Dapagliflozin in pediatric heart failure with reduced ejection fraction (Dapa-Ped HF) - A randomized pilot study
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 100
- 试验地点
- 1
- 主要终点
- Composite of
研究概览
简要总结
Dapaglifozin is shown to reduce mortality in adults with all forms ofheart failure. There are case reports and a case series showing safety andbenefit of Dapagliflozin in children with heart failure. There are nowell-structured studies on the use of dapagliflozin in children with heartfailure. The efficacy of the drug needs to evaluated in children as it has beenfound to effective in adults with HFrEF in reducing heart failurehospitalization and cardiovascular mortality. The drug has been studied in childrenwith proteinuric CKD and diabetes and has not shown any significant safetyconcern. Hence, in this pilot study, we plan to evaluate the efficacy andsafety of dapagliflozin in children with heart failure with reduced ejectionfraction. Our objectives are to study the effect of adding Dapagliflozin to standard heart failure therapy in children aged 1 to 12 years and the primary outcome measure is the composite of worsening heart failure, heart failure hospitalizations and cardiovascular death. The study duration is planned for 18 months with enrollment for 12 months and each patient being followed up for 6 months from enrollment. A written informed consent will be obtained from the parent(s) or legalguardian(s) before any study specific procedures are performed. Patient will begiven a unique ID on enrolment and eligibility will be checked using the aboveinclusion and exclusion criteria. Randomization will be performed with computergenerated block randomization with unequal block sizes with opaque sealedenvelopes assigning them to one of the groups. The first investigator will beblinded regarding which group the patient belong. Dispensing of drugs andoptimization of medical and non-pharmacological treatment of heart failure willbe done by a second investigator to avoid bias. Treatment group will beprovided age-appropriate dose of Dapagliflozin along with standard heartfailure therapy. Control group will be given standard of therapy.
研究设计
- 研究类型
- Interventional
- 分配方式
- Stratified block randomization
- 盲法
- Investigator Blinded
入排标准
- 年龄范围
- 1.00 Year(s) 至 12.00 Year(s)（—）
- 性别
- All
入选标准
- •1.Age 1 year to <12 years 2.Chronic heart failure resulting from dilated cardiomyopathy and ventricular dysfunction receiving standard of care heart failure therapy 3.NYHA class II-IV (children 6 years to <12 years) or Ross HF classification II-IV (Children <6 years) any time prior to enrolment 4.LV ejection fraction <40% by echocardiogram 5.Etiologies of heart failure include: Idiopathic cardiomyopathy or history of myocarditis.
- •6.eGFR ≥30 ml/min/1.73 m2 (Shwartz formula) at enrolment.
排除标准
- •1.Receiving therapy with an SGLT2 inhibitor within 8 weeks prior to enrolment or previous intolerance of an SGLT2 inhibitor 2.Patient with type I diabetes mellitus 3.Patients with hemodynamically significant structural heart disease operated or unoperated 4.Patients with sustained or symptomatic dysrhythmias not controlled with drug or device therapy 5.HF due to restrictive cardiomyopathy, acute myocarditis (within 3 months of onset), constrictive pericarditis, hypertrophic (obstructive) cardiomyopathy, Familial or genetic cardiomyopathy, neuromuscular disease or uncorrected primary valvular disease 6.Symptomatic hypotension or BP below 5th percentile systolic BP for age at enrolment 7.Current acute decompensated HF or hospitalization due to decompensated HF <4 weeks prior to enrolment 8.Hepatic impairment aspartate transaminase [AST] or alanine transaminase [ALT] >3x the upper limit of normal [ULN]; or total bilirubin >2x ULN at time of enrolment 9.Severe (eGFR <30 mL/min/1.73 m2 by Schwartz formula), unstable or rapidly progressing renal disease at the time of enrolment.
结局指标
主要结局
Composite of
时间窗: 6 months
1.First worsening heart failure; defined by signs and symptoms of worsening heart failure that requires intensification of HF therapy.
时间窗: 6 months
2.First hospitalization for HF
时间窗: 6 months
3.CV death
时间窗: 6 months
次要结局
- BNP(6 months)
- Change in NYHA/ROSS HF class from enrolment to 6 months of therapy(6 months)
- To study the incidence of adverse events(•Serious adverse events)