Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia Intermedia
Status:
Completed
Trial end date:
1969-12-31
Target enrollment:
Participant gender:
Summary
OBJECTIVES: I. Determine whether arginine butyrate with or without epoetin alfa can stimulate
gamma-globin chain production to a degree that decreases anemia and results in hematologic
improvement in patients with thalassemia intermedia.
II. Determine whether a proportional increase in gamma-globin synthesis and mRNA and an
improvement in nonalfa and alfaglobin chain imbalance by at least 10% over baseline correlate
with improved hematologic response in these patients when treated with this regimen.
III. Determine whether a decrease in hemolysis, as assayed by a decrease in LDH, compared to
baseline levels correlates with improved hematologic response in these patients when treated
with this regimen.
IV. Determine whether any particular genotypes are more responsive than others to this
therapy in these patients.
V. Determine whether baseline epoetin alfa levels, gender, and/or baseline reticulocyte
counts (or percent circulating nucleated erythroblasts) correlate with improved hematologic
response in these patients when treated with this regimen.