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

• Status: Terminated
• Trial end date: 2013-10-01
• Target enrollment: 0
• Participant gender: All

Summary

This is a Phase I trial for patients with intermediate or high risk myelodysplastic syndrome (MDS).

The study agent, clofarabine, is produced by Genzyme Pharmaceuticals.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Roswell Park Cancer Institute
• Collaborator: Genzyme, a Sanofi Company
• Treatments: Clofarabine

Criteria

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.