Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema

Recruitment Status
COMPLETED
(See Contacts and Locations)Verified October 2025 by Pharvaris Netherlands B.V.
Sponsor
Pharvaris Netherlands B.V.
Information Provided by (Responsible Party)
Pharvaris Netherlands B.V.
Clinicaltrials.gov Identifier
NCT06343779
Other Study ID Numbers:
PHA022121-C306
First Submitted
March 17, 2024
First Posted
April 2, 2024
Last Update Posted
November 20, 2025
Last Verified
October 2025

ClinicalTrials.gov processed this data on November 2025. Link to the current ClinicalTrials.gov record .

History of Changes

Study Details

Study Description

The study consists of a Screening Phase during which eligibility is confirmed, a Treatment Phase in which participants will be randomized and receive double blinded study drug to treat 2 qualifying HAE attacks (i.e., 2 Treatment Periods within the Treatment Phase), and an End-of-Study Follow-up Phase after the second attack treated with study drug. In addition, for adolescent participants (age ≥12 to \<18 years), PK samples are collected after administration of deucrictibant at Day 1 in a non-attack state.

Condition or DiseaseIntervention/Treatment
Hereditary AngioedemaHereditary Angioedema Type IHereditary Angioedema Type IIHereditary Angioedema Types I and IIHereditary Angioedema AttackHereditary Angioedema With C1 Esterase Inhibitor DeficiencyHereditary Angioedema - Type 1Hereditary Angioedema - Type 2C1 Esterase Inhibitor [C1-INH] DeficiencyC1 Esterase Inhibitor DeficiencyC1 Esterase Inhibitor, Deficiency ofC1 Inhibitor DeficiencyHereditary Angioedema - Type 3Hereditary Angioedema Type III
Drug: Deucrictibant, PlaceboDrug: Deucrictibant, Placebo

Study Design

Study TypeInterventional
Actual Enrollment134 participants
Design AllocationRandomized
Interventional ModelCrossover Assignment
MaskingQuadruple
Primary PurposeTreatment
Official TitleA Phase 3, Randomized, Double-blind, Placebo-controlled, Cross-over Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Attacks in Adolescents and Adults With Hereditary Angioedema
Study Start DateFebruary 25, 2024
Actual Primary Completion DateOctober 16, 2025
Actual Study Completion DateOctober 16, 2025

Groups and Cohorts

Group/CohortIntervention/Treatment
Arm 1
Deucrictibant administered for first HAE attack, placebo administered for second HAE attack.
Drug: Deucrictibant, Placebo
Deucrictibant Soft Capsules for Oral Use
Arm 2
Placebo administered for first HAE attack, deucrictibant administered for second HAE attack.
Drug: Deucrictibant, Placebo
Deucrictibant Soft Capsules for Oral Use

Outcome Measures

Primary Outcome Measures
  1. Time to onset of symptom relief, defined as Patient Global Impression of Change (PGI-C) rating of at least "a little better" for 2 consecutive timepoints within 12 hours post-treatment.
    The PGI-C (7-point scale) is used to evaluate the change in the HAE attack symptoms as compared to pre-treatment.
Secondary Outcome Measures
  1. Proportion of study drug-treated attacks achieving PGI-C rating of at least "a little better" at 4 hours post-treatment.
    The PGI-C (7-point scale) is used to evaluate the change in the HAE attack symptoms as compared to pre-treatment.
  2. Time to substantial symptom relief, defined as achieving PGI-C rating of at least "better" for 2 consecutive timepoints within 12 hours post-treatment.
    The PGI-C (7-point scale) is used to evaluate the change in the HAE attack symptoms as compared to pre-treatment.
  3. Time to substantial symptom relief by Patient Global Impression of Severity (PGI-S).
    Defined as achieving &ge;1 point reduction in PGI-S (5-point scale) from pre-treatment for 2 consecutive timepoints within 12 hours post-treatment.
  4. Time to complete symptom resolution, defined as achieving PGI-S rating of "none" within 48 hours post-treatment.
    The PGI-S (5-point scale) is used to evaluate the severity of HAE attack symptoms.
  5. Time to End of Progression (EoP) in attack symptoms within 12 hours.
    EoP time defined as the earliest post-treatment timepoint after which all subsequent PGI-C ratings are stable or improved.
  6. Proportion of study drug-treated attacks requiring rescue medication within 24 hours post-treatment.
    Rescue medication is defined as the participant&#039;s usual acute on-demand HAE treatment taken if symptoms persist or progress after study drug administration.
  7. Proportion of attacks achieving symptom resolution.
    Defined as achieving PGI-S rating of &quot;none&quot; with one dose of study drug at 24 hours post-treatment.
  8. Time to substantial symptom relief by Angioedema Symptom Rating Scale (AMRA).
    Defined as a &ge;50% reduction in AMRA composite score from pre-treatment for 2 consecutive timepoints within 12 hours post-treatment.
  9. Time to almost complete or complete symptom relief by AMRA.
    Defined as all item scores in AMRA having a value &le;10 for 2 consecutive timepoints within 24 hours post-treatment.
  10. Proportion of study drug-treated attacks reaching almost complete or complete symptom relief by AMRA.
    Defined as all item scores in AMRA having a value &le;10 at 24 hours post-treatment.
  11. Time to EoP in attack symptoms within 12 hours.
    Defined as the earliest post-treatment timepoint after which every individual AMRA item is stable or improved at all subsequent timepoints.

Eligibility Criteria

Ages Eligible for Study(Child, Adult, Older Adult)
Sexes Eligible for StudyAll
Accepts Healthy VolunteersNo
Inclusion Criteria
1. Provision of written informed consent/assent. 2. Male or female, aged ≥12 to ≤75 years at the time of providing written informed consent/assent. 3. Diagnosis of HAE-1/2/3. 4. History of at least 2 HAE attacks in the last 3 months before screening. 5. Experience with using standard-of-care treatment to effectively manage on-demand treatment for HAE attacks. 6. Participants on long-term prophylactic therapy with plasma-derived C1-INH (danazol, anti-fibrinolytics, berotralstat, or lanadelumab) must be on a stable dose and regimen and intend to remain on the same dose for 6 months before screening and the duration of the study. OR, Participant has stopped using plasma-derived C1-INH (danazol, anti-fibrinolytics, berotralstat) at least 2 weeks or lanadelumab at least 10 weeks before screening. 7. Capable of recording, without assistance, electronic HAE diary and ePRO data using an electronic device. 8. For adolescent participants aged ≥12 and \<18 years of age: body weight ≥40 kg. 9. Female participants of childbearing potential must agree to the protocol specified pregnancy testing and contraception methods.
Exclusion Criteria
1. Any female who is pregnant, plans to become pregnant, or is breastfeeding. 2. Any diagnosis of angioedema other than HAE. 3. Any clinically significant comorbidity or systemic dysfunction that would interfere with the participant's safety or ability to participate in the study. 4. Use of attenuated androgens for short-term prophylaxis within 2 weeks before screening. 5. Abnormal hepatic function. 6. Abnormal renal function (eGFR \<60 ml/min/1.73 m2). 7. History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse. 8. Has received prior on-demand HAE treatment with deucrictibant. 9. Currently participating in any other investigational drug study or receiving other investigational treatment within the last 30 days, or within 5 half-lives (whichever is longer) of the time of randomization. 10. Prior gene therapy for any indication at any time. 11. Use of concomitant medications with systemic absorption that are strong inhibitors of CYP3A4 or strong inducers of CYP3A4 within the last 30 days, or within 5 half-lives (whichever is longer) of the time of randomization. 12. Known hypersensitivity to study drug or any of the excipients of study drug.

Contacts and Locations

Sponsors and CollaboratorsPharvaris Netherlands B.V.
Locations
Study Site | Birmingham Alabama, United States, 35209Study Site | Paradise Valley Arizona, United States, 85258Study Site | Little Rock Arkansas, United States, 72205Study Site | San Diego California, United States, 92122Study Site | Santa Monica California, United States, 90404Study Site | Walnut Creek California, United States, 94598Study Site | Colorado Springs Colorado, United States, 80907-6231Study Site | Chevy Chase Maryland, United States, 20815Study Site | Boston Massachusetts, United States, 02115Study Site | Detroit Michigan, United States, 48202Study Site | St Louis Missouri, United States, 63141Study Site | Hershey Pennsylvania, United States, 17033Study Site | Dallas Texas, United States, 75231Study Site | Buenos Aires , Argentina, B1629AHJStudy Site | Salta , Argentina, 4400Study Site | Campbelltown New South Wales, Australia, 2560Study Site | Box Hill , Australia, 3128Study Site | Graz , Austria, 8036Study Site | Linz , Austria, 4021Study Site | Vienna , Austria, 1090Study Site | Paraná , Brazil, 80810-100Study Site | Ribeirão Preto , Brazil, 14048-900Study Site | Salvador , Brazil, 41950-640Study Site | Santo André , Brazil, 09060-870Study Site | São Paulo , Brazil, 05403-000Study Site | Sofia , Bulgaria, 1431Study Site | Sofia , Bulgaria, 1680Study Site | Edmonton Alberta, Canada, T6G 1Z1Study Site | Bogotá , Colombia, 00Study Site | Bogotá , Colombia, 111221Study Site | Bogotá , Colombia, 111711Study Site | Medellín , Colombia, 050021Study Site | Brno , Czechia, 602 00Study Site | Lille , France, 59037Study Site | Paris , France, 75571Study Site | Berlin , Germany, 12203Study Site | Frankfurt am Main , Germany, 60590Study Site | Frankfurt am Main , Germany, 60596Study Site | Lübeck , Germany, 23538Study Site | Hong Kong , Hong Kong, Study Site | Budapest , Hungary, 1088Study Site | Dublin , Ireland, D08 A978Study Site | Catania , Italy, 95124Study Site | Milan , Italy, 20138Study Site | Milan , Italy, 20097Study Site | Padova , Italy, 35128Study Site | Palermo , Italy, 90146Study Site | Roma , Italy, 00133Study Site | Chiba , Japan, 260-8677Study Site | Hiroshima , Japan, 730-8518Study Site | Kanagawa , Japan, 216-8511Study Site | Osaka , Japan, 569-8686Study Site | Tokyo , Japan, 113-8431Study Site | Amsterdam , Netherlands, 1105 AZStudy Site | Skopje , North Macedonia, 1000Study Site | Krakow , Poland, 31-503Study Site | San Juan , Puerto Rico, 00918Study Site | San Juan , Puerto Rico, 00927Study Site | Sângeorgiu de Mureş , Romania, 547530Study Site | Riyadh , Saudi Arabia, 11471Study Site | Singapore , Singapore, 308433Study Site | Cape Town , South Africa, 7700Study Site | Daegu , South Korea, 41944Study Site | Seoul , South Korea, 03080Study Site | Barcelona , Spain, 08035Study Site | Barcelona , Spain, 08907Study Site | Lund , Sweden, 22185Study Site | Ankara , Turkey (Türkiye), 06203Study Site | Istanbul , Turkey (Türkiye), 34093Study Site | Izmir , Turkey (Türkiye), 35100Study Site | Bristol , United Kingdom, BS10 5NBStudy Site | Camberley , United Kingdom, GU16 7UJStudy Site | Cambridge , United Kingdom, CB2 0QQStudy Site | Leeds , United Kingdom, LS9 7TFStudy Site | London , United Kingdom, E1 2ESStudy Site | Plymouth , United Kingdom, PL6 8DH
Investigators
Study Director: Study Director, Pharvaris, Pharvaris Netherlands B.V.