Propanolol Effect on Red Cell Adhesion in Non-Asthmatic Children With Sickle Cell Disease: A Dose Finding Study
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- measurement of the sickle red cell response to epinephrine
研究概览
简要总结
Propanolol is a beta blocker which has been found to inhibit the ability of epinephrine to upregulate sickle red cell adhesion to laminin and endothelial cells in vitro. The purpose of this pilot study is to administer one dose of propanolol to children with sickle cell disease and to measure pre and post dose red cell adhesion. The hypothesis is that a single dose of propanolol will decrease red cell adhesion to laminin and endothelial cells as compared to baseline.
详细描述
A similar pilot study has already been conducted in adults and is now being tried in children to gather preliminary data for a grant submission. No safety issues were found in the adult pilot study. This study will evaluate the effect of different doses of propanolol. The risks of this study involve the risks of three (3) blood draws and the risks of propanolol. In order to minimize the risks children with sickle cell disease and asthma will be excluded because asthma is a contraindication to the use of propanolol. In addition, patients will not be hypertensive or bradycardic.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 10 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •diagnosis of HbSS or HbSBeta0Thal
- •age 10-17 years
- •Weight 30kg or greater
- •Hb 7mg/dL or greater
- •informed consent
排除标准
- •History of vaso-occlusive crisis during the past 6 weeks, or history of transfusion during the past 3 months.
- •history of heart failure, myocardial infarction, asthma, bradyarrythmias, hypotension, thyroid disease, diabetes, renal insufficiency
- •concurrent medications: any antihypertensive medication, diuretics, thyroid replacement medications, any arrythmia medication, insulin, hypoglycaemic medication
- •history of allergy to sulfonamides
- •elevated BUN or creatinine
研究组 & 干预措施
cohort 1 10mg propranolol
first study arm will consist of 5 subjects who will be administered a dose of 10mg propanolol and followed 1-4 weeks.
Data safety and monitoring committee has reviewed the data for safety and instructed the study to continue based on the findings.
干预措施: propranolol (Drug)
cohort 2 20mg propranolol
This cohort will involve the administration of 5 subjects with 20mg propanolol. The data safety and monitoring committee will review the data for safety when the 5 subjects are recruited and instruct the study to continue depending on the findings.
干预措施: propranolol (Drug)
cohort 3 40mg propranolol
This cohort will involve the administration of 10 subjects with 40mg propanolol. The data safety and monitoring committee will review the data for safety when the 10 subjects are recruited and instruct the study to continue depending on the findings.
干预措施: propranolol (Drug)
结局指标
主要结局
measurement of the sickle red cell response to epinephrine
时间窗: 1-4 weeks
At study initiation 5mL of blood is drawn to evaluate for red cell adhesion (screening visit or visit 1). At the intervention visit 1-4 weeks later another 5mL of blood will be drawn and patient will have an ECG performed, followed by one dose of propanolol at the stratum that s/he is in. Vitals signs will be monitored (including blood pressure) for 4 hours after drug administration.
次要结局
- Safety data regarding the use of propanolol in children with sickle cell disease(within 24 hours after drug administration)
研究者
Ofelia Alvarez
Professor of Clinical Pediatrics
University of Miami