Myelodysplastic Syndromes
Conditions
Keywords
Myelodysplastic Syndromes, Clofarabine, Low Dose Cytarabine, Poor risk MDS
Brief summary
This is an interventional, multicenter, open label, phase II study designed to evaluate the safety and efficacy of Clofarabine in combination with low dose Cytarabine in untreated patients with poor risk of Myelodisplastic Syndromes.
Interventions
DRUGClofarabine plus low dose Ara-C
Clofarabine 10 mg/m2 by a 1-hr i.v infusion, once daily, from day 1 to 5. Ara-C 10 mg/m2, subcutaneously, twice a day (q12 hrs), from day 1 to 15. Clofarabine will be given 4 hours before the 2nd dose of Ara-C.
Sponsors
Fondazione Italiana Sindromi Mielodisplastiche-ETS
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
55 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* Male and female patients age 55 to 80 years * Written informed consent to participate in the clinical trial * Morphologically confirmed diagnosis of MDS according to WHO classification, and IPSS INT-2 or high risk according to IPSS index * ECOG performance status 0-2 * No previous chemotherapy * Serum bilirubin ≤1.5 times upper limit of normal (ULN) (unless elevation is due primarily to elevated unconjugated hyperbilirubinemia secondary to Gilbert's syndrome or hemolysis, but not to liver dysfunction) * AST and ALT ≤2.5 times ULN * Alkaline phosphatase ≤2.5 times ULN * Serum creatinine ≤ 1 mg/dl: if serum creatinine \> 1.0 mg/dl, then the estimated glomerular filtration rate (GFR) must be \>60 mL/min/1.73 m2 as calculated by the Modification of Diet in Renal Disease equation where Predicted GFR (ml/min/1.73 m2) = 186 x (Serum Creatinine) -1.154 x age in years -0-023 x 0.742 (if patient is female) x 1.212 (if patient is black) * HIV negative
Exclusion criteria
* Have had any other chemotherapy or any investigational therapy as a treatment for MDS. Patients who received chemotherapy for other cancers than MDS/AML can be enrolled, provided that at least 6 months elapsed from accomplishment of the last course of chemo. * Have had a prior hematopoietic stem cell transplant for MDS * Have an uncontrolled systemic fungal, bacterial, viral, or other infection (defined as exhibiting ongoing signs/symptoms related to the infection and without improvement, despite appropriate antibiotics or other treatment). * Have a psychiatric disorder that would interfere with consent, study participation, or follow-up. * Have any other severe concurrent disease, or have a history of serious organ dysfunction or disease involving the heart * Have had any prior treatment with Clofarabine * Have had a diagnosis of another malignancy, unless the patient has been disease-free for at least 3 years following the completion of curative intent therapy with the following exceptions: a.)Patients with treated non-melanoma skin cancer, in situ carcinoma, or cervical intraepithelial neoplasia, regardless of the disease -free duration, are eligible for this study if definitive treatment for the condition has been completed. b.)Patients with organ-confined prostate cancer with no evidence of recurrent or progressive disease based on prostate-specific antigen (PSA) values are also eligible for this study if hormonal therapy has been initiated or a radical prostatectomy has been performed. * Have prior positive test for the Human Immunodeficiency Virus (HIV), HCV, HBV.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To assess the remission rate according to the combination regimen | 19 months |
Secondary
| Measure | Time frame |
|---|---|
| Duration of response and survival | 19 months |
| Time to transformation in AML | 19 months |
| To determine the relationship of cytogenetic abnormalities and response to treatment | 19 months |
| To determine the safety and tolerability of the combination regimen | 19 months |
Countries
Italy
Outcome results
None listed