Status:
COMPLETED
Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia
Lead Sponsor:
Carelon Research
Collaborating Sponsors:
National Heart, Lung, and Blood Institute (NHLBI)
Conditions:
Thalassemia
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief 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...
Detailed Description
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...
Eligibility Criteria
Inclusion
- 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
- 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:
- Alanine aminotransferase (ALT) level greater than 3 times the upper limit of normal in the 8 weeks before study entry
- Serum albumin level less than 3 g/dL in the 8 weeks before study entry
- 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
Key Trial Info
Start Date :
January 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 1 2010
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT00661726
Start Date
January 1 2008
End Date
September 1 2010
Last Update
April 25 2014
Active Locations (3)
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1
Children's Hospital and Research Center at Oakland
Oakland, California, United States, 94609
2
Children's Hospital Philadelphia
Philadelphia, Pennsylvania, United States, 19104
3
University Health Network
Toronto, Canada, M5G 2C4