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

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What is this study about?

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.

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 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 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 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 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 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 Study?

  • 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 Study?

  • 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

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Ente Ecclesiastico Ospedale Generale Regionale Miulli Acquaviva Delle Fonti Italy
Medizinische Hochschule Hannover Hanover Germany
Medical University Of Vienna Vienna Austria
Centre Hospitalier Universitaire De Lille Lille France
Bellvitge University Hospital L'hospitalet De Llobregat Spain
CHU Grenoble Alpes La Tronche France
Policlinico “Tor Vergata”, Università degli Studi di Roma TOR VERGATA Rome Italy
Hôpital Archet 2 Nice France

Other Sites

Site Name City Country Status
Hopital Saint Antoine Paris France
Klinikum der Technischen Universitaet Muenchen (TUM Klinikum) Munich Germany
ASST Fatebenefratelli Sacco Milan Italy
Univerzitna Nemocnica Martin Martin Slovakia
Azienda Ospedaliera Universitaria San Giovanni Di Dio E Ruggi d’Aragona Salerno Italy
Azienda Ospedaliera di Padova Padua Italy
Diagnostics And Consultation Center Convex Ltd. Sofia Bulgaria
Centru Clinic Mediquest S.R.L. Sangeorgiu De Mures Romania
Diagnostic-consultative center “Aleksandrovska” EOOD Sofia Bulgaria
Virgen del Rocío University Hospital Sevilla Spain
IRCCS Policlinico San Donato San Donato Milanese Italy
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Semmelweis University Budapest Hungary
Assistance Publique Hopitaux De Paris Paris France
Fondazione Salvatore Maugeri Clinica Del Lavoro E Della Riabilitazione Pavia Italy
HZRM Haemophilie-Zentrum Rhein Main GmbH Mörfelden-Walldorf Germany
Szpital Uniwersytecki w Krakowie Cracow Poland
Abqqzjh Siupgkklg Twiabszkaqsb Dq Mrbxoegf &zzzxhb Ojqquyau Cbuoijetpv Mbggfz Civitanova Marche Italy
Agobbpe Ottazmrvaey Otvmwqdr Rjezejc Vbtxb Swyhg Conuizwp Palermo Italy
Uwlieafjjlukwldapifng Miokltez Ahq Munster Germany
Gsvdcu Uwzgldozif Fsvhmytql Frankfurt Germany
Huxgnkzl Vots ddshmgik 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
13.01.2025
Bulgaria Bulgaria
Recruiting
13.01.2025
France France
Recruiting
13.01.2025
Germany Germany
Recruiting
13.01.2025
Hungary Hungary
Recruiting
13.01.2025
Ireland Ireland
Recruiting
13.01.2025
Italy Italy
Recruiting
13.01.2025
Poland Poland
Recruiting
13.01.2025
Romania Romania
Not recruiting
13.01.2025
Slovakia Slovakia
Recruiting
13.01.2025
Spain Spain
Recruiting
13.01.2025

Trial locations

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.

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 ID:
2024-516248-24-00
Protocol code:
PHA022121-C307
Trial Phase:
Therapeutic confirmatory (Phase III)

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