CHAPTER 4

  • Research type

    Research Study

  • Full title

    A Long-term, Open-label Study to Evaluate the Safety and Efficacy of Orally Administered Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults with Hereditary Angioedema

  • IRAS ID

    1010655

  • Contact name

    Kathryn Hutchinson

  • Contact email

    regulatoryaffairs@ams-europe.com

  • Sponsor organisation

    Pharvaris Netherlands BV

  • Eudract number

    2024-516248-24

  • Clinicaltrials.gov Identifier

    NCT066779881

  • Research summary

    This is a study to test the long-term safety and efficacy of a 40 mg dose of deucrictibant taken once a day in preventing attacks of hereditary angioedema (HAE) in patients aged ≥12 years. The study is being conducted at approximately 80 to 90 sites globally.
    The study will include patients who have HAE Type I or II and are not taking long-term treatments to prevent HAE attacks. Patients take 40 mg deucrictibant once a day for 130 weeks. Study treatment
    will be taken orally, once a day. Deucrictibant is a bradykinin B2 receptor antagonist.
    The primary objective of the study is to evaluate the safety and tolerability of long-term prophylactic treatment in HAE. The primary endpoints for assessing the objective will be treatment emergent
    safety events, changes from baseline in vital signs, safety blood and urine tests and 12-lead ECG assessments.
    Throughout the study patients will also complete a daily electronic diary of study drug intake and whether they experienced any HAE attacks.
    The total duration of the trial for any individual patient, would be 138 weeks (approximately 2.5 years) and would include 16 study visits, of which 4-5 may be conducted remotely.

  • REC name

    Wales REC 3

  • REC reference

    24/WA/0335

  • Date of REC Opinion

    17 Dec 2024

  • REC opinion

    Further Information Favourable Opinion