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Deucrictibant

Deucrictibant is an investigational drug being studied for the treatment of hereditary angioedema (HAE), a rare genetic disorder characterized by recurrent episodes of severe swelling. Clinical trials are evaluating deucrictibant as both a preventive medication and an on-demand treatment for acute HAE attacks. This article summarizes key information about ongoing deucrictibant clinical trials for patients with HAE.

Table of Contents

What is Deucrictibant?

Deucrictibant is a new medication being developed to treat hereditary angioedema (HAE). It is also known by other names such as PHA-022121, PHA121, and PHVS416[1]. Hereditary angioedema is a rare genetic condition that causes sudden, severe swelling in various parts of the body, including the skin, intestines, and airways. These swelling episodes, called “attacks,” can be painful, disfiguring, and even life-threatening if they affect the throat.

What Conditions Does Deucrictibant Treat?

Deucrictibant is specifically designed to treat hereditary angioedema types I and II[2]. These types are caused by a deficiency or dysfunction of a protein called C1 inhibitor. The medication is being studied for two main purposes:

  1. Acute treatment: To treat HAE attacks when they occur
  2. Prophylaxis: To prevent HAE attacks from happening

This dual approach aims to provide comprehensive management for patients with HAE, potentially improving their quality of life by both treating and preventing angioedema attacks.

How is Deucrictibant Administered?

One of the most promising aspects of deucrictibant is its method of administration. Unlike many current HAE treatments that require injections or intravenous infusions, deucrictibant is taken orally[3]. It comes in two forms:

  • Soft capsules: Used for acute treatment of HAE attacks
  • Extended-release tablets: Used for long-term prevention of HAE attacks

This oral administration could potentially make treatment more convenient and less invasive for patients with HAE.

Ongoing Clinical Trials

Deucrictibant is currently being studied in several clinical trials to evaluate its safety and effectiveness. These trials include:

  • CHAPTER-1 (NCT06679881): A long-term study evaluating the safety and efficacy of daily deucrictibant for preventing HAE attacks in adolescents and adults[3].
  • CHAPTER-2 (NCT06669754): A study comparing deucrictibant to placebo for preventing HAE attacks over 24 weeks[4].
  • CHAPTER-3 (NCT06343779): A study evaluating deucrictibant for on-demand treatment of HAE attacks[2].
  • RAPIDe-1 (NCT04618211): A dose-ranging study for acute treatment of HAE attacks[5].
  • RAPIDe-2 (NCT05396105): An extension study for long-term evaluation of deucrictibant in acute treatment of HAE attacks[1].
  • CHAPTER-1 (NCT05047185): A dose-ranging study for prevention of HAE attacks[6].

Potential Benefits

Based on the ongoing clinical trials, deucrictibant shows promise in several areas:

  • Rapid relief: Studies are measuring how quickly symptoms improve after taking deucrictibant, with some trials looking at relief as soon as 4 hours after treatment[2].
  • Attack prevention: Long-term studies are evaluating how well deucrictibant can reduce the frequency of HAE attacks[3].
  • Quality of life improvement: Researchers are using various questionnaires to assess how deucrictibant affects patients’ overall well-being and ability to carry out daily activities[4].
  • Convenience: The oral administration could make it easier for patients to take their medication regularly, potentially improving treatment adherence.

Safety and Side Effects

As with any new medication, safety is a primary concern in the deucrictibant clinical trials. Researchers are closely monitoring for any adverse effects, including:

  • Changes in heart rate, blood pressure, and body temperature
  • Alterations in blood and urine tests
  • Changes in electrocardiogram (ECG) readings
  • Any other unexpected side effects

It’s important to note that the full safety profile of deucrictibant will not be known until all clinical trials are completed and the data is thoroughly analyzed[1]. Patients interested in participating in clinical trials or learning more about deucrictibant should discuss this with their healthcare provider.

Aspect Details
Drug Name Deucrictibant (also known as PHA-022121, PHA121, PHVS416)
Condition Hereditary Angioedema (HAE) Types I and II
Administration Oral (extended-release tablets or soft capsules)
Trial Types Phase 2 and 3 studies for both prophylaxis and on-demand treatment
Key Outcomes Number and severity of HAE attacks, time to symptom relief, quality of life measures
Safety Assessments Adverse events, vital signs, laboratory tests, electrocardiograms
Patient Population Adolescents and adults (ages 12 and up) with HAE types I or II

FAQ

  • What is deucrictibant? Deucrictibant is an experimental oral medication being studied for the treatment of hereditary angioedema (HAE). It is being evaluated both as a preventive therapy to reduce HAE attacks and as an on-demand treatment for acute attacks.
  • How is deucrictibant taken? In clinical trials, deucrictibant is being studied in different forms – as extended-release tablets taken once daily for prevention of HAE attacks, and as soft capsules taken as needed to treat acute attacks.
  • What types of clinical trials are being conducted with deucrictibant? There are several types of trials, including long-term safety studies, dose-ranging studies to determine optimal dosing, and placebo-controlled trials to evaluate efficacy for both prevention and treatment of HAE attacks.
  • What are some of the outcomes being measured in deucrictibant trials? Key outcomes include the number and severity of HAE attacks, time to symptom relief after taking the medication, quality of life measures, and safety assessments like adverse events and laboratory tests.
  • Who can participate in deucrictibant clinical trials? Most trials are enrolling adolescents and adults (ages 12 and up) who have been diagnosed with HAE type I or II. Specific eligibility criteria may vary between studies.

Ongoing Clinical Trials on Deucrictibant

  • Study to evaluate the efficacy and safety of deucrictibant for the prevention and treatment of angioedema attacks in adults with acquired angioedema

    Recruiting

    3 1
    Investigated drugs:
    Austria Bulgaria France Germany Hungary Italy +3

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

    Recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    Austria Bulgaria France Germany Hungary Ireland +5

  • Study on the Safety of Long-Term Use of Deucrictibant for Treating Hereditary Angioedema Attacks in Patients with C1-Inhibitor Deficiency

    Recruiting

    4 1 1
    Investigated drugs:
    Austria Bulgaria Czechia France Germany Hungary +8

  • Study of Deucrictibant Extended-Release Tablets for Prevention of Hereditary Angioedema Attacks in Adults and Adolescents

    Not recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    Bulgaria France Germany Hungary Ireland Italy +4

  • Study on Deucrictibant and Icatibant for Treating Hereditary Angioedema Attacks in Adolescents and Adults

    Not recruiting

    3 1 1
    Investigated drugs:
    Austria Bulgaria Czechia France Germany Hungary +8

Glossary

  • Hereditary Angioedema (HAE): A rare genetic disorder characterized by recurrent episodes of severe swelling in various parts of the body, including the limbs, face, intestinal tract, and airway.
  • C1 Inhibitor Deficiency: The underlying cause of HAE types I and II, where there is a lack of functional C1 inhibitor protein in the body.
  • Prophylaxis: Preventive treatment taken regularly to reduce the frequency or severity of HAE attacks.
  • On-demand treatment: Medication taken at the onset of an HAE attack to reduce its severity and duration.
  • Extended-release tablet: A form of medication designed to release the active ingredient slowly over time, allowing for less frequent dosing.
  • Placebo: An inactive substance used in clinical trials to compare the effects of a drug against no treatment.
  • Visual Analogue Scale (VAS): A measurement tool used in clinical trials where patients rate their symptoms on a scale from 0 to 100.
  • Patient Global Impression of Change (PGI-C): A scale used in clinical trials where patients rate the change in their condition after treatment.
  • Angioedema Quality of Life Questionnaire (AE-QoL): A tool used to assess how HAE impacts a patient's quality of life.
  • Pharmacokinetics (PK): The study of how a drug is absorbed, distributed, metabolized, and eliminated by the body.

References