2024-516247-62-00招募中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.20 个研究点 分布在 10 个国家目标入组 37 人开始时间: 2025年2月12日最近更新:
适应症
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 37
- 试验地点
- 20
- 主要终点
- Time-normalized (per 4 weeks) number of Investigator confirmed HAE attacks during the 24 week Treatment Period (Day 1 through Day 168)
研究概览
简要总结
To evaluate the efficacy of deucrictibant 40 mg extended release (XR) tablet compared with placebo for prophylaxis against angioedema attacks
入排标准
- 年龄范围
- 0 years 至 65+ years（65+ Years, 18-64 Years, 0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Provision of written informed consent. At the time of signing informed consent, male and female participants must be aged ≥12 years. If the participant is an adolescent (ie, aged ≥12 to <18 years or as determined by local law), written assent will be obtained from the participant and consent will be obtained from the participant’s parent/legal guardian. A participant who is an adolescent will sign the ICF if they reach the age of 18 years or as determined by local law during their participation in the study
- •Participants who previously completed Study PHA022121-C306 may be eligible to be screened for this study. For these participants, there is no need to repeat HAE diagnostic test as described in Inclusion Criterion #3 if the low C1 esterase inhibitor (C1INH) function was confirmed by the central laboratory in Study PHA022121-C306
- •Diagnosis of HAE based upon all of the following: a. Documented clinical history consistent with HAE (cutaneous or submucosal, nonpruritic swelling without accompanying urticaria) b. At least one of the following: • Age at reported onset of first angioedema symptoms ≤30 years • Family history consistent with HAE • C1q within normal range c. Diagnostic testing results to confirm HAE: • C1INH functional level <50% of the normal level must be shown by chromogenic assay performed by the central laboratory as part of the Screening procedures
- •History of at least 3 HAE attacks within the 3 consecutive months prior to Screening Visit
- •Participants must experience at least XX Investigator-confirmed attacks during the up to 8 week Run-in Period
- •Participant is assessed by the Investigator to have reliable access and ability to use standard of care on-demand treatments to effectively manage acute HAE attacks.
- •Investigator considers that the participant (and parent/caregiver for adolescent participants) is willing and able to adhere to all protocol requirements, including ensuring that the participant is capable of, and compliant with, eDiary and ePRO data recording
- •Female participants of childbearing potential (or who become of childbearing potential during the study) must agree to the protocol specified pregnancy testing and to be abstinent from heterosexual intercourse or to use a highly effective contraception method as defined in the protocol (Section 8.2.2) and as available locally, from enrollment until 30 days after the last study drug administration.
排除标准
- •Any diagnosis of angioedema other than HAE
- •Any clinically significant history of angina, myocardial infarction, syncope, stroke, left ventricular hypertrophy or cardiomyopathy, uncontrolled hypertension, bradycardia, or any other clinically significant cardiovascular abnormality within the previous year that, in the opinion of the Investigator, would interfere with the participant's safety or ability to participate in the study
- •History of epilepsy and/or other significant neurological diseases
- •Any clinically significant and uncontrolled gastrointestinal dysfunction (eg, chronic diarrhea, inflammatory bowel disease) which impacts study drug absorption
- •History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse
- •Use of concomitant medications with systemic absorption and foods that are moderate and strong inhibitors of cytochrome P450 (CYP) 3A4 such as clarithromycin, erythromycin, diltiazem, itraconazole, ketoconazole, ritonavir, verapamil, and grapefruit juice or strong inducers of CYP3A4 such as carbamazepine, phenytoin, and rifampin 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
- •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 was longer)
- •Has received prior prophylactic treatment or on-demand treatment with deucrictibant, with the exception of participants from Study PHA022121-C306
- •Exposure to angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications with systemic absorption (such as oral contraceptives or hormonal replacement therapy) within 4 weeks of Screening
- •Prior gene therapy for any indication at any time
- •Receiving prophylactic treatment for HAE and are satisfied with this treatment. Patients who are not satisfied (eg, tolerability issues, lack of efficacy) and have previously stopped long-term prophylactic HAE treatment can sign the ICF and begin the Screening Period only if their last dose of treatment was received prior to the time point before Screening indicated below: a. Long-term prophylactic therapy for HAE (C1INH, oral kallikrein inhibitors, or anti fibrinolytics): 2 weeks prior to Screening b. Long-term prophylactic therapy for HAE with attenuated androgens: 4 weeks prior to Screening c. Long-term prophylactic monoclonal antibody therapy for HAE (ie, lanadelumab): 5 half-lives prior to Screening d. Short-term prophylaxis for HAE: 7 days prior to Screening Note: Short-term prophylaxis is defined as intravenous (iv) C1INH to avoid angioedema complications from medically indicated procedures
- •Any females who are pregnant, plan to become pregnant, or are currently breast-feeding
- •Abnormal hepatic function (aspartate aminotransferase [AST] >2× upper limit of normal [ULN], alanine aminotransferase [ALT] >2× ULN, or total bilirubin >1.5× ULN or any hepatic impairment via Child-Pugh Scoring System. Participants with Gilbert’s syndrome, defined as isolated increase of total bilirubin ≤3× ULN and AST and ALT within the normal range, are not excluded
- •Moderate or severe renal impairment (estimated glomerular filtration rate [eGFR] <60 mL/min/1.73 m2)
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 (Day 1 through Day 168)
Time-normalized (per 4 weeks) number of Investigator confirmed HAE attacks during the 24 week Treatment Period (Day 1 through Day 168)
次要结局
- Proportion of time without angioedema symptoms during the 24 week Treatment Period
- Time-normalized number of Investigator confirmed HAE attacks treated with on-demand medication during the 24 week Treatment Period
- Time-normalized number of Investigator confirmed moderate or severe HAE attacks during the 24 week Treatment Period
- Time-normalized number of Investigator confirmed severe HAE attacks during the 24 week Treatment Period
- Proportion of participants achieving ≥50% reduction in HAE attack rate relative to baseline during the 24 week Treatment Period
- Proportion of participants achieving ≥70% reduction in HAE attack rate relative to baseline during the 24 week Treatment Period
- Proportion of participants achieving ≥90% reduction in HAE attack rate relative to baseline during the 24 week Treatment Period
- Proportion of participants that are HAE attack-free during the 24 week Treatment Period
- Treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs), adverse events of special interest (AESIs), and TEAEs leading to study drug discontinuation
- Changes in clinical laboratory tests from baseline
- Changes in vital signs from baseline
- Changes in electrocardiogram (ECG) parameters from baseline
- Deucrictibant plasma concentration-time profiles
- Angioedema Quality of Life (AE-QoL) questionnaire
- Patient Global Assessment of Change (PGA-Change)
- Angioedema Control Test 4-week version (AECT-4wk)
- Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI-SHP)
- Abbreviated Treatment Satisfaction Questionnaire for Medication (TSQM-9)
研究者
Clinical Trials Information
Scientific
Pharvaris Netherlands B.V.
研究点 (20)
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