Study of Propranolol as Anti-Adhesive Therapy in Sickle Cell Disease (SCD)

Completed · Phase 2 · Has a placebo group

Conditions studied: Sickle Cell Disease

In brief

An open label, prospective, randomized cross-over phase II study in up to 60 sickle cell patients who are either homozygous for Hb S or have HbSB0 thalassemia. Initially, each patient will be treated for 6 weeks with placebo or a standard dose of propranolol (40 mg) every 12 hrs. This will be followed by a 2-week washout period after which, patients will receive the other treatment modality (placebo or propranolol). We Hypothesize that propranolol administered in vivo on a daily basis for 6 weeks (1) will decrease baseline adhesion to endothelial cells and will substantially abrogate epinephrine-stimulated adhesion to endothelial cells, as measured in vitro; (2) will improve biomarkers of endothelial activation and dysfunction; and (3) can be safely used in patients with SCD. Thus, the use of propranolol in SCD may represent a safe and effective means of anti-adhesive therapy in SCD. Study Objectives: Primary Objective: • To establish the safety and efficacy of long-term therapy with propranolol as an anti-adhesive therapy for SCD. Secondary Objective: • To evaluate changes in soluble markers of endothelial activation and dysfunction. Correlative Science Objective: • To determine whether response to propranolol therapy is associated with polymorphisms in genes encoding the proteins involved in the upregulation of Sickle Red Blood Cell (SS RBC) adhesion by epinephrine.

Key facts

Study ID

NCT01077921

Run by

Laura M. De Castro, MD

People needed

31

Starts

2010-06-01

Expected to finish

2013-12-01

Last updated by the study team

2015-01-22

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Diagnosis by electrophoresis (HEP) of Hemoglobin (Hgb) SS or Hgb Sβ0 thalassemia (all patients followed at our clinic have HEP-confirmed diagnosis on file)
• Age ≥ 18 years
• Blood pressure (BP) Systolic ≥ 95mm Hg and Diastolic ≥ 50mm Hg
• Heart rate (HR) ≥ 70 and ≤ 110 bpm
• Oxygen saturation by pulse oximeter and at room air ≥ 92%
• Hematocrit (Hct) ≥ 20% and Hb > 6.0 g/dL
• Euthyroid status as indicated by normal Thyroid Stimulating Hormone (TSH)
• SS RBCs obtained during screening period demonstrating an adhesion response to epinephrine of 40% over non-stimulated baseline adhesion to endothelial cells
• Capacity to understand and sign informed consent

You may not qualify if…

• History of vaso-occlusive episode during the 6 wks prior to screening
• RBC transfusion during the 3 months prior to study entry
• Ongoing pregnancy
• History of heart failure, myocardial infarct (MI), bradyarrhythmias, conduction defects
• History of asthma or reactive airway disease
• History of thyroid disease
• Diabetes
• Renal insufficiency (BUN >21 mg/dL and/or Creatinine >1.4 mg/dL)
• Use during the screening or study period of any of the following medications: antihypertensives, diuretics, thyroid replacement therapy, anti-arrhythmia medications, bronchodilators, inhaled steroids, insulin, or hypoglycemic medication
• History of allergy to sulfonamides

Where it is running

• Duke University Medical Center — Durham, North Carolina, United States

Full record on ClinicalTrials.gov

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