A Long-term, Open-label Study to Evaluate the Safety and Efficacy of Orally Administered Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema
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
- 170
- 试验地点
- 23
- 主要终点
- Treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs), adverse events of special interest (AESIs), and TEAEs leading to study drug discontinuation
- 状态
- 招募中
- 最后更新
- 上个月
概览
简要总结
This is a Phase 3, multicenter, long-term, open-label study to evaluate the safety and efficacy of once-daily orally administered deucrictibant extended-release tablet 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 (only for participants not rolling over within 28 days from a previous deucrictibant prophylactic study), a Treatment Period in which participants will receive open-label deucrictibant extended-release tablet once daily for approximately 130 weeks, followed by an End of Study visit after maximum 4 weeks. Participants will undergo regular safety (e.g. lab draws) and efficacy assessments, will complete an electronic diary daily, and also complete questionnaires at predefined timepoints during the study.
研究者
入排标准
入选标准
- •Provision of the signed ICF by the participant and/or legally designated representative.
- •Male or female, aged ≥12 years at the time of providing written informed consent/assent.
- •Diagnosis of hereditary angioedema (HAE)
- •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
- •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 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 ICF signature (whichever is longer)
- •Prior gene therapy for any indication at any time
- •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
- •Exposure to ACE inhibitors or any estrogen-containing medications with systemic absorption within 4 weeks of Screening
- •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
Deucrictibant
Deucrictibant
干预措施: Deucrictibant
结局指标
主要结局
Treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs), adverse events of special interest (AESIs), and TEAEs leading to study drug discontinuation
时间窗: 130 weeks
Change in heart rate
时间窗: 130 weeks
Change in blood pressure
时间窗: 130 weeks
Change in body temperature
时间窗: 130 weeks
Change in clinical laboratory tests from baseline
时间窗: 130 weeks
Hematology, blood chemistry, and urinalysis. Descriptive in nature, no formal statistical hypothesis testing will be performed.
Change in electrocardiograms (ECGs) from baseline
时间窗: 130 weeks
Digital triplicate 12-lead ECG. Descriptive in nature, no formal statistical hypothesis testing will be performed.
次要结局
- Patient reported outcome: Work Productivity and Activity Impairment Questionnaire - Specific Health Problem (WPAI-SHP)(130 weeks)
- Patient reported outcome: Abbreviated Treatment Satisfaction Questionnaire for Medication (TSQM-9)(130 weeks)
- Pharmacokinetics [PK]: Pre-dose plasma concentration of deucrictibant and deucrictibant metabolites (Ctrough)(130 weeks)
- Pharmacokinetics [PK]: Urine concentration of deucrictibant and deucrictibant metabolites(130 weeks)
- Time-normalized number of Investigator-confirmed HAE attacks during the Treatment Period(130 weeks)
- Time-normalized number of Investigator-confirmed HAE attacks treated with on-demand medication during the Treatment Period(130 weeks)
- Time-normalized number of Investigator-confirmed moderate or severe HAE attacks during Treatment Period(130 weeks)
- Time-normalized number of Investigator-confirmed severe HAE attacks during the Treatment Period(130 weeks)
- Proportion of time without angioedema symptoms during the Treatment Period(130 weeks)
- Patient reported outcome: Angioedema Quality of Life (AE-QoL) questionnaire(130 weeks)
- Patient reported outcome: Patient Global Assessment of Change (PGA-Change)(130 weeks)
- Patient reported outcome: Angioedema Control Test 4-week version (AECT-4wk)(130 weeks)