Propanolol and Red Cell Adhesion Non-asthmatic Children Sickle Cell Disease

Phase 1TerminatedNCT02012777

University of Miami9 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Sickle Cell Disease

Interventions

propranololDRUG

Propranolol 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.

Eligibility

Sex: ALLMin age: 10 YearsMax age: 17 Years

Medical Language ↔ Plain English

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

Locations (1)

University of Miami

Miami, Florida, United States

Outcomes

Primary Outcomes

measurement of the sickle red cell response to epinephrine

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.

Time frame: 1-4 weeks

Secondary Outcomes

Safety data regarding the use of propanolol in children with sickle cell disease

1. New onset wheezing 2. Onset of low blood pressure defined as blood pressure less than 90/50 3. Onset of heart rate less than 60 beats/min

Time frame: within 24 hours after drug administration

Data from ClinicalTrials.gov

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