Study Description: The purpose of this study is to test the safety and efficacy of escalating doses of panobinostat in sickle cell disease. Panobinostat is an experimental drug made by Novartis Pharmaceuticals Corporation. Panobinostat is not available ?on the market? but is only available in clinical trials like this one. Studies have shown that increasing the amount of fetal hemoglobin (HbF) in the blood may prevent sickling of the red blood cells and reduce complications of sickle cell disease. Studies in the lab and in patients with other conditions, such as cancer, have shown that panobinostat increases HbF levels and has an anti-inflammatory effect in human blood. This is the first study in humans to look at how the drug works in individuals with sickle cell disease. This study will include qualified adult patients with sickle cell disease who have failed to respond to hydroxyurea therapy (clinically or hematologically) or are intolerant of hydroxyurea therapy or refuse hydroxyurea therapy. The treatment phase of this study is twelve weeks in duration, with a total time of patient participation at approximately five months. During the treatment phase, participants will take panobinostat by mouth three times per week according to a specified dosing amount and schedule. Patients will have regularly scheduled safety assessments, including physical exam, vital signs (heart rate, temperature, breathing rate, blood pressure, and weight), blood and urine tests, and ECG.
Principal Investigator: Abdullah Kutlar
Eligibility Criteria: This study is open to adults (ages 18 years and above) who carry a diagnosis of sickle cell anemia (SS) or S-B0 Thalassemia, and who are not taking hydroxyurea either due to intolerance, refusal, or failure to respond. Participants must have clinically significant sickle cell disease, as defined by either: 2 sickle-related hospitalizations in the past year; 3 or more pain crises in the past year; recurrent leg ulcers; history of Acute Chest Syndrome within the past 5 years; history of priapism; or history of stroke.
Compensation: Yes, subjects will be compensated.
Compensation Explanation: Patients will receive $50 per visit for participating in the study, for a total of no more than $550. No payment will be provided for missed visits. Additional reimbursement for mileage will be considered for subjects living greater than 50 miles from the clinic.
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