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(S)-N-(1-Deutero-1-(3-Chloro-5-Fluoro-2-((2-Methyl-4-(1-Methyl-1H-1,2,4-Triazol-5-Yl)Quinolin-8-Yloxy)Methyl)Phenyl)Ethyl)-2-(Difluoromethoxy)Acetamide

This article explores recent clinical trials investigating the use of deucrictibant, also known as PHA-022121, for the treatment of hereditary angioedema (HAE). Deucrictibant is a novel oral medication being developed as both a preventive and on-demand treatment for HAE attacks. These trials aim to evaluate the safety, efficacy, and long-term use of deucrictibant in patients with HAE due to C1-inhibitor deficiency. The studies focus on its potential to reduce the frequency and severity of angioedema attacks, offering a new treatment option for individuals living with this rare genetic disorder.

Table of Contents

What is Deucrictibant?

Deucrictibant, also known as PHA-022121, is a new oral medication being developed to treat hereditary angioedema (HAE)[1]. It is currently undergoing clinical trials to evaluate its safety and effectiveness in preventing and treating HAE attacks. Deucrictibant belongs to a class of drugs called bradykinin B2 receptor antagonists[2].

What is Hereditary Angioedema?

Hereditary angioedema is a rare genetic disorder that causes sudden, severe swelling in various parts of the body, including the hands, feet, face, and airways[1]. These swelling episodes, called “attacks,” can be painful, disfiguring, and even life-threatening if they affect the throat or airways. HAE is caused by a deficiency or dysfunction of a protein called C1 inhibitor, which helps regulate swelling in the body[1].

How Does Deucrictibant Work?

Deucrictibant works by blocking the bradykinin B2 receptor[2]. Bradykinin is a substance that causes blood vessels to expand and become leaky, leading to swelling. By blocking the receptor that bradykinin acts on, deucrictibant aims to prevent or reduce the swelling associated with HAE attacks.

Ongoing Clinical Trials

Deucrictibant is currently being studied in several clinical trials:

  1. A Phase II trial (Study PHA022121-C301) is evaluating the safety and efficacy of deucrictibant for preventing HAE attacks[1]. This study is testing different doses of the medication taken orally.
  2. A Phase II/III extension study (Study PHA022121-C303) is investigating the long-term safety and effectiveness of deucrictibant for treating acute HAE attacks[2].

These trials aim to determine the best dosage, assess how well the medication works, and monitor any potential side effects over time.

Potential Benefits

If proven effective, deucrictibant could offer several advantages for people with HAE:

  • Oral administration: Unlike some current HAE treatments that require injections or infusions, deucrictibant is taken by mouth, which may be more convenient for patients[1].
  • Prevention of attacks: The medication is being studied for its ability to reduce the frequency of HAE attacks, potentially improving quality of life for patients[1].
  • Treatment of acute attacks: Deucrictibant is also being evaluated for its effectiveness in treating HAE attacks when they occur[2].

Safety and Side Effects

As deucrictibant is still in clinical trials, its full safety profile is not yet known. The ongoing studies are closely monitoring participants for any adverse effects[1][2]. Common side effects and any serious adverse events will be carefully documented and analyzed to ensure the medication’s safety before it can be approved for general use.

Who Can Use Deucrictibant?

Currently, deucrictibant is only available to participants in clinical trials. The studies have specific eligibility criteria, including:

  • Diagnosed with HAE Type I or II[1]
  • Age 18 to 75 years (for some studies)[1]
  • History of HAE attacks[1]
  • No recent use of certain other HAE treatments[1][2]

It’s important to note that these criteria may vary between different studies and may change as research progresses.

Conclusion

Deucrictibant (PHA-022121) represents a promising new approach to treating hereditary angioedema. As an oral medication, it has the potential to offer a more convenient option for both preventing and treating HAE attacks. However, it’s crucial to remember that the drug is still in the testing phase, and more research is needed to fully understand its effectiveness and safety profile. Patients with HAE should continue to work closely with their healthcare providers to manage their condition and stay informed about emerging treatment options.

Aspect Details
Drug Name Deucrictibant (PHA-022121)
Condition Hereditary Angioedema (HAE) due to C1-Inhibitor Deficiency (Type I or II)
Administration Oral (soft capsules)
Trial Phases Phase II and Phase II/III
Study Designs Double-blind, placebo-controlled, randomized, dose-ranging (Phase II)
Open-label extension study (Phase II/III)
Primary Objectives Evaluate safety and efficacy for prophylaxis and acute treatment of HAE attacks
Key Endpoints Number of HAE attacks, time to symptom relief, adverse events, quality of life measures
Patient Population Adults and adolescents with confirmed HAE Type I or II
Dosing Various doses tested, up to 60 mg daily
Treatment Duration Up to 48 months (long-term extension study)

FAQ

  • What is deucrictibant (PHA-022121)? Deucrictibant, also known as PHA-022121, is a new oral medication being studied for the treatment of hereditary angioedema (HAE). It is a bradykinin B2 receptor antagonist designed to prevent and treat acute angioedema attacks in patients with HAE.
  • What types of HAE is deucrictibant being studied for? Deucrictibant is being studied for hereditary angioedema due to C1-inhibitor deficiency, specifically Type I and Type II HAE. These are the most common forms of HAE caused by genetic mutations affecting the C1-inhibitor protein.
  • How is deucrictibant administered? Deucrictibant is administered orally in the form of soft capsules. This oral route of administration is a potential advantage over some existing HAE treatments that require injections or infusions.
  • What are the main objectives of the clinical trials for deucrictibant? The main objectives of the clinical trials are to evaluate the safety and efficacy of deucrictibant for both long-term prevention (prophylaxis) and acute treatment of HAE attacks. The studies aim to assess its ability to reduce the frequency and severity of attacks, as well as its long-term safety profile.
  • What are some of the key endpoints being measured in these trials? Key endpoints include the number of HAE attacks during treatment periods, time to onset of symptom relief, safety assessments such as adverse events, and quality of life measures. The trials also look at the need for additional doses or rescue medications after initial treatment with deucrictibant.

Ongoing Clinical Trials on (S)-N-(1-Deutero-1-(3-Chloro-5-Fluoro-2-((2-Methyl-4-(1-Methyl-1H-1,2,4-Triazol-5-Yl)Quinolin-8-Yloxy)Methyl)Phenyl)Ethyl)-2-(Difluoromethoxy)Acetamide

  • 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 on Oral PHA-022121 for Preventing Angioedema Attacks in Patients with Hereditary Angioedema

    Not recruiting

    2 1
    Investigated diseases:
    Investigated drugs:
    Austria Bulgaria Germany Ireland Italy Poland

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: A genetic condition where the body doesn't produce enough of the C1-inhibitor protein, leading to uncontrolled production of bradykinin and resulting in angioedema attacks.
  • Bradykinin B2 Receptor Antagonist: A type of medication that blocks the action of bradykinin, a peptide that causes blood vessels to expand and is responsible for the swelling in HAE attacks.
  • Prophylaxis: Preventive treatment given to stop or reduce the frequency and severity of HAE attacks before they occur.
  • On-demand Treatment: Medication given at the onset of an HAE attack to reduce its severity and duration.
  • Efficacy: The ability of a treatment to produce the desired beneficial effect.
  • Adverse Event: Any unfavorable and unintended sign, symptom, or disease temporarily associated with the use of a medical treatment or procedure.
  • Quality of Life (QoL): A measure of an individual's well-being and ability to carry out daily activities, often used to assess the impact of a medical condition or treatment.
  • Pharmacokinetics (PK): The study of how a drug moves through the body, including its absorption, distribution, metabolism, and excretion.
  • Pharmacodynamics (PD): The study of the biochemical and physiological effects of drugs on the body, including their mechanisms of action and relationship between drug concentration and effect.

References

  1. http://clinicaltrials.eu/trial/study-on-oral-pha-022121-for-preventing-angioedema-attacks-in-patients-with-hereditary-angioedema/
  2. http://clinicaltrials.eu/trial/study-on-the-safety-of-long-term-use-of-deucrictibant-for-treating-hereditary-angioedema-attacks-in-patients-with-c1-inhibitor-deficiency/