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

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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 attacks in various parts of the body, including the face, hands, and throat. The study is specifically looking at patients with Hereditary Angioedema due to a deficiency in a protein called C1-Inhibitor, which can be of Type I or Type II. The treatment being tested is an oral medication called Deucrictibant (PHA-022121), which is designed to be taken during an acute attack to help manage the symptoms.

The purpose of this study is to evaluate the safety of long-term use of Deucrictibant for treating these acute attacks, including those affecting the throat, but without causing breathing difficulties. Participants in the study will take the medication as needed during an attack, and their health will be monitored over time to ensure the treatment is safe. The study will also involve a comparison with a placebo, which is a substance with no active medication, to better understand the effects of Deucrictibant.

Throughout the study, participants will be asked to report any side effects they experience, and regular health checks will be conducted, including monitoring vital signs and performing laboratory tests. The study aims to provide valuable information on how well Deucrictibant works in relieving symptoms and improving the quality of life for those with Hereditary Angioedema. This research is important for developing effective treatments for managing this challenging condition.

1 joining the study

Upon joining the study, the participant will begin the trial focused on evaluating the safety of long-term treatment for hereditary angioedema attacks.

The study involves the use of the medication deucrictibant, which is administered orally in the form of a soft capsule.

2 medication administration

The participant will take deucrictibant as needed for acute angioedema attacks. The medication is taken orally.

The frequency and dosage will be determined based on the occurrence of attacks and the participant’s response to the treatment.

3 monitoring and assessments

Throughout the study, the participant’s health will be monitored through various assessments.

These assessments include checking vital signs, conducting clinical laboratory tests, and performing ECGs (a test that records the electrical activity of the heart).

4 evaluation of symptom relief

The study will evaluate the time it takes for the participant to experience symptom relief after taking the medication.

This includes measuring the time to onset of symptom relief and the time to substantial symptom relief, using tools like the Patient Global Impression of Change (PGI-C) and the Visual Analog Scale (VAS).

5 completion of the study

The study is expected to continue until June 2027, with ongoing assessments and monitoring.

The participant will continue to take the medication as needed and attend scheduled assessments until the study concludes.

Who Can Join the Study?

  • The patient must provide written informed consent. If the patient is under 18 years old, consent must be obtained from a parent or legal guardian, and the patient must also agree in writing.
  • If the patient participated in Study C201, they must have received at least one dose of the study drug. If they participated in Study C306, they must have been part of the study and completed it, with at least two attacks treated, or after the study was closed by the Sponsor.
  • Female patients who can become pregnant must agree to pregnancy testing as specified in the study and must either not engage in heterosexual intercourse or use an acceptable method of birth control from the start of the study until 30 days after the last dose of the study drug.
  • The patient, and if applicable, their parent or caregiver, must be willing and able to follow the study’s requirements, as determined by the study doctor.

Who Cannot Join the Study?

  • Patients who have a different type of hereditary angioedema than Type 1 or 2 C1-Inhibitor deficiency cannot participate.
  • Patients who have breathing difficulties during laryngeal attacks are not eligible.
  • Patients who are not within the specified age range for the study cannot join. The age range includes children, adolescents, and adults.
  • Patients who are not part of the specified clinical trial group are excluded.
  • Patients who are considered part of a vulnerable population, which means they might need special protection or care, are not eligible.

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
Azienda Ospedaliera Universitaria Federico II Di Napoli Naples Italy
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
Azienda Ospedaliero-Universitaria Policlinico G. Rodolico-San Marco Di Catania Catania Italy
Hospital General Universitario Gregorio Maranon Madrid Spain
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
Hsuowxg Ssrhh Ankuwgh Paris France
Alliwpt Ovvmvxjarcr Osknifjr Rvvzxil Vjjri Spqrb Cfmddxap Palermo Italy
Auqqpduhl Uzm Amsterdam The Netherlands
Gnvyxj Umwzzalkcf Fuupdipvs Frankfurt Germany
Hiwuoipv Vrwh dtyikzfj 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
Recruiting
05.12.2022
Bulgaria Bulgaria
Recruiting
05.12.2022
Czechia Czechia
Recruiting
05.12.2022
France France
Recruiting
05.12.2022
Germany Germany
Recruiting
05.12.2022
Hungary Hungary
Recruiting
05.12.2022
Ireland Ireland
Not recruiting
05.12.2022
Italy Italy
Recruiting
05.12.2022
Poland Poland
Recruiting
05.12.2022
Romania Romania
Not recruiting
05.12.2022
Slovakia Slovakia
Not recruiting
05.12.2022
Spain Spain
Recruiting
05.12.2022
Sweden Sweden
Recruiting
05.12.2022
The Netherlands The Netherlands
Recruiting
05.12.2022

Trial locations

PHA-022121 is an oral medication being studied for its effectiveness in treating sudden swelling attacks in patients with hereditary angioedema, a condition caused by a deficiency in a specific protein. This medication is intended to be used as needed when an attack occurs, helping to manage symptoms quickly and effectively.

Deucrictibant is another medication involved in the study, focusing on its safety for long-term use in treating acute hereditary angioedema attacks. It is designed to be used on-demand, meaning patients take it when they experience an attack, including those affecting the throat, to help alleviate symptoms without causing breathing difficulties.

Hereditary Angioedema – Hereditary angioedema is a rare genetic disorder characterized by recurrent episodes of severe swelling, known as angioedema. This condition is caused by a deficiency or dysfunction of the C1 inhibitor protein, which normally helps regulate inflammation. The swelling can affect various parts of the body, including the extremities, face, intestinal tract, and airway. Attacks can occur spontaneously or be triggered by stress, trauma, or other factors. The swelling episodes can be painful and may last for several days. While the condition is chronic, the frequency and severity of attacks can vary widely among individuals.

Trial ID:
2023-505766-28-00
Protocol code:
PHA022121-C303
NCT ID:
NCT05396105
Trial Phase:
Therapeutic use (Phase IV)

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