A Multicenter Study to Evaluate Long-term Safety and Tolerability of Open Label Sacubitril/Valsartan in Pediatric Patients With Heart Failure Due to Systemic Left Ventricle Systolic Dysfunction Who Have Completed Study CLCZ696B2319
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 216
- 试验地点
- 2
- 主要终点
- Number of Participants With Serious Adverse Events
研究概览
简要总结
The purpose of this study was to evaluate long-term safety and tolerability and to provide post-trial access to sacubitril/valsartan to eligible participants who successfully completed CLCZ696B2319 (PANORAMA-HF) core study Part 2 as per protocol.
详细描述
This was a multicenter, open-label long-term extension study for participants who successfully completed PANORAMA-HF core study Part 2 of the trial or who discontinued study drug treatment early in Part 2 due to the implementation of Urgent safety measure (USM) of PANORAMA-HF core study. Provided that they fulfilled the protocol requirements, these patients were eligible to participate in the OLE. Depending on when and where the participant was enrolled, the duration of the study was of a minimum of 1 year, or until receipt of local marketing authorization and commercial availability, or reaching the maximum limit allowed by local regulations, or until Dec-2023, whichever occurred first.
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
- 1 Year 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed informed consent
- •On study drug at PANORAMA-HF Part 2 EOS visit. Does not have any significant safety issue
排除标准
- •Subject only participated in PANORAMA-HF Part 1 or was a SF in PANORAMA-HF or permanently discontinued study drug in PANORMA-HF Part 2
- •Use of investigational drugs within 5 half-lives of enrollment or within 30 days (longer duration); with the exception of PANORAMA-HF study drug (requires >/=36-hour washout before baseline visit)
- •History of hypersensitivity or allergy to study treatment, its excipients or drugs of similar chemical class, ACEIs, ARBs, or NEP inhibitor and known/suspected contraindications to sacubitril/valsartan
- •Renal vascular hypertension (including renal artery stenosis)
- •Significant renal estimated glomerular filtration rate disorder (eGFR calculated using modified Schwartz formula <30% mean GFR for age); hepatic disorder (serum aspartate aminotransferase or alanine aminotransferase > 3 times upper limit of normal); gastrointestinal disorder or biliary disorder
- •History of angioedema
- •Parents or legal guardians of subject who do not give consent or allow the child to give assent, or inability of patient or parents/legal guardians to follow instructions or comply with follow-up procedures
- •Any medical condition(s) that may put the patient at risk in the investigator's opinion or that the investigator deems unsuitable for the study
- •Other protocol defined inclusion/exclusion criteria may apply
研究组 & 干预措施
sacubitril/valsartan
single arm, open label sacubitril/valsartan
干预措施: sacubitril/valsartan (Drug)
结局指标
主要结局
Number of Participants With Serious Adverse Events
时间窗: to end of study, up to 4.5 years
Number of participants with at least one Serious Adverse Events (SAEs)
Number of Participants With Adverse Events
时间窗: to end of study, up to 4,5 years
Number of participants with at least one Adverse Events (AEs)
Duration of Drug Exposure
时间窗: Up to 4.5 years
Median duration of exposure to sacubitril/valsartan (including temporary interruptions)
次要结局
未报告次要终点