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Study of deucrictibant tablets for prevention of hereditary angioedema attacks in adults and adolescents

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical study focuses on testing a medication called deucrictibant for people with Hereditary Angioedema (HAE), a rare genetic condition that causes unexpected episodes of severe swelling in various parts of the body. These swelling attacks can affect the skin, digestive tract, and airways, potentially causing significant discomfort and health risks.

The purpose of this research is to evaluate how safe and effective deucrictibant extended-release tablets are when taken regularly to prevent HAE attacks. The medication will be given as a 40 mg oral tablet that patients take to help reduce the frequency and severity of swelling episodes.

This is a long-term study where participants will receive the study medication and be monitored for any side effects and changes in their condition. During the study, patients will continue to have access to their usual emergency medications for treating sudden HAE attacks. Participants will need to keep track of their symptoms and complete various questionnaires about their quality of life and how well the treatment is working for them.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial screening and enrollment

    If eligible, you will begin participation in a long-term study of deucrictibant, a medication for hereditary angioedema (HAE)

    Your medical history and HAE diagnosis will be reviewed

    Laboratory tests will confirm your HAE diagnosis if not previously confirmed

  2. Step 2

    Treatment initiation

    You will receive deucrictibant 40 mg extended-release tablets

    The medication will be taken by mouth

    You will need to maintain access to your regular HAE attack treatment medication throughout the study

  3. Step 3

    Ongoing monitoring

    Regular health assessments will include:

    Blood tests to monitor your health

    Vital signs measurements

    Heart monitoring through electrocardiogram (ECG)

    Recording of any side effects

    Blood samples to measure medication levels before taking your dose

  4. Step 4

    Attack monitoring and documentation

    You will need to record information about any HAE attacks in an electronic diary

    The doctor will review and confirm each reported attack

    Any use of attack treatment medication should be documented

  5. Step 5

    Quality of life assessments

    You will complete several questionnaires about:

    Your quality of life with angioedema

    Changes in your condition

    How well your symptoms are controlled

    Impact on your work and daily activities

    Your satisfaction with the treatment

  6. Step 6

    Study duration

    The study will run from January 2025 to November 2028

    Regular follow-up visits will continue throughout the study period

Who can join the trial?

11 criteria

  • Must be at least 12 years old and provide written consent. For participants aged 12-17, both the participant's assent and parent/guardian consent are required
  • May qualify if previously participated in HAE studies with deucrictibant treatment
  • Must have a confirmed diagnosis of Hereditary Angioedema (HAE) with:
    • Documented history of non-itchy swelling of skin or internal tissues
    • First symptoms appeared before age 30 OR family history of HAE
    • Normal C1q levels
    • C1 esterase inhibitor functioning below 50% of normal levels (confirmed by laboratory test)
    • For new participants: Must have experienced at least one HAE attack in the past 3 months before screening
    • Must have access to and ability to use standard treatments for acute HAE attacks
    • Must be willing and able to follow study requirements, including using an electronic diary
    • Female participants who can become pregnant must:
      • Agree to pregnancy testing as specified in the protocol
      • Use approved contraception during the study and for 30 days after the last dose

Who cannot join the trial?

15 criteria

  • Patients under 18 years of age
  • History of severe allergic reactions to similar medications
  • Currently pregnant or breastfeeding women
  • Active liver disease or abnormal liver function tests (tests showing how well your liver works)
  • Severe kidney problems (impaired kidney function)
  • Uncontrolled high blood pressure
  • Major surgery within the last 3 months
  • Participation in another clinical trial within the last 30 days
  • Current use of medications that might interact with the study drug
  • History of drug or alcohol abuse within the past year
  • Any severe psychiatric condition requiring ongoing treatment
  • Active cancer or cancer treatment within the last 5 years
  • Serious heart conditions or abnormal heart rhythm
  • History of blood clotting disorders
  • Any condition that, in the opinion of the study doctor, would make participation unsafe
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Investigated drugs

Deucrictibant is an extended-release tablet medication being studied for preventing angioedema attacks in people with hereditary angioedema (HAE). This medication works by helping to prevent sudden swelling episodes that can occur in various parts of the body in patients with HAE. The extended-release formulation is designed to provide long-lasting protection by releasing the medicine slowly throughout the day.

What is already known about the treatment

Deucrictibant – An oral medication administered in extended-release tablet form (40 mg) that is currently being investigated in clinical trials for the prophylactic treatment of Hereditary Angioedema (HAE). This novel drug belongs to the class of bradykinin B2 receptor antagonists, working by blocking the binding of bradykinin to prevent the blood vessel dilation and fluid leakage that causes angioedema attacks. The medication is designed for long-term preventive use in adolescents and adults with HAE, representing a potential advancement in treatment as an oral alternative to existing therapies. The extended-release formulation allows for sustained drug delivery throughout the day, which may help maintain consistent protection against angioedema attacks.

Investigated diseases

Hereditary Angioedema - A rare genetic condition that causes recurring episodes of severe swelling in various parts of the body. The swelling can affect the hands, feet, face, intestinal walls, and airways. Episodes can occur spontaneously or be triggered by stress, injury, or certain medications. The condition is caused by a deficiency or dysfunction of certain blood proteins that help regulate swelling. Between episodes, patients may have no visible symptoms. The swelling episodes typically develop slowly over 24 hours and can last for several days if untreated.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-516248-24-00Protocol codePHA022121-C307Estimated enrolment170 patientsSponsorPharvaris Netherlands B.V.

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