A Multicenter, Open-label, Single-arm, Study to Evaluate Safety and Tolerability of Repatha in Patients With Homozygous Familial Hypercholesterolemia (HoFH) in India
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- Amgen
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
研究概览
简要总结
To describe the safety and tolerability of evolocumab in participants with homozygous familial hypercholesterolemia (HoFH) in India. All participants will receive evolocumab over an 8-week period.
详细描述
An open-label, multicentre, phase 4 study to describe the safety and tolerability of evolocumab in 30 Indian participants with HoFH. Subjects who meet the inclusion/exclusion criteria and laboratory assessments at screening will be enrolled and will be required to maintain their current lipid-lowering drug therapy throughout the duration of the trial. Participants will receive evolocumab 420 mg subcutaneous (SC) once monthly (QM) and study visits will occur approximately every 4 weeks. Apheresis participants will receive evolocumab 420 mg SC every 2 weeks to correspond with their apheresis schedule. Final administration of evolocumab (for all participants) will occur at week 8. The end of study (EOS) visit will occur at week 12 for all participants.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 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 80 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female ≥ 12 to ≤ 80 years of age at the time of signing the informed consent
- •Diagnosis of HoFH based on low-density lipoprotein cholesterol (LDL-C), familial history and xanthoma
- •On a low-fat diet and receiving background lipid-lowering therapy stable for 4 weeks prior to screening and during the time frame of the trial
- •Fasting LDL-C at screening > 130 mg/dL (3.4 mmol/L)
- •Fasting triglycerides at screening ≤ 400 mg/dL (4.5 mmol/L)
排除标准
- •Use of mipomersen or lomitapide within 6 months of screening.
- •Known active infection or major hematologic, renal, metabolic, gastrointestinal, hepatic, or endocrine dysfunction
- •Currently receiving treatment in another investigational device or drug study, or less than 30 days since ending treatment on another investigational device or drug study(ies)
- •Female subject is pregnant or breastfeeding or planning to become pregnant or breastfeed
- •Female subjects of childbearing potential unwilling to use an acceptable method of effective contraception
- •Subject has known sensitivity to any of the products to be administered during dosing
- •History or evidence of any other clinically significant disorder, condition or disease
- •Subject has previously received evolocumab or any other proprotein convertase subtilisin/kexin type 9 (PKSK-9)-inhibiting therapy
研究组 & 干预措施
Evolocumab
Evolocumab 420 mg subcutaneous (SC) once monthly (QM) or every 2 weeks (Q2W; for participants on apheresis).
干预措施: evolocumab (Drug)
结局指标
主要结局
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
时间窗: 12 weeks
Includes both serious and non-serious adverse events (AEs). AE: any untoward medical occurrence in a participant. SAE: an AE that meets 1 on the following serious criteria: fatal; life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity; congenital anomaly/birth defect; other medically important serious event. TEAE: any AE starting on or after the first dose of study drug and up to and including 30 days after the end of study drug or the end of study date, whichever is earlier.
次要结局
- Percent Change From Baseline to Week 12 in Apolipoprotein B (ApoB)(baseline, week 12)
- Percent Change From Baseline to Week 12 in Low-Density Lipoprotein Cholesterol (LDL-C)(baseline, week 12)
- Percent Change From Baseline to Week 12 in Lipoprotein(a) (Lp[a])(baseline, week 12)