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

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What is this study about?

This study focuses on people with Hereditary Angioedema, a rare genetic condition that causes unexpected episodes of severe swelling in various parts of the body. The research evaluates a new oral medication called deucrictibant, which is being tested as a preventive treatment to reduce the frequency of swelling attacks. The study will compare deucrictibant extended-release tablets to a placebo in adolescents and adults.

The study will test if taking a 40 mg tablet of deucrictibant daily can prevent or reduce the occurrence of swelling attacks compared to taking a placebo. For comparison purposes, some participants will also have access to icatibant, an already approved medication that is injected under the skin to treat acute attacks when they occur.

The treatment period will last 24 weeks, during which participants will take either deucrictibant or placebo tablets. Throughout the study, participants will need to keep track of any swelling attacks they experience and may use emergency treatment if needed. The study will monitor how well the medication works and check for any potential side effects through regular health assessments.

1 Initial phase

After qualifying for the study, you will enter an up to 8-week Run-in Period. During this time, your HAE attacks (sudden swelling episodes) will be monitored and confirmed by the investigator.

You will need to record your symptoms in an electronic diary (eDiary) provided to you.

2 Treatment period start

The treatment period will last for 24 weeks (168 days).

You will be randomly assigned to receive either deucrictibant 40 mg extended-release tablets or placebo tablets (tablets without active medication).

The tablets should be taken by mouth (oral route).

3 During treatment

Throughout the 24-week period, you will need to record any swelling attacks in your electronic diary.

You will have access to your regular on-demand medication (including icatibant given by injection under the skin) to treat any attacks that occur.

You will complete several questionnaires about your quality of life and satisfaction with treatment.

Regular health checks will include blood tests, vital signs measurements, and heart recordings (ECG).

4 Monitoring

The study team will track the number and severity of your swelling attacks.

Any side effects or health changes will be recorded and monitored.

Blood samples will be taken to measure the levels of the study medication in your body.

5 Study completion

The study will end after the 24-week treatment period.

Final health assessments will be performed to evaluate the overall effects of the treatment.

Who Can Join the Study?

  • Must be at least 12 years old and provide written informed consent. For participants under 18, parental consent is required
  • Adolescents (12-18 years) must weigh at least 40 kg
  • Must have a confirmed diagnosis of Hereditary Angioedema (HAE) type 1/2 or type 3 with appropriate laboratory test results
  • Must have experienced at least 3 HAE attacks within 3 months before screening
  • Must have reliable access to standard treatments for managing acute HAE attacks
  • Must be willing and able to use electronic diary and patient reporting tools
  • Must be able to follow all study requirements
  • For women who can become pregnant:
    • Must agree to pregnancy testing
    • Must either remain abstinent or use highly effective contraception during the study and for 30 days after the last dose
  • For participants with HAE type 1/2:
    • Must have documented history of swelling without itching or hives
    • Must have either: symptoms starting before age 30, family history of HAE, or normal C1q levels
  • For participants with HAE type 3:
    • Must have confirmed normal C1-inhibitor function
    • Must have either genetic mutation associated with HAE type 3 or family history with confirmed elevated bradykinin levels
    • Must show response to icatibant treatment

Who Cannot Join the Study?

  • Age below 12 years old
  • Women who are pregnant or breastfeeding
  • History of severe allergic reactions to similar medications
  • Current use of other medications for hereditary angioedema prevention
  • Participation in other clinical trials within the past 30 days
  • Severe kidney or liver disease
  • Uncontrolled high blood pressure
  • Major surgery planned during the study period
  • History of drug or alcohol abuse within the past year
  • Mental conditions that could interfere with following study procedures
  • Any condition that, in the investigator’s opinion, makes participation unsafe
  • Inability or unwillingness to follow study procedures
  • History of blood clotting disorders
  • Active cancer or cancer treatment
  • Serious heart conditions or recent heart problems

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
Bellvitge University Hospital L'hospitalet De Llobregat Spain
Centre Hospitalier Universitaire De Lille Lille France
CHU Grenoble Alpes La Tronche France
Policlinico “Tor Vergata”, Università degli Studi di Roma TOR VERGATA Rome Italy

Other Sites

Site Name City Country Status
Hopital Saint Antoine Paris France
Diagnostics And Consultation Center Convex Ltd. Sofia Bulgaria
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
Centru Clinic Mediquest S.R.L. Sangeorgiu De Mures Romania
Azienda Ospedaliera di Padova Padua Italy
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
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie Cracow Poland
Assistance Publique Hopitaux De Paris Paris France
Centre Hospitalier Universitaire De Nice Nice France
Fondazione Salvatore Maugeri Clinica Del Lavoro E Della Riabilitazione Pavia Italy
HZRM Haemophilie-Zentrum Rhein Main GmbH Mörfelden-Walldorf Germany
Ufkuzitnlwzjeogtzxiia Mrvmtgfh Aju Munster Germany
Gtfqvy Uvnvzcgcuj Fskitxoop Frankfurt Germany
Hbtjfcht Vzea dupofayv Barcelona Spain

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

Trial status

Country Status Recruitment Start
Bulgaria Bulgaria
Not recruiting
13.01.2025
France France
Not recruiting
13.01.2025
Germany Germany
Not recruiting
13.01.2025
Hungary Hungary
Not recruiting
13.01.2025
Ireland Ireland
Not recruiting
13.01.2025
Italy Italy
Not recruiting
13.01.2025
Poland Poland
Not recruiting
13.01.2025
Romania Romania
Not recruiting
13.01.2025
Slovakia Slovakia
Not recruiting
13.01.2025
Spain Spain
Not recruiting
13.01.2025

Trial locations

Investigated drugs:

Deucrictibant is a medication being studied as a preventive treatment for hereditary angioedema (HAE), a rare condition that causes sudden swelling attacks in different parts of the body. This medication comes in an extended-release tablet form that is taken by mouth. It works by helping to prevent the swelling attacks that are typical in people with hereditary angioedema. The medication is designed to be taken regularly as a preventive measure, rather than treating attacks after they occur.

Investigated diseases:

Hereditary Angioedema – A genetic condition that causes recurring episodes of severe swelling in various parts of the body. The swelling typically affects the hands, feet, face, airway, and intestinal walls. Episodes can occur spontaneously or be triggered by stress, minor injuries, or other factors. 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.

Trial ID:
2024-516247-62-00
Protocol code:
PHA022121-C305
NCT ID:
NCT06669754
Trial Phase:
Therapeutic confirmatory (Phase III)

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