Hereditary Angioedema
Conditions
Interventions
Deucrictibant 40mg extended-release tablet by mouth once daily for 24 weeks.
Sponsors
Sundberg Aimee
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Provision of written informed consent/assent. 2. Male or female, aged 12 years and older at the time of providing written informed consent/assent. 3. Diagnosis of hereditary angioedema (HAE) type 1/2 4. History of at least 3 HAE attacks within the 3 consecutive months prior to Screening Visit 5. Predefined number of attacks during the Screening Period 6. Reliable access and ability to use standard of care on-demand treatments to effectively manage acute HAE attacks. 7. Willing and able to adhere to all protocol requirements, including eDiary and ePRO data recording. 8. Female participants of childbearing potential must agree to the protocol specified pregnancy testing and a highly effective contraception methods as available locally.
Exclusion criteria
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 Screening (whichever is longer) 3. Has received prior prophylactic treatment or on-demand treatment with deucrictibant, with the exception of participants from Study PHA022121-C306 4. Exposure to ACE inhibitors or any estrogen-containing medications with systemic absorption within 4 weeks of Screening 5. Prior gene therapy for any indication at any time 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Time-normalized (per 4 weeks) number of Investigator-confirmed HAE attacks during the 24-week Treatment Period. | — |
Countries
Argentina, Brazil, Bulgaria, Canad, China, Colombia, France, Germany, Hong Kong, Hungary, Ireland, Italy, Japan, New Zealand, Poland, Puerto Rico, Qatar, Romania, Saudi Arabia, Singapore, Slovakia, South Africa, South Korea, Spain, Sweden, Switzerland, Turkey, UK, USA
Contacts
Public ContactAkane Maeno
A2 Healthcare Corporation
Outcome results
None listed