Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE

Launched by PHARVARIS NETHERLANDS B.V. · Oct 31, 2024

Keywords

ClinConnect Summary

This clinical trial is testing a new medication called deucrictibant, which is taken as a daily tablet to help prevent attacks of hereditary angioedema (HAE) in adolescents and adults. HAE is a condition that causes sudden swelling in various parts of the body, which can be very uncomfortable and sometimes dangerous. The study aims to find out if taking this medication regularly can reduce the number of these attacks compared to a placebo, which is a look-alike pill that contains no active medication.

To participate in this trial, you need to be at least 12 years old and have been diagnosed with HAE, experiencing at least three attacks in the three months before the study. If you join, you'll be randomly assigned to either receive the medication or the placebo, and you will need to keep track of your health and any HAE attacks in a diary. The study is currently looking for participants, and it’s important to know that there are some health conditions and recent treatments that might exclude you from joining. If you’re interested, it's a good idea to talk to your doctor about whether this trial is right for you.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Provision of written informed consent/assent.
• • 2. Male or female, aged ≥12 years at the time of providing written informed consent/assent.
• • 3. Diagnosis of hereditary angioedema (HAE)
• • 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 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 Screening (whichever is longer)
• • 3. Has received prior prophylactic treatment with deucrictibant
• • 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