Phase II Study of Azacitidine and Phenylbutyrate in Patients With Thalassemia Major
I. Determine the safety and efficacy of azacitidine and phenylbutyrate in treatment of patients with thalassemia major.
|Study Design:||Primary Purpose: Treatment|
|Study Start Date:||November 2000|
PROTOCOL OUTLINE: Patients receive azacitidine IV continuously on days 1-4 and oral phenylbutyrate three times a day on days 14-42. Bone marrow needle aspiration is performed on days 6, 14, and 42 to assess disease response to treatment. If no response on day 42, a second course of azacitidine and phenylbutyrate begins 7 days later.
Patients are followed weekly for 3 months and then monthly thereafter.
|United States, Maryland|
|Clinical Hematology Branch|
|Bethesda, Maryland, United States, 20892|
|Study Chair:||Griffin Platt Rodgers||National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)|