Losartan for Diffuse Myocardial Fibrosis in Sickle Cell Disease: A Prospective, Phase II Study.
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 14
- 试验地点
- 1
- 主要终点
- Change in extracellular volume fraction (ECV) after 1 year of losartan treatment
研究概览
简要总结
This study is a pilot, phase II, open-label study of the angiotensin II receptor blocker, losartan, in patients with Sickle Cell Disease (SCD) 6 years or older for 12 months. The investigators will enroll 24 patients with SCD over the course of 1 year with a goal to complete all study procedures in 2 years. The short-term goal is to obtain clinical pilot data regarding the safety and efficacy of losartan in stabilizing or decreasing extracellular volume fraction (ECV) after 12 months of therapy.
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
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •6 years old or older
- •Diagnosis of HbSS or Sbeta0-thalassemia
- •Ability to cooperate with and undergo CMR without sedation or anesthesia
- •Ability to cooperate with and undergo echocardiogram without sedation or anesthesia
- •Patients who are on a stable dose of sickle cell disease-modifying therapy: Hydroxyurea, Voxelotor, L-Glutamine, or Crizanlizumab, for 3 months prior to enrollment will be eligible.
排除标准
- •Current chronic transfusion therapy. Patients who received a simple transfusion for an acute event will be eligible 3 months after completion of transfusion
- •SCD genotypes other than specified in inclusion criteria
- •Any contraindication to CMR such as metallic implants
- •Inability to cooperate with CMR or echocardiography imaging
- •Known congenital heart disease
- •Estimated GFR ≤ to 30 mL/min/1.73 m2 by creatinine clearance
- •Pregnant or lactating females or females of child-bearing potential who are unable to use a medically accepted form of contraception throughout the study
- •Treatment with a renin-angiotensin pathway inhibitor during the 2 weeks prior to enrollment
- •Hypersensitivity to angiotensin receptor II blockers
- •Hyperkalemia (K>5.5 mEq/L) on a non-hemolyzed sample despite low-potassium diet
- •Hepatic dysfunction defined as serum ALT > 5x the upper normal limit for age
- •Current lithium therapy
- •Chronic daily use of NSAID
- •HIV infection.
研究组 & 干预措施
Losartan
Participants will receive oral losartan as tablets or oral solution one time daily. The dosing will depend on age and will be based on drug label and dosing used in studies on patients with SCD.
干预措施: Losartan (Drug)
结局指标
主要结局
Change in extracellular volume fraction (ECV) after 1 year of losartan treatment
时间窗: after 1 year of losartan treatment.
Efficacy of losartan in stabilizing or reducing ECV (diffuse myocardial fibrosis) in SCD after one year.
次要结局
- Predicting Myocardial Fibrosis(At baseline and after one year of losartan treaement)
- Change in Diastolic Function(after 1 year of losartan treatment.)
- Change in Exercise Capacity(after 1 year of losartan treatment.)