NCT05505916已完成3 期
An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900, an Oral Plasma Kallikrein Inhibitor, for On-demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema Type I or II
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 145
- 试验地点
- 71
- 主要终点
- Frequencies and percentages of patients with AEs, AEs within 2 days of IMP administration, serious AE's and AEs causing premature discontinuation.
研究概览
简要总结
This is an open-label, multicenter extension trial to evaluate the long-term safety of KVD900 in patients who are 12 years or older with HAE type I or II.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Patients may roll over from KVD900-
- •Inclusion Criteria:
- •Confirmed diagnosis of HAE type I or II at any time in the medical history
- •Patient has had at least 2 documented HAE attacks within 3 months prior to the Enrollment Visit.
- •If a patient is receiving long-term prophylactic treatment with one of the protocol-allowed therapies, they must have been on a stable dose and regimen for at least 3 months prior to the Enrollment Visit (except for danazol, which requires a stable dose and regimen for at least 6 months prior to the Enrollment Visit).
- •Male or female patients 12 years of age and older.
- •Patients must meet the contraception requirements.
- •Patients must be able to swallow trial tablets whole.
- •Patients, as assessed by the Investigator, must be able to appropriately receive and store IMP, and be able to read, understand, and complete the eDiary.
- •Investigator believes that the patient is willing and able to adhere to all protocol requirements.
- •Patient provides signed informed consent or assent (when applicable). A parent or legally authorized representative (LAR) must also provide signed informed consent when required.
排除标准
- •Discontinued from the KVD900-301 trial for reasons of noncompliance, withdrawal of consent, or safety.
- •Presence of any safety concerns that would preclude participation in the open-label trial as determined by the investigator.
- •Any concomitant diagnosis of another form of chronic angioedema, such as acquired C1 inhibitor deficiency, HAE with normal C1-INH (previously known as HAE type III), idiopathic angioedema, or angioedema associated with urticaria.
- •A clinically significant history of poor response to bradykinin receptor 2 (BR2) blocker, C1-INH therapy, or plasma kallikrein inhibitor therapy for the management of HAE, in the opinion of the Investigator.
- •Use of attenuated androgens other than danazol (e.g., stanozolol, oxandrolone, methyltestosterone, testosterone), or anti-fibrinolytics (e.g., tranexamicacid) within 28 days prior to the Enrollment Visit.
- •Use of Angiotensin-converting enzyme (ACE) inhibitors within 7 days prior to the Enrollment Visit.
- •Any estrogen-containing medications with systemic absorption (such as oral contraceptives including ethinylestradiol or hormonal replacement therapy) within 7 days prior to the Enrollment Visit.
- •Inadequate organ function, including but not limited to:
- •Alanine aminotransferase (ALT) >2x Upper Limit Normal (ULN)
- •Aspartate aminotransferase (AST) >2x ULN
- •Bilirubin direct >1.25x ULN
- •International Normalized Ratio (INR) >1.2
- •Clinically significant hepatic impairment defined as a Child-Pugh B or C
- •Any clinically significant comorbidity or systemic dysfunction, which in the opinion of the Investigator, would jeopardize the safety of the patient by participating in the trial.
- •History of substance abuse or dependence that would interfere with the completion of the trial, as determined by the Investigator.
- •Known hypersensitivity to KVD900 or to any of the excipients.
- •Participation in any gene therapy treatment or trial for HAE.
- •Participation in any interventional investigational clinical trial, including an investigational COVID-19 vaccine trial, within 4 weeks of the last dosing of investigational drug prior to the Enrollment Visit.
- •Any pregnant or breastfeeding patient.
研究组 & 干预措施
KVD900 600 mg
Experimental
干预措施: KVD900 600 mg (Drug)
KVD900 300 mg
Experimental
干预措施: KVD900 300 mg (Drug)
结局指标
主要结局
Frequencies and percentages of patients with AEs, AEs within 2 days of IMP administration, serious AE's and AEs causing premature discontinuation.
时间窗: AEs will be recorded from the first dose of IMP in the KVD900-302 trial up to and including the end of study (EOS) visit, a maximum of 2 years for each patient.
Number and percentage of patients with normal or abnormal laboratory results at each scheduled visit.
时间窗: Throughout the duration of the trial.
Number and percentage of patients with normal or abnormal vital sign results at each scheduled visit
时间窗: Throughout the duration of the trial.
次要结局
- Patient Global Impression of Change (PGI-C).(within 12 hours of initial dose of IMP administration.)
- Patient Global Impression of Severity (PGI-S): time to first incidence of 2 time points in a row decrease from baseline(within 12 hours of initial dose of IMP administration.)
- PGI-S: time to HAE attack resolution(within 24 hours of initial dose of IMP administration.)
研究者
研究点 (71)
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