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

University of Miami

Status and phase: Terminated; Phase 1

Conditions: Sickle Cell Disease

Treatments: Drug: propranolol

Study type: Interventional

Funder types: Other

Identifiers

NCT02012777

20100334

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 9 patients

Sex: All

Ages: 10 to 17 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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9 participants in 3 patient groups

cohort 1 10mg propranolol

Active Comparator group

Description:

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.

Treatment:

Drug: propranolol

cohort 2 20mg propranolol

Active Comparator group

Description:

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.

Treatment:

Drug: propranolol

cohort 3 40mg propranolol

Active Comparator group

Description:

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.

Treatment:

Drug: propranolol