Safety and Tolerability of Oral Clofarabine in Intermediate to High Risk Myelodysplastic Patients

This is an interventional treatment trial for Myelodysplastic Syndrome focused on measuring Myelodysplastic syndrome, Clofarabine, low-dose oral clofarabine, intermediate risk Myelodysplastic syndrome, high risk Myelodysplastic syndrome Read More

Inclusion Criteria:

• Provide signed written informed consent.
• Patients with MDS must have IPSS score that falls in the intermediate or high risk disease (intermediate 1 will have to be transfusion dependent).
• Patients may have received up to two prior therapies for MDS including one hypomethylating agent and/or a biologic agent (biologic agents include GM-CSF or equivalent, danazol or equivalent, Sunitinib, Revlimid, ATG, or a vaccine).
• Age ≥ 18

• Have adequate renal and hepatic functions as indicated by the following laboratory values:

  • Serum creatinine ≤ 1 mg/dL; if serum creatinine >l mg/dL, then the estimated glomerular filtration rate (GFR) must be >50 mL/min/1.73 m2 as calculated by the Modification of Diet in Renal Disease equation.
  • Serum bilirubin ≤1.5 mg/dL x upper limit of normal (ULN)
  • Aspartate transaminase (AST)/alanine transaminase (ALT) ≤2.5 x ULN
  • Alkaline phosphatase ≤2.5 x ULN
• Capable of understanding the investigational nature, potential risks and benefits of the study, and able to provide valid informed consent.
• Female patients of childbearing potential must have a negative serum pregnancy test within 2 weeks prior to enrollment.
• Male and female patients must use an effective contraceptive method during the study and for a minimum of 6 months after study treatment.

Exclusion Criteria:

• Have any other severe concurrent disease, or have a history of serious organ dysfunction or disease involving the heart, kidney, liver, or other organ system that may place the patient at undue risk to undergo treatment.
• Active CNS disease
• Patients with a systemic fungal, bacterial, viral, or other infection not controlled (defined as exhibiting ongoing signs/symptoms related to the infection and without improvement, despite appropriate antibiotics or other treatment).
• Pregnant or lactating patients.
• Any significant concurrent disease, illness, or psychiatric disorder that would compromise patient safety or compliance, interfere with consent, study participation, follow up, or interpretation of study results.
• 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:

  • 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.
  • 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 (HN).
• Have prior positive test for the Human Immunodeficiency Virus (HN).
• Have currently active gastrointestinal disease, or prior surgery that may affect the ability of the patient to absorb oral clofarabine.
• Patients taking proton pump inhibitors such as omeprazole (Prilosec®), lansoprazole (Prevacid®), or esomeprazole (Nexium®). Those who cannot stop taking these drugs should be switched to H2 blockers such as famotidine (Pepcid®)or ranitidine (Zantac®).
• Patients taking alternative medicines (such as herbal or botanical) are not permitted.