Phase 2
N=6
Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia
Thalassemia
Bottom Line
View on ClinicalTrials.gov: NCT00661726 ↗Enrolled (actual)
6
Serious AEs
60.0%
Results posted
Apr 2014
Primary outcome: Primary: Number of Evaluable Patients With an Increase From Baseline in Hemoglobin (Hb) of ≥1.5 g/dL — 2 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Decitabine (USAN, INN) (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Carelon Research
- Primary completion
- May 2010
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 |
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:
- 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.