Phase 2
Completed N=6
Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia
Source: ClinicalTrials.gov NCT00661726 ↗Enrolled (actual)
6
Serious AEs
60.0%
Results posted
Apr 2014
Primary outcomePrimary: Number of Evaluable Patients With an Increase From Baseline in Hemoglobin (Hb) of ≥1.5 g/dL — 2 participants
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Evaluable Patients With an Increase From Baseline in Hemoglobin (Hb) of ≥1.5 g/dL |
2 | — |
| PRIMARY Change in Total Hemoglobin (Hb) From Baseline to Peak (the Follow-up Time Point With the Highest Value) |
1.16 | <0.01 sig |
| SECONDARY Change in Absolute Fetal Hemoglobin (HbF) From Baseline to Peak (the Follow-up Time Point With the Highest Value) |
0.65 | <0.01 sig |
| SECONDARY Change in Indirect Bilirubin From Baseline to Nadir (the Follow-up Time Point With the Lowest Value) |
-17.36 | 0.045 sig |
| SECONDARY Change in Serum Lactate Dehydrogenase (LDH) From Baseline to Nadir (the Follow-up Time Point With the Lowest Value) |
-116.60 | 0.083 |
| SECONDARY Change in Absolute Reticulocyte Count From Baseline to Nadir (the Follow-up Time Point With the Lowest Value) |
-34.00 | 0.039 sig |
| SECONDARY Change in Erythropoietin Levels From Baseline to Nadir (the Follow-up Time Point With the Lowest Value) |
-43.78 | 0.18 |
| SECONDARY Change in Platelet Count From Baseline to Peak (the Follow-up Time Point With the Highest Value) |
355.0 | <0.01 sig |
| SECONDARY Change in Neutrophil Counts From Baseline to Nadir (the Follow-up Time Point With the Lowest Value) |
-3.15 | 0.069 |
| SECONDARY Change in Red Blood Cell (RBC) Deformability From Baseline to Peak (the Follow-up Time Point With the Highest Value) |
0.43; 0.09 | 0.18 |
| SECONDARY Change in Percentage of Red Blood Cell (RBC) Hb Concentration From Baseline to Peak (the Follow-up Time Point With the Highest Value) |
7.06 | 0.022 sig |
| SECONDARY Change in Percentage of Annexin-positive Cells From Baseline to Nadir (the Follow-up Time Point With the Lowest Value) |
-1.26 | 0.11 |
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:
- 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
Data sourced from ClinicalTrials.gov (NCT00661726). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.