Long-Term, Open-label Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE

Launched by PHARVARIS NETHERLANDS B.V. · Nov 5, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new medication called deucrictibant, which is an oral tablet taken once a day. The goal is to see if this medication can help prevent attacks of angioedema in people with Hereditary Angioedema (HAE). HAE is a condition that causes sudden swelling in various parts of the body, which can be very uncomfortable and sometimes dangerous. This study is open to both adolescents and adults aged 12 and older who have been diagnosed with HAE and have had at least one attack in the past three months.

To participate, individuals need to give their consent and have access to treatments for HAE attacks. They should not have other types of angioedema, recent participation in other drug studies, or certain medical conditions that could affect their safety. Those who join the trial will take the medication daily and will be asked to keep track of their health and any attacks they experience using a diary. This study is currently recruiting participants, and it's a great opportunity for those who are looking for new ways to manage their HAE.

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. 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