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Study design

Participation in the RAPIDe-3 Study will vary in duration based on how long it takes for a participant to experience and treat 2 qualifying HAE attacks with deucrictibant and placebo. This includes a screening period, a Day 1 visit/non-attack visit, an attack treatment phase with a follow-up visit for the first qualifying HAE attack, and an end-of-study visit after a second qualifying HAE attack.

Deucrictibant and the placebo are both given in the form of softgel capsules, which will be taken orally, according to a participant’s assigned order. The participants will be randomly assigned to receive them in 1 of 2 orders: deucrictibant first and then the placebo or the placebo first and then deucrictibant. If the study drug does not provide sufficient symptom relief during an HAE attack, the participant may contact their study doctor again to discuss how they feel and determine if they should take a second dose of the study drug. However, if needed, the participant may use their own prescribed HAE medication or study-provided icatibant.

Eligibility criteria

Eligible participants must:

• Be male or female between 12 and 75 years at screening
• Have a diagnosis of HAE based on the following:
  • For participants with HAE type 1/2:
    • Documented clinical history consistent with HAE (subcutaneous or mucosal, nonpruritic swelling without accompanying urticaria)
    • At least one of the following:
      • Age ≤ 30 years at reported onset of first angioedema symptoms
      • Family history consistent with HAE type 1/2
      • C1q performed by the central laboratory is above the lower limit of the normal range
    • Diagnostic testing results to confirm HAE type 1/2:
      • Participants on long-term prophylactic HAE therapies with C1-INH or danazol at the time of study entry must provide documentation of a historical laboratory test showing C1-INH functional level < 50%.
        Note: If a confirmatory C1-INH test is required, the sample should be collected at least 5 half-lives after the last dose of C1-INH or danazol. This procedure will not be considered an interruption of stable LTP treatment.
      • For all other participants with HAE type 1/2, C1-INH functional level of < 50% must be shown by chromogenic assay performed by the central laboratory as part of the screening procedures.
  • For participants with HAE type 3:
    • Recurrent angioedema attacks with diagnostic testing results obtained during screening to confirm C1-INH function ≥ 50% of normal and C4 level not below lower level of normal range performed by the central laboratory
      Note: The C1-INH sample should be collected at least 5 half-lives after the last dose of C1-INH therapy, if applicable.
    • Must meet one of the following:
      • Documented genetic mutation associated with HAE type 3 as listed in the HAEA and WAO/EAACI Guidelines (Appendix 1)
        OR
      • If no documented mutation:
        • Clinical diagnosis with family history of HAE type 3 and an elevated BK peptide level confirmed by a commercially available assay
          AND
        • Attacks not responding to treatments with high-dose antihistamine (cetirizine 40 mg/day or equivalent high-dose second-generation antihistamine medication) and no clinical attack symptoms relief if treated with corticosteroid, montelukast, or omalizumab
    • Documented effective attack symptom relief with on-demand icatibant treatment
• Have a history of at least 2 HAE attacks 3 months prior to screening
• Either:
  • Is on a stable dose and regimen of long-term prophylactic therapy with one of the following medications indicated for HAE: plasma-derived C1-INH, danazol at ≤ 200 mg/day, anti-fibrinolytics, berotralstat, or lanadelumab, for at least 6 months before the screening visit and intends to remain on the same dose for the duration of the study
    OR
  • Is receiving only on-demand treatment and has not used long-term prophylactic treatment within the following durations before the screening visit, as specified below:
    • Plasma-derived C1-INH, berotralstat, anti-fibrinolytics, or attenuated androgens for at least 2 weeks
    • Lanadelumab for at least 10 weeks
• Have experience using standard-of-care treatment to effectively manage on-demand treatment for HAE attacks
• Capable of recording, without assistance, electronic HAE diary and ePRO data using an electronic device
• Not have any diagnosis of angioedema other than HAE
• Female participants of childbearing potential must agree to the protocol-specified pregnancy testing and contraceptive requirements