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

Completed · Phase 3 · Has a placebo group

Conditions studied: Hereditary Angioedema, Hereditary Angioedema Type I, Hereditary Angioedema Type II, Hereditary Angioedema Types I and II, Hereditary Angioedema Attack, Hereditary Angioedema With C1 Esterase Inhibitor Deficiency, Hereditary Angioedema - Type 1, Hereditary Angioedema - Type 2, C1 Esterase Inhibitor [C1-INH] Deficiency, C1 Esterase Inhibitor Deficiency, C1 Esterase Inhibitor, Deficiency of, C1 Inhibitor Deficiency, Hereditary Angioedema - Type 3, Hereditary Angioedema Type III

In brief

This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled, 2-period, 2-treatment cross-over study to evaluate the efficacy and safety of orally administered deucrictibant compared to placebo for the on-demand treatment of HAE attacks, including non-severe laryngeal attacks, in participants ≥12 to ≤75 years of age with HAE type 1, type 2, or type 3, a proportion of whom are using long-term prophylactic medication for HAE.

Key facts

Study ID

NCT06343779

Run by

Pharvaris Netherlands B.V.

People needed

134

Starts

2024-02-26

Expected to finish

2025-10-17

Last updated by the study team

2025-11-21

Who can join

Age: 12 and older, up to 75. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Provision of written informed consent/assent.
• Male or female, aged ≥12 to ≤75 years at the time of providing written informed consent/assent.
• Diagnosis of HAE-1/2/3.
• History of at least 2 HAE attacks in the last 3 months before screening.
• Experience with using standard-of-care treatment to effectively manage on-demand treatment for HAE attacks.
• 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.
• Capable of recording, without assistance, electronic HAE diary and ePRO data using an electronic device.
• For adolescent participants aged ≥12 and <18 years of age: body weight ≥40 kg.
• Female participants of childbearing potential must agree to the protocol specified pregnancy testing and contraception methods.

You may not qualify if…

• Any female who is pregnant, plans to become pregnant, or is breastfeeding.
• Any diagnosis of angioedema other than HAE.
• Any clinically significant comorbidity or systemic dysfunction that would interfere with the participant's safety or ability to participate in the study.
• Use of attenuated androgens for short-term prophylaxis within 2 weeks before screening.
• Abnormal hepatic function.
• Abnormal renal function (eGFR <60 ml/min/1.73 m2).
• History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse.
• Has received prior on-demand HAE treatment with deucrictibant.
• 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.
• Prior gene therapy for any indication at any time.
• 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.
• Known hypersensitivity to study drug or any of the excipients of study drug.

Where it is running

• Study Site — Paradise Valley, Arizona, United States
• Study Site — Little Rock, Arkansas, United States
• Study Site — San Diego, California, United States
• Study Site — Santa Monica, California, United States
• Study Site — Walnut Creek, California, United States
• Study Site — Colorado Springs, Colorado, United States
• Study Site — Chevy Chase, Maryland, United States
• Study Site — Boston, Massachusetts, United States
• Study Site — Detroit, Michigan, United States
• Study Site — St Louis, Missouri, United States
• Study Site — Hershey, Pennsylvania, United States
• Study Site — Dallas, Texas, United States
• Study Site — Buenos Aires, Argentina
• Study Site — Salta, Argentina
• Study Site — Campbelltown, New South Wales, Australia
• Study Site — Box Hill, Australia
• Study Site — Graz, Austria
• Study Site — Linz, Austria
• Study Site — Vienna, Austria
• Study Site — Paraná, Brazil
• Study Site — Ribeirão Preto, Brazil
• Study Site — Salvador, Brazil
• Study Site — Santo André, Brazil
• Study Site — São Paulo, Brazil
• Study Site — Birmingham, Alabama, United States

Full record on ClinicalTrials.gov

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