Open-Label Safety, Pharmacokinetic, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With Hereditary Angioedema Type I or II
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 36
- 试验地点
- 25
- 主要终点
- The proportion of pediatric patients with HAE Types I or II who take any sebetralstat dose, who experience any AE(s) (including fatal AEs) during the study, irrespective of uses of other medications and sebetralstat discontinuations for any reason.
研究概览
简要总结
KVD900-303 is an open-label, multicenter clinical trial in patients aged 2 to 11 years old 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
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female patients 2 to 11 years of age.
- •Confirmed diagnosis of HAE Type I or II.
- •For patients ≥20 kg at screening, patient has had at least 1 documented HAE attack in the last year prior to screening.
- •Caregiver, as assessed by the Investigator, must be able to appropriately store and administer IMP and be able to read, understand, and complete the diary.
- •Investigator believes that the patient and caregiver are willing and able to adhere to all protocol requirements.
- •Parent or Legally Authorized Representative (LAR) provides signed informed consent and patient provides assent (when applicable).
排除标准
- •Any concomitant diagnosis of another form of chronic angioedema, such as acquired C1 inhibitor deficiency, HAE with normal C1-INH, idiopathic angioedema, or angioedema associated with urticaria.
- •A clinically significant history of poor response to bradykinin receptor 2 blocker, C1-INH therapy, or plasma kallikrein inhibitor therapy for the management of HAE, in the opinion of the Investigator.
- •Patient weighs <9.5 kg.
- •Use of angiotensin-converting enzyme inhibitors after the Screening Visit.
- •Any estrogen-containing medications with systemic absorption (such as oral contraceptives including ethinylestradiol or hormonal replacement therapy) within 7 days prior to the Screening Visit.
- •Patients who require sustained use of strong cytochrome P450 3A4 (CYP3A4) inhibitors or inducers or moderate CYP3A4 inducers.
- •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.
- •Known hypersensitivity to sebetralstat or to any of the excipients.
- •Participation in any interventional investigational clinical trial within 4 weeks of the last dosing of investigational drug prior to the Screening Visit.
研究组 & 干预措施
300 mg Dose Group
Patients will take a single 300 mg dose of KVD900.
干预措施: KVD900 300 mg (Drug)
600 mg Dose Group
Patients will take a single 600 mg dose of KVD900.
干预措施: KVD900 600 mg (Drug)
150 mg Dose Group
Patients will take a single 150 mg dose of KVD900.
干预措施: KVD900 150 mg (Drug)
结局指标
主要结局
The proportion of pediatric patients with HAE Types I or II who take any sebetralstat dose, who experience any AE(s) (including fatal AEs) during the study, irrespective of uses of other medications and sebetralstat discontinuations for any reason.
时间窗: Throughout the duration of the trial, up to 1 year.
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
时间窗: From first dose of IMP until the Final/Early Termination (ET) Visit, up to a maximum of 54 weeks.
A TEAE was defined as an adverse event (AE) that met any of the following conditions: (1) began on or after the first dose of IMP, (2) began before the first dose of IMP and increased in severity on or after first dose, (3) was completely missing a start date and the stop date, (4) was completely missing a start date and the stop date was on or after the first dose of IMP. An on-treatment TEAE was defined as any TEAE occurring within 3 days of IMP administration. A treatment-related TEAE was an AE considered related to the IMP by the investigator. A serious AE was defined as any untoward medical occurrence that at any dose resulted in death, was life-threatening, required in-patient hospitalization or prolongation, resulted in significant disability/incapacity, was a congenital anomaly/birth defect, or was an important medical event (based upon medical and scientific judgment).
次要结局
- Caregiver Global Impression of Change (CaGI-C): Time to beginning of symptom relief defined as at least "a little better" (2 time points in a row)(Within 12 hours of the first IMP administration.)
- Caregiver Global Impression of Severity (CaGI-S): Time to first incidence of decrease from baseline (2 time points in a row)(Within 12 hours of the first IMP administration.)
- CaGI-S: Time to HAE attack resolution defined as "none"(Within 24 hours of the first IMP administration)
- Plasma Concentrations of Sebetralstat(At 0.5 hours (±5 min), 2 hours (±15 min), and 4 hours (±15 min) post-dose. PK assessments for sebetralstat 300 mg ED were conducted at the Enrollment Visit, and for 600 mg ED at the Dose Increase Visit (up to 12.6 months after Enrollment Visit).)