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Sickle Cell Disease (SCD) is a hereditary anemia that causes the red blood cells to change their shape from a round and doughnut-like shape to a half-moon/crescent, or sickled shape. People who have SCD have a different type of hemoglobin (protein that carries oxygen). This different type of hemoglobin makes the red blood cells change into a crescent shape under certain conditions. Sickle-shaped cells are a problem because they often get stuck in the blood vessels blocking the flow of blood and can cause inflammation and injury to important areas of the body. All babies are born with hemoglobin called fetal hemoglobin (HbF). Soon after birth, HbF production slows down and another hemoglobin called adult hemoglobin (HbA) is made. Clinical studies have shown that increasing the amount of HbF in the blood may prevent sickling of the red blood cells. Vorinostat has been used in the treatment of cancers and in other research studies and information from those suggests that it may help treat SCD by increasing the amount of HbF in the blood. The purpose of this research study is to determine the effectiveness and safety of vorinostat when used to treat SCD.
- Since we are looking for the highest dose of vorinostat that can be administered safely without severe or unmanageable side effects in participants who have SCD, the participants dose may change while they are enrolled in the study.
- Participants will receive an initial dose of vorinostat for the first cycle (or 4 weeks). If they tolerate this dose, they will receive a higher dose for the second cycle. The dose will be increased a 2nd time if they tolerate the dose of the second cycle. Participants will continue on that highest dose for as long as they can tolerate it, or, for a maximum of 4 cycles. The maximum dose participants could receive is 400mg a day, 3 times a week.
- Participants will be given a study drug diary for each study cycle to record each time they take vorinostat and indicate if they have any changes in their health or in medications they are taking.
- During the study participants will come to the clinic for visits with the study team and may have some of the following tests: physical examination, EKG, blood tests, urine tests and echocardiogram.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Sickle Cell Disease
Brigham and Women's Hospital
Dana-Farber Cancer Institute
Published on BioPortfolio: 2014-08-27T03:18:28-0400
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One of the sickle cell disorders characterized by the presence of both hemoglobin S and hemoglobin C. It is similar to, but less severe than sickle cell anemia.
An abnormal hemoglobin resulting from the substitution of valine for glutamic acid at position 6 of the beta chain of the globin moiety. The heterozygous state results in sickle cell trait, the homozygous in sickle cell anemia.
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A disease characterized by chronic hemolytic anemia, episodic painful crises, and pathologic involvement of many organs. It is the clinical expression of homozygosity for hemoglobin S.
The condition of being heterozygous for hemoglobin S.
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