临床试验/NCT06343779

NCT06343779已完成3 期

A Phase 3, Randomized, Double-blind, Placebo-controlled, Cross-over Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Attacks in Adolescents and Adults With Hereditary Angioedema

Pharvaris Netherlands B.V.1 个研究点分布在 1 个国家目标入组 134 人开始时间: 2024年2月26日最近更新: 2025年11月21日

适应症

干预措施

试验速览

阶段: 3 期
状态: 已完成
发起方
入组人数: 134
试验地点: 1
主要终点: Time to onset of symptom relief, defined as Patient Global Impression of Change (PGI-C) rating of at least "a little better" for 2 consecutive timepoints within 12 hours post-treatment.

研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验相关资讯

研究概览

简要总结

This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled, 2-period, 2-treatment cross-over study to evaluate the efficacy and safety of orally administered deucrictibant compared to placebo for the on-demand treatment of HAE attacks, including non-severe laryngeal attacks, in participants ≥12 to ≤75 years of age with HAE type 1, type 2, or type 3, a proportion of whom are using long-term prophylactic medication for HAE.

详细描述

The study consists of a Screening Phase during which eligibility is confirmed, a Treatment Phase in which participants will be randomized and receive double blinded study drug to treat 2 qualifying HAE attacks (i.e., 2 Treatment Periods within the Treatment Phase), and an End-of-Study Follow-up Phase after the second attack treated with study drug. In addition, for adolescent participants (age ≥12 to <18 years), PK samples are collected after administration of deucrictibant at Day 1 in a non-attack state.

研究设计

研究类型: Interventional
分配方式: Randomized
干预模型: Crossover
主要目的: Treatment
盲法: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

入排标准

年龄范围: 12 Years 至 75 Years（Child, Adult, Older Adult）
性别: All
接受健康志愿者: 否

入选标准

•Provision of written informed consent/assent.
•Male or female, aged ≥12 to ≤75 years at the time of providing written informed consent/assent.
•Diagnosis of HAE-1/2/
•History of at least 2 HAE attacks in the last 3 months before screening.
•Experience with using standard-of-care treatment to effectively manage on-demand treatment for HAE attacks.
•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.
•Capable of recording, without assistance, electronic HAE diary and ePRO data using an electronic device.
•For adolescent participants aged ≥12 and <18 years of age: body weight ≥40 kg.
•Female participants of childbearing potential must agree to the protocol specified pregnancy testing and contraception methods.

排除标准

•Any female who is pregnant, plans to become pregnant, or is breastfeeding.
•Any diagnosis of angioedema other than HAE.
•Any clinically significant comorbidity or systemic dysfunction that would interfere with the participant's safety or ability to participate in the study.
•Use of attenuated androgens for short-term prophylaxis within 2 weeks before screening.
•Abnormal hepatic function.
•Abnormal renal function (eGFR <60 ml/min/1.73 m2).
•History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse.
•Has received prior on-demand HAE treatment with deucrictibant.
•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.
•Prior gene therapy for any indication at any time.
•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.
•Known hypersensitivity to study drug or any of the excipients of study drug.

研究组 & 干预措施

Arm 1

Experimental

Deucrictibant administered for first HAE attack, placebo administered for second HAE attack.

干预措施: Deucrictibant, Placebo (Drug)

Arm 2

Experimental

Placebo administered for first HAE attack, deucrictibant administered for second HAE attack.

干预措施: Deucrictibant, Placebo (Drug)

结局指标

主要结局

Time to onset of symptom relief, defined as Patient Global Impression of Change (PGI-C) rating of at least "a little better" for 2 consecutive timepoints within 12 hours post-treatment.

时间窗: Pre-treatment to 12 hours post-treatment.

The PGI-C (7-point scale) is used to evaluate the change in the HAE attack symptoms as compared to pre-treatment.

次要结局

Proportion of study drug-treated attacks achieving PGI-C rating of at least "a little better" at 4 hours post-treatment.(Pre-treatment to 4 hours post-treatment.)
Time to substantial symptom relief, defined as achieving PGI-C rating of at least "better" for 2 consecutive timepoints within 12 hours post-treatment.(Pre-treatment to 12 hours post-treatment.)
Time to substantial symptom relief by Patient Global Impression of Severity (PGI-S).(Pre-treatment to 12 hours post-treatment.)
Time to complete symptom resolution, defined as achieving PGI-S rating of "none" within 48 hours post-treatment.(Pre-treatment to 48 hours post-treatment.)
Time to End of Progression (EoP) in attack symptoms within 12 hours.(Pre-treatment to 12 hours post-treatment.)
Time to EoP in attack symptoms within 12 hours.(Pre-treatment to 12 hours post-treatment.)
Proportion of study drug-treated attacks reaching almost complete or complete symptom relief by AMRA.(Pre-treatment to 24 hours post-treatment.)
Proportion of attacks achieving symptom resolution.(Pre-treatment to 24 hours post-treatment.)
Proportion of study drug-treated attacks requiring rescue medication within 24 hours post-treatment.(Pre-treatment to 24 hours post-treatment.)
Time to substantial symptom relief by Angioedema Symptom Rating Scale (AMRA).(Pre-treatment to 12 hours post-treatment.)
Time to almost complete or complete symptom relief by AMRA.(Pre-treatment to 24 hours post-treatment.)

研究者

发起方

Pharvaris Netherlands B.V.

申办方类型

Industry

责任方

Sponsor

研究点 (1)

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