NCT07727538招募中3 期
An Open-Label Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Pediatric Patients With Familial Chylomicronemia Syndrome (FCS)
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 12
- 试验地点
- 4
- 主要终点
- Percent Change from Baseline in Fasting Triglycerides (TG)
研究概览
简要总结
The primary purpose of the study is to evaluate the efficacy of olezarsen administered by subcutaneous injection to pediatric participants with FCS.
详细描述
This is a Phase 3 multi-center open-label study to evaluate safety, pharmacokinetics (PK), efficacy, and pharmacodynamics (PD) of olezarsen in pediatric participants (aged 2 to less than (<)18 years) with FCS. This study consists of three to four periods with the following approximate timeframes: 1-month screening period, 1-year treatment period, an optional 1-year long-term extension treatment period, and a 3-month post-treatment follow-up period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Parental or legally authorized representative consent must be obtained, and the participants must provide age-appropriate or cognition-appropriate assent, as determined by the Investigator. The parent or legal guardian must be able to understand and comply with the study visit schedule and all other study procedures.
- •Must be able to comply with all study procedures.
- •Age 12 to less than 18 years at the time of informed consent/assent (Cohort 1); age 2 to less than 12 years at time of informed consent/assent (Cohort 2).
- •Willing to fast for at least 10 hours before visits requiring fasted blood sampling.
- •A diagnosis of Familial Chylomicronemia Syndrome (type 1 Hyperlipoproteinemia) by documentation of confirmed homozygote, compound heterozygote or double heterozygote for loss-of-function mutations in type 1-causing genes.
- •Fasting TGs ≥880 mg/dL at screening. If fasting TG is < 880 mg/dL, up to two additional tests may be performed during the screening period with any single test used to qualify.
排除标准
- •Diabetes mellitus with any of the following:
- •Newly diagnosed within 12 weeks prior to screening or during the screening period.
- •Hemoglobin A1c (HbA1c) ≥9.5% at screening.
- •Change in basal insulin regimen >20% within 3 months prior to screening or during the screening period.
- •Type 1 diabetes.
- •History of bleeding, diathesis, or coagulopathy.
- •Major surgery within 3 months of screening.
- •Plasma apheresis within 4 weeks prior to screening or planned during the study.
- •Treatment with another investigational drug, biological agent, or device within one month of screening, or 5 half-lives of investigational agent, whichever is longer.
- •Active pancreatitis within 4 weeks prior to screening or during the screening period.
- •Malignancy diagnosed or treated within 5 years prior to screening or during the screening period.
- •Note: Other protocol-specified inclusion/exclusion criteria may apply.
研究组 & 干预措施
Cohort 1
Experimental
Participants aged 12 to <18 years will receive multiple doses of olezarsen, at a dose level that depends on body weight, once every month by subcutaneous injection for up to 1 year. All participants may continue to the optional 1-year long-term extension period with the last dose received being after 2 years of treatment.
干预措施: Olezarsen (Drug)
Cohort 2
Experimental
Participants aged 2 to <12 years will receive multiple doses of olezarsen, at a dose level based on information obtained from Cohort 1, once every month by subcutaneous injection for up to 1 year. All participants may continue to the optional 1-year long-term extension period with the last dose received being after 2 years of treatment.
干预措施: Olezarsen (Drug)
结局指标
主要结局
Percent Change from Baseline in Fasting Triglycerides (TG)
时间窗: At 6 Months
次要结局
- Number of Participants with Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs Including Independently Adjudicated Events of Pancreatitis, and Withdrawals due to Adverse Events (AEs)(Up to 24 Months)
- Change From Baseline in Vital Sign Parameter - Heart Rate (Beats per Minute)(Baseline up to 24 Months)
- Change From Baseline in Vital Sign Parameter - Blood Pressure (Systolic and Diastolic, mmHg)(Baseline up to 24 Months)
- Change From Baseline in Vital Sign Parameter - Oxygen Saturation (%)(Baseline up to 24 Months)
- Change From Baseline in Vital Sign Parameter - Respiratory Rate (Breaths per Minute)(Baseline up to 24 Months)
- Change From Baseline in Vital Sign Parameter - Body Temperature (°C)(Baseline up to 24 Months)
- Change From Baseline in Body Weight (kg)(Baseline up to 24 Months)
- Change From Baseline in Height (cm)(Baseline up to 24 Months)
- Change From Baseline in Pubertal Development Parameter - Sexual Maturity Rating (Tanner Stage)(Baseline up to 24 Months)
- Change From Baseline in Pubertal Development Parameter - Menarche (Age to the Nearest Month and Year)(Baseline up to 24 Months)
- Change From Baseline in Clinical Laboratory Parameter - Amylase(Baseline up to 24 Months)
- Change From Baseline in Clinical Laboratory Parameter - Lipase(Baseline up to 24 Months)
- Change From Baseline in Clinical Laboratory Parameter - Alanine Aminotransferase (ALA)(Baseline up to 24 Months)
- Change From Baseline in Clinical Laboratory Parameter - Aspartate Aminotransferase (AST)(Baseline up to 24 Months)
- Change From Baseline in Clinical Laboratory Parameter - Platelets(Baseline up to 24 Months)
- Change From Baseline in Electrocardiogram (ECG) Parameter - Heart Rate(Baseline up to 24 Months)
- Change From Baseline in ECG Parameters Including PR Interval, QRS Duration, QT Interval, and QT Corrected Interval Fridericia's (QTcF)(Baseline up to 24 Months)
- Peak, Trough (Pre-Dose), and Post-treatment Plasma Concentration of Olezarsen(Up to 24 Months)
- Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Generic Core Scales(Baseline up to 24 Months)
- Change from Baseline in PedsQL Pediatric Pain Questionnaire (PPQ)(Baseline up to 24 Months)
- Change from Baseline in PedsQL Gastrointestinal Symptoms Module(Baseline up to 24 Months)
- Percent Change from Baseline in Fasting TG(Baseline, 3 Months, 12 Months, 18 Months, 24 Months)
- Proportion of Participants who Achieve Fasting TG <880 milligrams per deciliter (mg/dL)(3 Months, 6 Months, 12 Months, 18 Months, 24 Months)
- Proportion of Participants who Achieve Fasting TG <500 mg/dL(3 Months, 6 Months 12 Months, 18 Months, 24 Months)
- Proportion of Participants who Achieve ≥40 Percent (%) Reduction in Fasting TG from Baseline(3 Months, 6 Months 12 Months, 18 Months, 24 Months)
- Percent Change from Baseline in Apolipoprotein C-III (apoC-III), Very Low-density Lipoprotein Cholesterol (VLDL-C), Non High-density Lipoprotein Cholesterol (non-HDL-C)(Baseline, 3 Months, 6 Months, 12 Months, 18 Months, 24 Months)
- Percent Change from Baseline in High-density Lipoprotein Cholesterol (HDL-C) Low-density Lipoprotein Cholesterol (LDL-C), Apolipoprotein B (apoB), Apolipoprotein B-48 (ApoB-48)(Baseline, 3 Months, 6 Months, 12 Months, 18 Months, 24 Months)
- Event Rate of Adjudicated Acute Pancreatitis(3 Months, 12 Months, 18 Months, 24 Months)
- Percentage of Participants with Abdominal Pain Prior to First Dose vs. TEAEs(3 Months, 12 Months, 18 Months, 24 Months)
- Change From Baseline in Liver Size as Assessed by Magnetic Resonance Imaging (MRI)(Baseline, 12 Months, 24 Months)
- Change From Baseline in Liver Fat Content as Assessed by MRI(Baseline, 12 Months, 24 Months)
- Incidence of Anti-Drug Antibodies (ADA) to Olezarsen(Pre-dose on Days 1, 1 Month, 3 Months, 6 Months, 9 Months, 12 Months, 18 Months and 24 Months; and post-dose on Days 1 and 6 Months)
- Titer of ADA to Olezarsen(Pre-dose on Days 1, 1 Month, 3 Months, 6 Months, 9 Months, 12 Months, 18 Months and 24 Months; and post-dose on Days 1 and 6 Months)
- Incidence of Participants with Negative, Treatment-Unaffected, or Treatment-Emergent ADA to Olezarsen(Pre-dose on Days 1, 1 Month, 3 Months, 6 Months, 9 Months, 12 Months, 18 Months and 24 Months; and post-dose on Days 1 and 6 Months)
- Onset of Treatment-Emergent ADA to Olezarsen(Pre-dose on Days 1, 1 Month, 3 Months, 6 Months, 9 Months, 12 Months, 18 Months and 24 Months; and post-dose on Days 1 and 6 Months)
- Time to Onset of Treatment-Emergent ADA to Olezarsen(Pre-dose on Days 1, 1 Month, 3 Months, 6 Months, 9 Months, 12 Months, 18 Months and 24 Months; and post-dose on Days 1 and 6 Months)
- Proportion of Participants who Achieve Fasting TG <500 mg/dL(3 Months, 6 Months, 12 Months, 18 Months, 24 Months)
- Proportion of Participants who Achieve ≥40 Percent (%) Reduction in Fasting TG from Baseline(3 Months, 6 Months, 12 Months, 18 Months, 24 Months)
- Event Rate of Adjudicated Acute Pancreatitis(3 Months, 6 Months, 12 Months, 18 Months, 24 Months)
- Percentage of Participants with Abdominal Pain Prior to First Dose vs. TEAEs(3 Months, 6 Months, 12 Months, 18 Months, 24 Months)
研究者
研究点 (4)
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