Sickle Cell-Beta0-Thalassemia clinical trials at UCSF
1 in progress, 0 open to new patients
Sorry, not currently recruiting here
Sickle cell anemia is a rare disease occurring in an estimated 66,000 children and adults, often poor and underserved, in the United States. Strokes and silent strokes contribute significantly to illness burden in adults with sickle cell anemia, resulting in impairment, challenges with school and job performance, and premature death. Five NIH studies have identified therapies to prevent overt and silent strokes in children with sickle cell anemia, including monthly blood transfusion therapy (for preventing initial and recurrent strokes) and hydroxyurea (for preventing initial strokes). However, no stroke trials have established therapeutic approaches for adults with sickle cell anemia, despite the observation that at least 99% of children with sickle cell anemia in high-income countries reach adulthood, and approximately 60% of adults will experience one or more strokes (~50% with silent strokes and ~10% with overt strokes). Strokes in adults with sickle cell anemia have simply not been well studied. Therapies applied in children may not be effective for stroke prevention in adults with sickle cell anemia. Identifying subgroups of adults with sickle cell anemia and higher incidence coupled with the contribution of established stroke risk factors in the general population (smoking, diabetes, obesity, renal disease) will provide the prerequisite data required for the first ever phase III clinical trials focused on secondary stroke prevention in adults. In six adult sickle cell disease centers, the investigators will conduct a prospective cohort study to test the primary hypothesis that the incidence of stroke recurrence in adults with silent strokes treated with hydroxyurea will be greater than in those without strokes treated with hydroxyurea. We will test two secondary hypotheses: 1) adults with sickle cell anemia and silent strokes have cognitive deficits when compared to adults with sickle cell anemia without silent strokes; and 2) adults with sickle cell anemia and strokes receiving regular blood transfusion will have a higher incidence of stroke recurrence than adults with sickle cell anemia without strokes. We will recruit at least 72 individuals with sickle cell anemia and history of silent stroke receiving hydroxyurea therapy, at least 72 individuals with sickle cell anemia and no history of stroke, and at least 50 individuals with sickle cell anemia and history of overt stroke receiving transfusion therapy.
Oakland, California and other locations