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

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What is this study about?

This clinical trial is focused on studying a condition known as Hereditary Angioedema, which is a rare genetic disorder that causes sudden swelling in various parts of the body, such as the face, hands, and feet. The study is testing a new treatment called Deucrictibant (PHA-022121), which is taken as a soft capsule by mouth. The purpose of the study is to see how effective Deucrictibant is in providing relief from the symptoms of Hereditary Angioedema attacks compared to a placebo.

Participants in the study will be randomly assigned to receive either the Deucrictibant capsule or a placebo during different periods of the study. This is known as a crossover study, meaning that each participant will have the chance to receive both the actual medication and the placebo at different times. The study will last for a period of up to 42 days, during which participants will take the medication as needed when they experience an attack. The researchers will monitor how quickly and effectively the medication provides relief from symptoms.

The study aims to gather information on how well Deucrictibant works in reducing the time it takes for symptoms to improve during an attack. This information will help determine if Deucrictibant can be a reliable on-demand treatment option for people with Hereditary Angioedema. Participants will be asked to record their symptoms and any changes they experience using an electronic diary, which will help the researchers assess the effectiveness of the treatment.

1 joining the study

Upon joining the study, the participant will provide written consent. If the participant is a minor, consent will be obtained from a parent or legal guardian, and the participant will provide written assent.

2 initial assessment

The participant will undergo an initial assessment to confirm eligibility. This includes verifying a diagnosis of hereditary angioedema (HAE) and a history of at least two HAE attacks in the last three months.

3 medication administration

The participant will receive deucrictibant in the form of a soft capsule, taken orally. This medication is used for on-demand treatment during HAE attacks.

A placebo, which looks like the deucrictibant capsule but does not contain the active substance, may also be administered as part of the study’s design.

4 monitoring and recording

The participant will record their symptoms and any changes using an electronic diary. This includes noting the time to onset of symptom relief and any other relevant observations.

5 evaluation of symptom relief

The primary goal is to evaluate the time to onset of symptom relief, defined as a noticeable improvement in symptoms within 12 hours after taking the medication.

Secondary evaluations include the proportion of attacks achieving symptom relief at various time points and the need for any additional medication.

6 completion of study participation

The study is expected to conclude by September 30, 2025. Participants will continue to be monitored and provide data until the end of their involvement in the study.

Who Can Join the Study?

  • The participant, or their parent/caregiver if they are an adolescent, must be willing and able to follow the study rules according to the Investigator’s opinion.
  • Female participants who can have children must agree to pregnancy testing and either not engage in heterosexual intercourse or use a reliable birth control method during the study and for 30 days after the last dose of the study drug. Male participants do not have these requirements.
  • Participants must be male or female, aged between 12 and 75 years at the time they agree to join the study.
  • Participants must have a diagnosis of Hereditary Angioedema (HAE) type 1 or 2, which includes:
    • A history of swelling under the skin or in mucous membranes without itching, and no hives.
    • At least one of the following: symptoms started before age 30, a family history of HAE, or a specific blood test result.
    • Blood test results confirming HAE type 1 or 2, with specific conditions for those on certain long-term treatments.
  • Participants must have had at least 2 HAE attacks in the 3 months before joining the study.
  • Participants must have experience using standard treatments to manage HAE attacks when they occur.
  • If participants are on long-term preventive treatment for HAE with certain medications, they must have been on a stable dose for at least 6 months before joining the study and plan to continue the same dose during the study.
  • Participants must be able to use an electronic device to record their HAE symptoms and other information without help, as shown during the screening phase.
  • Adolescent participants aged 12 to less than 18 years must weigh at least 40 kg (about 88 pounds).
  • Participants must provide written consent to join the study. If the participant is under 18, their parent or legal guardian must give consent, and the participant must also agree in writing, following local laws.

Who Cannot Join the Study?

  • Patients who are currently participating in another clinical trial.
  • Patients who have a history of severe allergic reactions to any of the study medications.
  • Patients who are pregnant or breastfeeding.
  • Patients with any other significant medical condition that might interfere with the study.
  • Patients who have used certain medications that could affect the study results.
  • Patients who have a history of drug or alcohol abuse.
  • Patients who are unable to comply with the study procedures.
  • Patients who have had a recent surgery or are planning to have surgery during the study period.
  • Patients with a known infection that could affect the study.
  • Patients who have a mental health condition that might interfere with their ability to participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Bellvitge University Hospital L'hospitalet De Llobregat Spain
Medical University Of Vienna Vienna Austria
Centre Hospitalier Universitaire De Lille Lille France
Medical University Of Graz Graz Austria
Policlinico “Tor Vergata”, Università degli Studi di Roma TOR VERGATA Rome Italy

Other Sites

Site Name City Country Status
Hopital Saint Antoine Paris France
Azienda Ospedaliero-Universitaria Policlinico G. Rodolico-San Marco Di Catania Catania Italy
Univerzitna Nemocnica Martin Martin Slovakia
Centru Clinic Mediquest S.R.L. Sangeorgiu De Mures Romania
Universitaetsklinikum Schleswig-Holstein AöR Kiel Germany
Azienda Ospedaliera di Padova Padua Italy
Diagnostic-consultative center “Aleksandrovska” EOOD Sofia Bulgaria
Region Skane Skanes Universitetssjukhus Lund Sweden
IRCCS Policlinico San Donato San Donato Milanese Italy
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Semmelweis University Budapest Hungary
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie Cracow Poland
Fakultni Nemocnice U Sv Anny V Brne Brno-Stred Czechia
Fondazione Salvatore Maugeri Clinica Del Lavoro E Della Riabilitazione Pavia Italy
Convex Ltd. Sofia Bulgaria
HZRM Haemophilie-Zentrum Rhein Main GmbH Mörfelden-Walldorf Germany
Amnpphz Okoxmwvcavh Odqfxubj Rhcnmwv Vgqeu Sxvok Cxxhtqty Palermo Italy
Augoelkby Uar Amsterdam The Netherlands
Goueui Usbrhjzwsn Ffukzjfrg Frankfurt Germany
Jbvnoczh Kwjfxu Uzsedtivuf Linz Austria
Hcnxlonh Vjmi dparmnfy Barcelona Spain

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Austria Austria
Not recruiting
24.06.2024
Bulgaria Bulgaria
Not recruiting
24.06.2024
Czechia Czechia
Not recruiting
24.06.2024
France France
Not recruiting
24.06.2024
Germany Germany
Not recruiting
24.06.2024
Hungary Hungary
Not recruiting
24.06.2024
Ireland Ireland
Not recruiting
24.06.2024
Italy Italy
Not recruiting
24.06.2024
Poland Poland
Not recruiting
24.06.2024
Romania Romania
Not recruiting
24.06.2024
Slovakia Slovakia
Not recruiting
24.06.2024
Spain Spain
Not recruiting
24.06.2024
Sweden Sweden
Not recruiting
24.06.2024
The Netherlands The Netherlands
Not recruiting
24.06.2024

Trial locations

Investigated drugs:

Deucrictibant is an oral medication being studied for its ability to provide relief during attacks of hereditary angioedema (HAE). This condition causes sudden swelling in various parts of the body, and the medication is designed to be taken as needed when an attack occurs. The goal of the trial is to determine how effectively deucrictibant can reduce symptoms and provide relief compared to not taking any active medication.

Hereditary Angioedema – Hereditary Angioedema is a genetic disorder characterized by recurrent episodes of severe swelling, often affecting the face, extremities, gastrointestinal tract, and airway. These episodes, known as attacks, can occur spontaneously or be triggered by stress, trauma, or certain medications. The swelling is due to a deficiency or dysfunction of a protein called C1 inhibitor, which leads to an overproduction of bradykinin, a peptide that increases vascular permeability. Attacks can vary in frequency and severity, with some individuals experiencing them more frequently than others. The condition is typically inherited in an autosomal dominant pattern, meaning a single copy of the altered gene in each cell is sufficient to cause the disorder. Swelling episodes can be painful and debilitating, but they do not cause permanent damage to tissues.

Trial ID:
2023-507268-37-00
Protocol code:
PHA022121-C306
Trial Phase:
Therapeutic confirmatory (Phase III)

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