A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Orally Administered Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema

Pharvaris Netherlands B.V.57 个研究点分布在 24 个国家目标入组 81 人2024年12月1日

适应症Hereditary Angioedema (HAE)

干预措施Deucrictibant Placebo

概览

阶段: 3 期
干预措施: Deucrictibant
疾病 / 适应症: Hereditary Angioedema (HAE)
发起方: Pharvaris Netherlands B.V.
入组人数: 81
试验地点: 57
主要终点: Time-normalized (per 4 weeks) number of Investigator-confirmed HAE attacks during the 24-week Treatment Period
状态: 进行中（未招募）
最后更新: 2个月前

概览研究者入排标准研究组 & 干预措施结局指标研究点相似试验相关资讯

概览

简要总结

This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of once-daily orally administered deucrictibant extended-release tablet compared to placebo for prophylaxis to prevent angioedema attacks in participants aged ≥ 12 years with hereditary angioedema.

详细描述

The study consists of a Screening Period during which eligibility is confirmed, a Treatment Period of 24 weeks, and a Follow-up Period of maximum 4 weeks or subjects may roll over into the open-label study PHA022121-C307 (CHAPTER-4). During the Treatment period participants will receive blinded study drug (deucrictibant or placebo randomized in a 2:1 ratio). Participants will undergo regular efficacy and safety assessments, complete an electronic diary daily, and also complete questionnaires at predefined timepoints during the study.

注册库

clinicaltrials.gov

开始日期

2024年12月1日

结束日期

2026年8月1日

最后更新

2个月前

研究类型

Interventional

研究设计

Parallel

性别

All

研究者

发起方

Pharvaris Netherlands B.V.

责任方

Sponsor

入排标准

入选标准

•Provision of written informed consent/assent.
•Male or female, aged ≥12 years at the time of providing written informed consent/assent.
•Diagnosis of hereditary angioedema (HAE)
•History of at least 3 HAE attacks within the 3 consecutive months prior to Screening Visit
•Predefined number of attacks during the Screening Period
•Reliable access and ability to use standard of care on-demand treatments to effectively manage acute HAE attacks.
•Willing and able to adhere to all protocol requirements, including eDiary and ePRO data recording.
•Female participants of childbearing potential must agree to the protocol specified pregnancy testing and contraception methods.

排除标准

•Any diagnosis of angioedema other than HAE
•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)
•Has received prior prophylactic treatment with deucrictibant
•Exposure to ACE inhibitors or any estrogen-containing medications with systemic absorption within 4 weeks of Screening
•Prior gene therapy for any indication at any time
•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
•Any females who are pregnant, plan to become pregnant, or are currently breast-feeding
•Abnormal hepatic function
•Moderate or severe renal impairment
•Any clinically significant comorbidity or systemic dysfunction that would interfere with the participant's safety or ability to participate in the study.

研究组 & 干预措施

Active

Deucrictibant 40mg extended-release tablet by mouth once daily

干预措施: Deucrictibant

Placebo

Placebo 1 tablet by mouth once daily

干预措施: Placebo

结局指标

主要结局

Time-normalized (per 4 weeks) number of Investigator-confirmed HAE attacks during the 24-week Treatment Period

时间窗: 24 weeks

次要结局

Time-normalized number of Investigator-confirmed HAE attacks treated with on-demand medication during the 24-week Treatment Period(24 weeks)
Time-normalized number of Investigator-confirmed moderate or severe HAE attacks during the 24-week Treatment Period(24 weeks)
Time-normalized number of Investigator-confirmed severe HAE attacks during the 24-week Treatment Period(24 weeks)
Proportion of participants achieving ≥50% reduction in HAE attack rate relative to baseline during the 24-week Treatment Period(24 weeks)
Proportion of participants achieving ≥70% reduction in HAE attack rate relative to baseline during the 24-week Treatment Period(24 weeks)
Proportion of participants achieving ≥90% reduction in HAE attack rate relative to baseline during the 24-week Treatment Period(24 weeks)
Proportion of participants that are HAE attack-free during the 24-week Treatment Period(24 weeks)
Proportion of time without angioedema symptoms during the 24-week Treatment Period(24 weeks)
Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), adverse events of special interest (AESIs), and TEAEs leading to study drug discontinuation(Up to 38 weeks)
Pharmacokinetics [PK]: Deucrictibant plasma concentration time profiles(24 weeks)
Patient reported outcome: Angioedema Quality of Life (AE-QoL) questionnaire(24 weeks)
Patient reported outcome: Patient Global Assessment of Change (PGA-Change)(24 weeks)
Patient reported outcome: Angioedema Control Test 4-week version (AECT-4wk)(24 weeks)
Patient reported outcome: Work Productivity and Activity Impairment Questionnaire - Specific Health Problem (WPAI-SHP)(Up to 34 weeks)
Patient reported outcome: Abbreviated Treatment Satisfaction Questionnaire for Medication (TSQM-9)(Up to 34 weeks)