Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia

• Status: Completed
• Trial end date: 2010-09-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: HealthCore-NERI; New England Research Institutes
• Collaborator: National Heart, Lung, and Blood Institute (NHLBI)
• Treatments: Azacitidine; Decitabine

Criteria

Inclusion Criteria:

• Beta-thalassemia and beta thalassemia-hemoglobin E (HbE), as confirmed by DNA testing

• Transfusion independent for at least 120 days before study entry

• Red blood cell folate levels above the lower limit of normal

Exclusion Criteria:

• Absolute neutrophil count (ANC) less than 2000/mm3 in the 8 weeks before study entry or a history of chronic neutropenia, defined as an ANC less than 2000/mm3

• Platelet count less than 100,000/mm3 or greater than 1,000,000/mm3 in the 8 weeks before study entry

• Family history of an inherited disease resulting in low ANC or bone marrow failure

• Serum creatinine level greater than 2 mg/dL in the 8 weeks before study entry

• Evidence of liver disease, as defined by one or more of the following conditions:

1. Alanine aminotransferase (ALT) level greater than 3 times the upper limit of normal in the 8 weeks before study entry

2. Serum albumin level less than 3 g/dL in the 8 weeks before study entry

3. Evidence of cirrhosis on liver biopsy obtained in the 6 months before study entry

• Approaching death; has concurrent liver, kidney, cardiac, or metabolic disease; or has any disease of such severity that death within 7 to 10 days of study entry is likely

• Pregnant, planning to become pregnant, or breastfeeding

• Sexually active female of childbearing potential who is unwilling to use at least two acceptable methods of contraception, as determined by the investigator

• Sexually active male whose partner is of child-bearing potential and who is unwilling to use at least two acceptable methods of contraception, as determined by the investigator, during and for 2 months after decitabine treatment

• Diagnosed with cancer (except non-melanoma skin cancer) in the 5 years before study entry. In particular, suspicion or evidence of myelodysplastic syndrome (MDS) on clinically indicated bone marrow aspirate or a family history of MDS or concurrent leukemia

• HIV infection

• Not expected to be able to complete 24 weeks of study follow-up

• Currently being treated with any experimental or fetal hemoglobin modulating agent

• Current participation in any other studies of investigational drugs or devices

• Unable to comply with study medication regimen

• Any condition, which in the opinion of the investigator, would place the individual at undue risk if treated with twice-weekly low-dose decitabine for 12 weeks