NCT06669754进行中（未招募）3 期
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 人开始时间: 2024年12月1日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- 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
研究概览
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 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 —（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 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.
- •History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse
- •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
- •Known hypersensitivity to deucrictibant or any of the excipients of the study drug
研究组 & 干预措施
Active
Experimental
Deucrictibant 40mg extended-release tablet by mouth once daily
干预措施: Deucrictibant (Drug)
Placebo
Experimental
Placebo 1 tablet by mouth once daily
干预措施: Placebo (Drug)
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)
研究者
研究点 (57)
Loading locations...
相似试验
已完成
3 期
Study to Evaluate the Efficacy and Safety of Imsidolimab (ANB019) in the Treatment of Subjects With GPPGeneralized Pustular PsoriasisNCT05352893Vanda Pharmaceuticals45
已完成
3 期
Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAEHAEHereditary AngioedemaNCT03485911BioCryst Pharmaceuticals121
已完成
3 期
Efficacy and Safety of Oral Ibrexafungerp (SCY-078) vs. Placebo in Subjects With Acute Vulvovaginal Candidiasis (VANISH 303)Candida VulvovaginitisNCT03734991Scynexis, Inc.376
已完成
3 期
Efficacy and Safety of Oral Ibrexafungerp (SCY-078) vs. Placebo in Subjects With Acute Vulvovaginal CandidiasisCandida VulvovaginitisNCT03987620Scynexis, Inc.455
已完成
3 期
PAVES: Pegfilgrastim Anti-vascular Endothelial Growth Factor (VEGF) Evaluation StudyFeverLocally AdvancedMetastatic Colorectal CancerNeutropeniaRectal CancerCancerColon CancerColorectal CancerNCT00911170Amgen847
相关资讯
Pharvaris Expands Deucrictibant Development to Acquired Angioedema Following Promising Early DataPharvaris announces plans to initiate a clinical trial for deucrictibant in acquired angioedema, expanding beyond its ongoing Phase 3 trials in hereditary angioedema. The decision follows encouraging results in three patients, as the company advances its bradykinin B2 receptor antagonist program with both immediate and extended-release formulations.last yearPharvaris Advances Deucrictibant for HAE and AAE, Outlines 2025 Priorities- Pharvaris is progressing with Phase 3 trials for deucrictibant, targeting both on-demand treatment and prophylactic use for hereditary angioedema (HAE).
- Clinical development plans are underway to explore deucrictibant's potential in treating acquired angioedema due to C1-INH deficiency (AAE-C1INH), addressing an unmet medical need.
- The company anticipates topline data from the RAPIDe-3 trial in Q1 2026 and CHAPTER-3 trial in the second half of 2026, with a strong cash position to support these initiatives.
- Recent presentations at medical congresses highlighted positive long-term extension data for deucrictibant, reinforcing its potential as a preferred HAE therapy.last year