Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema

Last updated: May 2, 2025
Sponsor: Pharvaris Netherlands B.V.
Overall Status: Active - Enrolling

Phase

2/3

Condition

Allergy

Hives (Urticaria)

Urticaria

Treatment

deucrictibant selected dose

deucrictibant low dose

deucrictibant medium dose

Clinical Study ID

NCT05396105
PHA022121-C303
2023-505766-28-00
  • Ages > 12
  • All Genders

Study Summary

This study evaluates the safety and efficacy of long-term on-demand treatment with orally administered deucrictibant for acute hereditary angioedema (HAE) attacks, including laryngeal attacks. The study will enroll patients from Study PHA022121-C201 (NCT04618211) and Study PHA022121-C306 (NCT06343779) who elect to participate in this extension study and meet the eligibility requirements.

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

  1. Provision of written informed consent. If the participant is a minor (i.e., <18years of age or as determined by local law), consent will be obtained from theparticipant's parent/legally designated representative/guardian and written assentwill be obtained from the participant, per country regulations.

  2. For participants from Study C201, received at least one dose of study drug (including the non-attack visit) in Study C201. For participants from Study C306,participant was randomized (and for adolescent participants 12 to <18 years receiveda dose of study drug in a non-attack state at Visit 1) and completed Study C306,with 2 attacks treated, or after closure of that study by the Sponsor. Enrollment of adolescents (≥12 to <18 years or age of adulthood as defined locally)from these studies is with consideration of local age requirements.

  3. Female participants of childbearing potential (or who become of childbearingpotential during the study) must agree to the protocol-specified pregnancy testingand to be abstinent from heterosexual intercourse or to use an acceptablecontraception method as defined in the protocol and as available locally fromenrollment until 30 days after the last study drug administration.

  4. In the opinion of the Investigator, the participant (and parent/caregiver foradolescent participants) is willing and able to comply with the protocol.

Exclusion

Key Exclusion Criteria:

  1. Any female who is pregnant, plans to become pregnant, or is breast-feeding.

  2. Any other systemic disease (e.g., cardiovascular, gastrointestinal, renal,respiratory, neurological) or significant disease or disorder that, in the opinionof the Investigator, would interfere with the participant's safety or ability toparticipate in the study.

  3. Use of lanadelumab for long-term HAE prophylactic therapy within 12 weeks prior toenrollment in Part A.

  4. For Part A: Use of C1-esterase inhibitor, oral kallikrein inhibitors, attenuatedandrogens, anti-fibrinolytics, or monoclonal HAE therapy within a defined periodprior to enrolment. For Part B: If a participant is receiving long-term prophylactic therapy with one ofthe following medications indicated for HAE: plasma-derived C1-INH, danazol at lessthan or equal to 200 mg/day, anti-fibrinolytics, berotralstat, or lanadelumab, theymust be on a stable dose and regimen for at least 3 months before screening andintends to remain on the same dose for the duration of the study.

  5. History of alcohol or drug abuse within defined period, or current evidence ofsubstance dependence or abuse

  6. Participation in any other investigational drug study within defined period

  7. Discontinued from parent study after enrollment for any study drug-related safetyreason or non-compliance including significant protocol deviation.

  8. Use of concomitant medications that are strong CYP3A4 inhibitors (e.g.,clarithromycin, erythromycin, itraconazole, ketoconazole, ritonavir) or strongCYP3A4 inducers (e.g., carbamazepine and phenytoin).

Study Design

Total Participants: 140
Treatment Group(s): 4
Primary Treatment: deucrictibant selected dose
Phase: 2/3
Study Start date:
December 28, 2022
Estimated Completion Date:
June 30, 2027

Study Description

Part A of the study will enroll adult participants from Study PHA022121-C201. The double-blind treatment assignment from Study PHA022121-C201 will be maintained.

Part B will include participants rolling over from Part A and additionally enroll participants from Study PHA022121-C201 who did not participate in Part A, and participants from Study PHA022121-C306 who elect to participate in this extension study and meet the eligibility requirements.

Connect with a study center

  • Study site

    Buenos Aires, B1629AHJ
    Argentina

    Site Not Available

  • Study site

    Campbelltown, New South Wales 2560
    Australia

    Site Not Available

  • Study site

    Graz, 8036
    Austria

    Site Not Available

  • Study site

    Wien, 1090
    Austria

    Site Not Available

  • Study site

    Sofia, 1680
    Bulgaria

    Site Not Available

  • Study site

    Edmonton, Alberta T6G 1Z1
    Canada

    Site Not Available

  • Study site

    Montréal, Quebec
    Canada

    Site Not Available

  • Study site

    Brno, 602 00
    Czechia

    Site Not Available

  • Study site

    Grenoble, 38043
    France

    Site Not Available

  • Study site

    La Tronche, 38700
    France

    Site Not Available

  • Study site

    Paris, 75571
    France

    Site Not Available

  • Study site

    Berlin, 12203
    Germany

    Site Not Available

  • Study site

    Frankfurt am Main, 60590
    Germany

    Site Not Available

  • Study site

    Lubeck, 23538
    Germany

    Site Not Available

  • Study site

    Hong Kong,
    Hong Kong

    Site Not Available

  • Study site

    Budapest, 1088
    Hungary

    Site Not Available

  • Study site

    Ashkelon, 78278
    Israel

    Site Not Available

  • Study site

    Catania, 95124
    Italy

    Site Not Available

  • Study site

    Milano, 20097
    Italy

    Site Not Available

  • Study site

    Napoli, 80131
    Italy

    Site Not Available

  • Study site

    Padova, 35128
    Italy

    Site Not Available

  • Study site

    Palermo, 90146
    Italy

    Site Not Available

  • Study site

    Roma, 00133
    Italy

    Site Not Available

  • Study site

    Hiroshima, 730-8518
    Japan

    Site Not Available

  • Study site

    Kanagawa, 216-8511
    Japan

    Site Not Available

  • Study site

    Osaka, 569-8686
    Japan

    Site Not Available

  • Study site

    Tokyo, 133-8431
    Japan

    Site Not Available

  • Study site

    Daegu, 41944
    Korea, Republic of

    Site Not Available

  • Study site

    Seoul, 03080
    Korea, Republic of

    Site Not Available

  • Study site

    Amsterdam, 1005 AZ
    Netherlands

    Site Not Available

  • Study site

    Kraków, 31-503
    Poland

    Site Not Available

  • Study site

    San Juan, 918
    Puerto Rico

    Site Not Available

  • Study site

    Cape Town, 7700
    South Africa

    Site Not Available

  • Study site

    Barcelona, 08035
    Spain

    Site Not Available

  • Study site

    Madrid, 28007
    Spain

    Site Not Available

  • Study site

    Lund, 22185
    Sweden

    Site Not Available

  • Study site

    Ankara, 06230
    Turkey

    Site Not Available

  • Study site

    Istanbul, 34093
    Turkey

    Site Not Available

  • Study site

    Birmingham, Alabama 35209
    United States

    Site Not Available

  • Study site

    Scottsdale, Arizona 85258
    United States

    Site Not Available

  • Study site

    Little Rock, Arkansas 72205
    United States

    Site Not Available

  • Study site

    Santa Monica, California 90404
    United States

    Site Not Available

  • Study site

    Walnut Creek, California 94598
    United States

    Site Not Available

  • Study site

    Colorado Springs, Colorado 80907
    United States

    Site Not Available

  • Study site

    Chevy Chase, Maryland 20815
    United States

    Site Not Available

  • Study site

    Detroit, Michigan 48202
    United States

    Site Not Available

  • Study site

    St. Louis, Missouri 63141
    United States

    Site Not Available

  • Study site

    Hershey, Pennsylvania 17033
    United States

    Site Not Available

  • Study site

    Dallas, Texas 75231
    United States

    Site Not Available

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