Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia
Thalassemia intermedia (TI) is an inherited blood disorder that can cause anemia due to low levels of hemoglobin. Decitabine is a medication that may be effective at increasing hemoglobin levels. This study will evaluate the safety and effectiveness of decitabine at increasing hemoglobin levels in people with TI.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase IIA Study of Subcutaneous 5-aza-2'- Deoxycytidine (Decitabine) in Patients With Thalassemia Intermedia|
- Short-term safety and efficacy of low-dose subcutaneous decitabine for the induction of fetal hemoglobin and resulting increase in total hemoglobin in people with TI [ Time Frame: Measured at Week 24 and at the time of study completion ] [ Designated as safety issue: Yes ]
- Hemoglobin response, changes in hemolysis and erythropoiesis, changes in red blood cell characteristics, adverse and serious adverse event rates, proportion of participants stopping therapy due to adverse events, and genotoxicity [ Time Frame: Measured at Week 24 and at the time of study completion ] [ Designated as safety issue: Yes ]
|Study Start Date:||January 2008|
|Study Completion Date:||September 2010|
|Primary Completion Date:||May 2010 (Final data collection date for primary outcome measure)|
Participants will receive injected decitabine for 12 weeks.
Drug: Decitabine (USAN, INN)
Participants will receive 0.2 mg/kg of decitabine subcutaneously twice a week for 12 weeks. The dose will be reduced for toxicities as needed. The maximum dose of decitabine to be given will be 0.2 mg/kg.
Thalassemias are inherited blood disorders that are characterized by low levels of hemoglobin and healthy red blood cells, which can lead to anemia. There are many different types of thalassemias, and TI is one type. People with TI often have moderate to severe anemia and may have a shortened life span, organ damage, and a lower quality of life as a result of the disease. Decitabine is a medication used to treat people with diseases that affect bone marrow and blood cells. The medication may be an effective treatment for people with TI because it may have the ability to interact with a person's DNA and increase hemoglobin levels. Previous studies in people with anemia have shown that decitabine has increased hemoglobin levels in some participants. The purpose of this study is to evaluate the safety and effectiveness of decitabine at increasing hemoglobin levels in people with TI.
This study will enroll people with TI. Following an 8-week screening period, participants will attend a baseline study visit, which will include a blood collection, pregnancy test, physical exam, and echocardiogram heart imaging procedure. Decitabine will be injected under the skin in the abdomen, thigh, or upper arm. Participants will be observed for a minimum of 30 minutes after the injection to assess pain or adverse reactions. Participants will then receive low doses of decitabine twice a week, on consecutive days, for 12 weeks. They will be closely monitored and dosages will be adjusted or stopped as needed. Every 2 weeks, participants will undergo a blood collection for safety testing. Every 4 weeks, participants will attend a study visit for a pregnancy test, physical exam, blood collection, and review of medication effects. Additionally, at the Week 12 visit, a repeat echocardiogram will occur. During Weeks 12 to 24, participants will not receive decitabine injections but will attend monthly study visits for repeat testing. Study researchers will contact participants by phone every 3 months during Year 1 and then every 6 months for the duration of the study to collect long-term survival and medical information.
|United States, California|
|Children's Hospital and Research Center at Oakland|
|Oakland, California, United States, 94609|
|United States, Pennsylvania|
|Children's Hospital Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104|
|University Health Network|
|Toronto, Canada, M5G 2C4|
|Study Chair:||Nancy Olivieri, MD||University Health Network/Toronto General Hospital|