Long-Term, Open-label Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE
Deucrictibant: Deucrictibant extended-release tablet for once daily oral use
Study summary
This is a Phase 3, multicenter, long-term, open-label study to evaluate the safety and efficacy of once-daily orally administered deucrictibant extended-release tablet for prophylaxis to prevent angioedema attacks in participants aged ≥12 years with Hereditary Angioedema
Eligibility
Sex
ALL
Min age
12 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
1. Provision of the signed ICF by the participant and/or legally designated representative.
2. Male or female, aged ≥12 years at the time of providing written informed consent/assent.
3. Diagnosis of hereditary angioedema (HAE)
4. For participants that did not participate in a previous deucrictibant prophylactic study: history of at least 1 attack in the last 3 consecutive months prior to Screening
5. Reliable access and ability to use standard of care on-demand treatments to effectively manage acute HAE attacks.
6. Willing and able to adhere to all protocol requirements, including eDiary and ePRO data recording.
7. Female participants of childbearing must agree to the protocol specified pregnancy testing and contraception methods.
Exclusion Criteria:
1. Any diagnosis of angioedema other than HAE
2. 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 ICF signature (whichever is longer)
3. Prior gene therapy for any indication at any time
4. Participants who discontinued from previous studies with deucrictibant prophylactic and/or on-demand treatment due to safety reasons or compliance issues that, in the opinion of the Investigator, would interfere with the participant's safety or compliance to participate in the study
5. Exposure to ACE inhibitors or any estrogen-containing medications with systemic absorption within 4 weeks of Screening
6. Use of prophylactic treatment for HAE within 2 weeks of Screening for C1INH, oral kallikrein inhibitors, or anti-fibrinolytics; within 4 weeks of Screening for attenuated androgens; within 5 half-lives of Screening for monoclonal antibodies, or within 7 days of Screening for short-term prophylaxis
7. Any females who are pregnant, plan to become pregnant, or are currently breast-feeding
8. Abnormal hepatic function
9. Moderate or severe renal impairment
10. Any clinically significant comorbidity or systemic dysfunction that would interfere with the participant's safety or ability to participate in the study.
11. History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse
12. Use of medications that are moderate and strong inhibitors or strong inducers of CYP3A4 within the last 30 days or within 5 half-lives (whichever is longer) of the time of randomization
13. Known hypersensitivity to deucrictibant or any of the excipients of the study drug
Primary outcome measure(s)
Treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs), adverse events of special interest (AESIs), and TEAEs leading to study drug discontinuation — 130 weeks
Change in heart rate — 130 weeks
Change in blood pressure — 130 weeks
Change in body temperature — 130 weeks
Change in clinical laboratory tests from baseline — 130 weeks Hematology, blood chemistry, and urinalysis. Descriptive in nature, no formal statistical hypothesis testing will be performed.
Change in electrocardiograms (ECGs) from baseline — 130 weeks Digital triplicate 12-lead ECG. Descriptive in nature, no formal statistical hypothesis testing will be performed.
Trial sites (67)
Facility
City
Region
Status
Study Site
Birmingham
Alabama
Recruiting
Study Site
Scottsdale
Arizona
Recruiting
Study Site
Little Rock
Arkansas
Recruiting
Study Site
San Diego
California
Recruiting
Study Site
Santa Monica
California
Recruiting
Study Site
Walnut Creek
California
Recruiting
Study Site
Colorado Springs
Colorado
Recruiting
Study Site
Wheaton
Illinois
Recruiting
Study Site
St Louis
Missouri
Recruiting
Study Site
Las Vegas
Nevada
Recruiting
Study Site
Hershey
Pennsylvania
Recruiting
Study Site
Dallas
Texas
Recruiting
Study Site
Milwaukee
Wisconsin
Recruiting
Study Site
Corrientes
Argentina
Recruiting
Study Site
Pilar
Argentina
Recruiting
Study Site
Salta
Argentina
Recruiting
Study Site
Box Hill
Australia
Recruiting
Study Site
Parkville
Australia
Recruiting
Study Site
Perth
Australia
Recruiting
Study Site
Sydney
Australia
Recruiting
Study Site
Vienna
Austria
Recruiting
Study Site
Ribeirão Preto
Brazil
Recruiting
Study Site
Salvador
Brazil
Recruiting
Study Site
Santo André
Brazil
Recruiting
Study Site
São Paulo
Brazil
Recruiting
Study Site
São Paulo
Brazil
Recruiting
Study Site
Sofia
Bulgaria
Recruiting
Study Site
Edmonton
Canada
Recruiting
Study Site
Montreal
Canada
Recruiting
Study Site
Beijing
China
Recruiting
Study Site
Grenoble
France
Recruiting
Study Site
Berlin
Germany
Recruiting
Study Site
Frankfurt
Germany
Recruiting
Study Site
Munich
Germany
Recruiting
Study Site
Hong Kong
Hong Kong
Recruiting
Study Site
Budapest
Hungary
Recruiting
Study Site
Dublin
Ireland
Recruiting
Study Site
Milan
Italy
Recruiting
Study Site
Padova
Italy
Recruiting
Study Site
Palermo
Italy
Recruiting
+ 27 more sites — see the full list on the official registry below.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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