Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia
Condition: Thalassemia
MeSH terms: Thalassemia; beta-Thalassemia
Intervention: Decitabine (USAN, INN)
Intervention description: 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.
Intervention MeSH terms: Decitabine; Azacitidine
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 6.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Thalassemia Intermedia

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.
    

Eligibility 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