Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema

Launched by PHARVARIS NETHERLANDS B.V. · Mar 26, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new oral medication called deucrictibant to see if it can help treat attacks of hereditary angioedema (HAE) in adolescents and adults. HAE is a condition that causes sudden swelling, which can be painful and sometimes dangerous. The trial aims to compare the effectiveness and safety of deucrictibant against a placebo (a pill with no active medication) to find out how well it works for managing these attacks. Participants in the study must be between 12 and 75 years old, have a diagnosis of type 1 or type 2 HAE, and have experienced at least two attacks in the past three months.

To participate, individuals should be able to provide consent, have experience managing their HAE attacks, and be stable on their current long-term treatment if they are using one. Participants will use an electronic diary to track their symptoms and medication use throughout the study. It's important to know that some people may not be eligible if they are pregnant, have certain other health conditions, or have used specific medications recently. Overall, this trial offers a chance to explore a new option for effectively managing HAE attacks, which could improve the quality of life for those affected by this condition.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Provision of written informed consent/assent.
• • 2. Male or female, aged ≥12 to ≤75 years at the time of providing written informed consent/assent.
• • 3. Diagnosis of HAE-1/2/3.
• • 4. History of at least 2 HAE attacks in the last 3 months before screening.
• • 5. Experience with using standard-of-care treatment to effectively manage on-demand treatment for HAE attacks.
• • 6. Participants on long-term prophylactic therapy with plasma-derived C1-INH (danazol, anti-fibrinolytics, berotralstat, or lanadelumab) must be on a stable dose and regimen and intend to remain on the same dose for 6 months before screening and the duration of the study. OR, Participant has stopped using plasma-derived C1-INH (danazol, anti-fibrinolytics, berotralstat) at least 2 weeks or lanadelumab at least 10 weeks before screening.
• • 7. Capable of recording, without assistance, electronic HAE diary and ePRO data using an electronic device.
• • 8. For adolescent participants aged ≥12 and \<18 years of age: body weight ≥40 kg.
• • 9. Female participants of childbearing potential must agree to the protocol specified pregnancy testing and contraception methods.
• Exclusion Criteria:
• • 1. Any female who is pregnant, plans to become pregnant, or is breastfeeding.
• • 2. Any diagnosis of angioedema other than HAE.
• • 3. Any clinically significant comorbidity or systemic dysfunction that would interfere with the participant's safety or ability to participate in the study.
• • 4. Use of attenuated androgens for short-term prophylaxis within 2 weeks before screening.
• • 5. Abnormal hepatic function.
• • 6. Abnormal renal function (eGFR \<60 ml/min/1.73 m2).
• • 7. History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse.
• • 8. Has received prior on-demand HAE treatment with deucrictibant.
• • 9. Currently participating in any other investigational drug study or receiving other investigational treatment within the last 30 days, or within 5 half-lives (whichever is longer) of the time of randomization.
• • 10. Prior gene therapy for any indication at any time.
• • 11. Use of concomitant medications with systemic absorption that are strong inhibitors of CYP3A4 or strong inducers of CYP3A4 within the last 30 days, or within 5 half-lives (whichever is longer) of the time of randomization.
• • 12. Known hypersensitivity to study drug or any of the excipients of study drug.