Sickle Cell Disease | Propanolol and Red Cell Adhesion Non-asthmatic Children Sickle Cell Disease
Sickle Cell Disease research study
What is the primary objective of this study?
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.
Who is eligible to participate?
Inclusion Criteria: 1. diagnosis of HbSS or HbSBeta0Thal 2. age 10-17 years 3. Weight 30kg or greater 4. Hb 7mg/dL or greater 5. informed consent Exclusion Criteria: 1. History of vaso-occlusive crisis during the past 6 weeks, or history of transfusion during the past 3 months. 2. pregnancy 3. history of heart failure, myocardial infarction, asthma, bradyarrythmias, hypotension, thyroid disease, diabetes, renal insufficiency 4. concurrent medications: any antihypertensive medication, diuretics, thyroid replacement medications, any arrythmia medication, insulin, hypoglycaemic medication 5. history of allergy to sulfonamides 6. elevated BUN or creatinine
Which medical condition, disease, disorder, syndrome, illness, or injury is researched?
Sickle Cell Disease
Interventions can include giving participants drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available or noninvasive approaches such as surveys, education, and interviews.
Drug:propranololPropranolol hydrochloride is a synthetic beta-adrenergic receptor-blocking agent. This will be administered in an open-label single administration to 3 cohorts (10mg, 20mg, and 40mg) of children with sickle cell disease. Patient blood will be evaluated for red cell adhesion and patient data evaluated for safety monitoring.
Research studies and clinical trials typically have two or more research arms. An arm is a group of people who receive the same treatment in the study.
cohort 1 10mg propranololfirst 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.
cohort 2 20mg propranololThis 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.
cohort 3 40mg propranololThis 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.
Start Date: June 2010
Completed Date: April 3, 2013
Phase: Phase 1
Primary Outcome: measurement of the sickle red cell response to epinephrine
Secondary Outcome: Safety data regarding the use of propanolol in children with sickle cell disease
Study sponsors, principal investigator, and references
Principal Investigator: Ofelia A Alvarez, MD
Lead Sponsor: University of Miami
Collaborator: Duke University
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