacks before will take the drug as needed and be monitored for safety and how fast symptoms improve. The goal is to see if this oral treatment can provide fast relief without the need for injections.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

About 150 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2022

Expected to finish

Jun 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: 1. 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. 2. 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. 3. 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. 4. In the opinion of the Investigator, the participant (and parent/caregiver for adolescent participants) is willing and able to comply with the protocol. 5. Adult participants with HAE type III (HAE-nC1INH) who are deucrictibant-treatment naïve, must meet all of the following: i. 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. ii. Must either have: 1. 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 2. 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). iii. 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. iv. Documented effective attack symptom relief with on-demand icatibant treatment. v. A history of at least 1 HAE attack in the last 3 months prior to Screening Key Exclusion Criteria: 1. Any female who is pregnant, plans to become pregnant, or is breast-feeding. 2. 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. 3. Use of lanadelumab for long-term HAE prophylactic therapy within 12 weeks prior to enrollment in Part A. 4. 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): i. For Part A: 1. 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. 2. 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. 3. 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. ii. For Part B: a. 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. 5. History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse 6. 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. 7. Discontinued from parent study after enrollment for any study drug-related safety reason or non-compliance including significant protocol deviation. 8. 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).

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Conditions

The condition(s) this trial relates to.

C1 inhibitor deficiency hereditary angioedema hereditary angioedema type 1 hereditary angioedema type 2 hereditary angioedema type 3 Hereditary Angioedema Types I and II hereditary angioedema with C1Inh deficiency hereditary angioedema with normal C1Inh

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

C1 esterase inhibitor [C1-inh] deficiency C1 esterase inhibitor deficiency C1 esterase inhibitor, deficiency of C1 inhibitor deficiency Hae with normal C1 esterase inhibitor Hereditary angioedema Hereditary angioedema (HAE) Hereditary angioedema - type 1 Hereditary angioedema - type 2 Hereditary angioedema - type 3 Hereditary angioedema attack Hereditary angioedema type I Hereditary angioedema type I and II Hereditary angioedema type II Hereditary angioedema type III Hereditary angioedema types I and II Hereditary angioedema with C1 esterase inhibitor deficiency

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    60 sites in 24 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Study site

    Birmingham, Alabama, 35209, United States

  • Study site

    Little Rock, Arkansas, 72205, United States

  • Study site

    San Diego, California, 92122, United States

  • Study site

    Santa Monica, California, 90404, United States

  • Study site

    Walnut Creek, California, 94598, United States

  • Study site

    Colorado Springs, Colorado, 80907, United States

  • Study site

    Chevy Chase, Maryland, 20815, United States

  • Study site

    Boston, Massachusetts, 02115, United States

  • Study site

    Detroit, Michigan, 48202, United States

  • Study site

    St Louis, Missouri, 63141, United States

  • Study site

    Hershey, Pennsylvania, 17033, United States

  • Study site

    Dallas, Texas, 75231, United States

  • Study site

    Buenos Aires, B1629AHJ, Argentina

  • Study site

    Salta, 4400, Argentina

  • Study site

    Campbelltown, New South Wales, 2560, Australia

  • Study site

    Graz, 8036, Austria

  • Study site

    Vienna, 1090, Austria

  • Study site

    Salvador, Estado de Bahia, 41950-640, Brazil

  • Study site

    Ribeirão Preto, 14048-900, Brazil

  • Study site

    Santo André, 09060-870, Brazil

  • Study site

    São Paulo, 05403-000, Brazil

  • Study site

    Sofia, 1431, Bulgaria

  • Study site

    Sofia, 1680, Bulgaria

  • Study site

    Edmonton, Alberta, T6G 1Z1, Canada

  • Study site

    Montreal, Quebec, Canada

  • Study site

    Brno, 602 00, Czechia

  • Study site

    Grenoble, 38043, France

  • Study site

    Paris, 75571, France

  • Study site

    Berlin, 12203, Germany

  • Study site

    Frankfurt am Main, 60590, Germany

  • Study site

    Frankfurt am Main, 60596, Germany

  • Study site

    Lübeck, 23538, Germany

  • Study site

    Hong Kong, Hong Kong

  • Study site

    Budapest, 1088, Hungary

  • Study site

    Ashkelon, 78278, Israel

  • Study site

    Catania, 95124, Italy

  • Study site

    Milan, 20097, Italy

  • Study site

    Milan, 20138, Italy

  • Study site

    Naples, 80131, Italy

  • Study site

    Padova, 35128, Italy

  • Study site

    Palermo, 90146, Italy

  • Study site

    Roma, 00133, Italy

  • Study site

    Hiroshima, 730-8518, Japan

  • Study site

    Kanagawa, 216-8511, Japan

  • Study site

    Osaka, 569-8686, Japan

  • Study site

    Tokyo, 113-8431, Japan

  • Study site

    Tokyo, 133-8431, Japan

  • Study site

    Amsterdam, 1005 AZ, Netherlands

  • Study site

    Krakow, 31-503, Poland

  • Study site

    San Juan, 918, Puerto Rico

  • Study site

    San Juan, 927, Puerto Rico

  • Study site

    Cape Town, 7700, South Africa

  • Study site

    Daegu, 41944, South Korea

  • Study site

    Seoul, 03080, South Korea

  • Study site

    Barcelona, 08907, Spain

  • Study site

    Madrid, 28007, Spain

  • Study site

    Lund, 22185, Sweden

  • Study site

    Ankara, 06230, Turkey (Türkiye)

  • Study site

    Istanbul, 34093, Turkey (Türkiye)

  • Study site

    London, E1 2ES, United Kingdom

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Other studies related to the condition(s) this trial covers.