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 the participant being capable of and compliant with data recording into an eDiary.
7. Female participants of childbearing age 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 angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications with systemic absorption (such as oral contraceptives or hormonal replacement therapy) 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 enrollment.
13. Known hypersensitivity to deucrictibant or any of the excipients of the study drug
Primary outcome measure(s)
Number of participants with treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs), adverse events of special interest (AESIs), and TEAEs leading to study drug discontinuation — Up to Week 240
Change in heart rate — Up to Week 240
Change in blood pressure — Up to Week 240
Change in body temperature — Up to Week 240
Number of participants with clinically significant changes in Hematology — Up to Week 240 Blood samples will be collected for the analysis of hematology parameters.
Number of participants with clinically significant changes in Clinical Chemistry — Up to Week 240 Blood samples will be collected for the analysis of clinical chemistry parameters.
Number of participants with clinically significant changes in Urinalysis — Up to Week 240 Urine samples will be collected for the analysis of urinalysis parameters.
Change from Baseline electrocardiograms (ECGs) — Up to Week 240 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
Study Site
Scottsdale
Arizona
Study Site
Little Rock
Arkansas
Study Site
San Diego
California
Study Site
Santa Monica
California
Study Site
Walnut Creek
California
Study Site
Colorado Springs
Colorado
Study Site
Wheaton
Illinois
Study Site
St Louis
Missouri
Study Site
Las Vegas
Nevada
Study Site
Hershey
Pennsylvania
Study Site
Dallas
Texas
Study Site
Milwaukee
Wisconsin
Study Site
Corrientes
Argentina
Study Site
Pilar
Argentina
Study Site
Salta
Argentina
Study Site
Box Hill
Australia
Study Site
Parkville
Australia
Study Site
Perth
Australia
Study Site
Sydney
Australia
Study Site
Vienna
Austria
Study Site
Ribeirão Preto
Brazil
Study Site
Salvador
Brazil
Study Site
Santo André
Brazil
Study Site
São Paulo
Brazil
Study Site
São Paulo
Brazil
Study Site
Sofia
Bulgaria
Study Site
Edmonton
Canada
Study Site
Montreal
Canada
Study Site
Beijing
China
Study Site
Grenoble
France
Study Site
Berlin
Germany
Study Site
Frankfurt
Germany
Study Site
Munich
Germany
Study Site
Hong Kong
Hong Kong
Study Site
Budapest
Hungary
Study Site
Dublin
Ireland
Study Site
Milan
Italy
Study Site
Padova
Italy
Study Site
Palermo
Italy
+ 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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