Efficacy of Vorinostat to Induce Fetal Hemoglobin in Sickle Cell Disease
Status:
Terminated
Trial end date:
2014-10-01
Target enrollment:
Participant gender:
Summary
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.
Phase:
Phase 2
Details
Lead Sponsor:
Dana-Farber Cancer Institute
Collaborators:
Boston Children's Hospital Boston Children’s Hospital Brigham and Women's Hospital Merck Sharp & Dohme Corp.