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Study of Oral Deucrictibant XR Tablet for Prophylaxis and Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adults With Acquired Angioedema Due to C1 Inhibitor Deficiency

ConditionAcquired Angioedema Due to C1-Inhibitor Deficiency (AAE-C1-INH)
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18+
SponsorPharvaris Netherlands B.V.

About this trial

This is a Phase 3, multicenter, 3-part study, with 2 randomized, double-blind, placebo-controlled parts and an open-label extension part, to evaluate the efficacy and safety of orally administered deucrictibant XR tablet for prophylaxis, and deucrictibant soft capsule for on-demand treatment of angioedema attacks in adult participants aged ≥ 18 years with AAE-C1INH.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Provision of written informed consent

Male or female (sex at birth) aged ≥18 years

Diagnosis of AAE-C1INH

History of AAE-C1INH attacks prior to the Screening Visit.

Disqualifiers

Participation in a clinical study with any other investigational drug within the last 30 days or within 5 half-lives of the investigational drug at the Screening Visit (whichever is longer).

Receiving long-term prophylaxis (LTP) treatment for AAE-C1INH and satisfied with this treatment. Participants who are not satisfied (eg, tolerability issues, lack of efficacy) and have previously stopped LTP treatment for AAE-C1INH, for reasons other than participation in this study, can sign the ICF and begin the Screening Period only if their last dose of the treatment was received prior to the timepoint before the Screening Visit

Any females who are pregnant, plan to become pregnant, or are currently breast-feeding

Abnormal hepatic function

Trial design

Design model

Parallel

Treatments tested in this trial

  • Deucrictibant

    Drug

    Part 1: Deucrictibant 40 mg extended-release tablet for once daily oral use

  • Placebo

    Drug

    Part 1: Placebo Comparator tablet for once daily oral use

  • Deucrictibant

    Drug

    Part 2: Deucrictibant 20 mg soft capsule oral use

  • Placebo

    Drug

    Part 2: Placebo Comparator soft capsule oral use

  • Deucrictibant

    Drug

    Part 3: Deucrictibant 20 mg soft capsule oral use

Treatment groups

48 Participants
are divided into 5 treatment groups
Group A: Part 1 - Arm 1 - ActiveExperimental treatment 1 intervention
Group B: Part 1 - Arm 2 - PlaceboPlacebo comparator 1 intervention
Group C: Part 2 - Arm 1Experimental treatment 2 interventions
Group D: Part 2 - Arm 2Experimental treatment 2 interventions
Group E: Part 3 - Open-labelExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Part 1: Time-normalized number of Investigator-confirmed AAE-C1INH attacks during Treatment Phase

Time frame
12 weeks
2

Part 2: Time to symptom relief, Patient Global Impression of Change (PGI-C) rating of at least "better"

Time frame
12 hours post-treatment
3

Part 3: Incidence of treatment-emergent adverse events (TEAEs), treatment-emergent adverse events of special interest (AESIs), and serious adverse events (SAEs)

Time frame
Through study termination, an average of 36 weeks
4

Part 3: Number of participants with clinically significant changes from baseline in Hematology parameters

Time frame
Through study termination, an average of 36 weeks

Secondary outcomes

1

Part 1: Proportion of participants who are AAE-C1INH attack-free during Treatment Phase

Time frame
12 weeks
2

Part 1: Time-normalized number of Investigator-confirmed AAE-C1INH attacks treated with on-demand medication during Treatment Phase

Time frame
12 weeks
3

Part 1: Time-normalized number of Investigator-confirmed moderate or severe AAE-C1INH attacks during Treatment Phase

Time frame
12 weeks
4

Part 1: Time-normalized number of Investigator-confirmed severe AAE-C1INH attacks during Treatment Phase

Time frame
12 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Pharvaris Netherlands B.V.

Lead sponsor

Source ClinicalTrials.gov
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