A Study of Oral Clofarabine in Adult Patients With Myelodysplastic Syndromes (MDS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00750334
Recruitment Status : Terminated (Terminated to focus on comparable trial, CLOMDS02507)
First Posted : September 10, 2008
Last Update Posted : May 21, 2015
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Brief Summary:
This study will be used to determine the maximum tolerated dose of oral clofarabine when administered daily for 14 consecutive days repeated every 21 days.

Condition or disease Intervention/treatment Phase
Myelodysplastic Syndromes Drug: clofarabine Phase 1

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 2 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I, Open-Label, Dose-Finding and Food Effect Study of Oral Clofarabine in Previously Treated Adult Patients With Myelodysplastic Syndromes (MDS)
Study Start Date : September 2008
Actual Primary Completion Date : October 2008
Actual Study Completion Date : October 2008

Resource links provided by the National Library of Medicine

Drug Information available for: Clofarabine

Arm Intervention/treatment
Experimental: Part A
clofarabine Dose Escalation
Drug: clofarabine
Drug given Daily x 14 days and 7 days of rest for 21 day cycle

Experimental: Part B
Part B is an open-label, replicated cross-over study in which 12 additional patients will be enrolled and treated at the MTD determined in part A to evaluate the effect of food on the PK disposition of oral clofarabine.
Drug: clofarabine
Drug given Daily X 14 days and 7 days of rest for 21 day cycle

Primary Outcome Measures :
  1. Maximum Tolerated dose levels [ Time Frame: First Cycle ]

Secondary Outcome Measures :
  1. PK profile [ Time Frame: After MTD is established ]
  2. Food effect on profile [ Time Frame: After MTD is established ]
  3. Activity of clofarabine in this dosing regimen [ Time Frame: Duration of Study ]

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Provide signed, written informed consent.
  • Be at least 18 years old.
  • Have a pathologically confirmed MDS and score according to the IPSS at study entry. Pathologic confirmation is the responsibility of the investigator.
  • Have been treated previously for MDS as follow: a.Patients must have had at least 1, but no more than 2, prior treatment regimens; b.Patients must not have refractory (i.e., disease progression or no evidence of response while on treatment) to more than 1 prior treatment regimen.
  • Have Eastern Cooperative Oncology Group (ECOG) performance status of 0-2
  • Be able to comply with study procedures and follow-up examinations.
  • Have adequate hepatic and renal function.
  • Be non-fertile or agree to use birth control during the study through the end of the last treatment visit and at least 90 days after.

Exclusion Criteria:

  • Have had a readjustment of dose and/or schedule of erythropoietin, granulocyte colony stimulating factor(G-CSF) or other growth factors within 8 weeks prior to the first dose of oral clofarabine.
  • Have had any other chemotherapy or any investigational therapy for MDS within 4 weeks of the first dose of oral clofarabine.
  • Have not recovered to ≤ Grade 2 in severity of any drug-related non-hematologic toxicity prior to the first dose of oral clofarabine.
  • Have an uncontrolled systemic fungal, bacterial, viral or other infection. Have a history of serious disease involving the heart.
  • Have a clinically significant cardiac assessment at screening or a known family history QT prolongation.
  • Currently uses a medication known to prolong the QT interval.
  • Have had any prior treatment with clofarabine (IV or oral).
  • Have had a diagnosis of another malignancy, unless the patient has been disease free for at least 3 years after completing curative intent therapy except for the following: 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 or 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).
  • Have gastrointestinal disease or prior surgery, which may affect the ability of the patient to absorb oral clofarabine.
  • Is currently participating in another concurrent investigational protocol that is not restricted to data and/or sample collection for patient demographic and/or disease purposes.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00750334

United States, Florida
Malignant Hematology Administration, H. Lee Moffitt Cancer Center
Tampa, Florida, United States
United States, Minnesota
Division of Hematology Mayo Clinic
Rochester, Minnesota, United States
United States, Texas
Cancer Care Centers of South Texas
San Antonio, Texas, United States
Sponsors and Collaborators
Genzyme, a Sanofi Company
Study Director: Medical Monitor Genzyme, a Sanofi Company

Responsible Party: Genzyme, a Sanofi Company Identifier: NCT00750334     History of Changes
Other Study ID Numbers: CLOMDS01206
First Posted: September 10, 2008    Key Record Dates
Last Update Posted: May 21, 2015
Last Verified: May 2015

Keywords provided by Sanofi ( Genzyme, a Sanofi Company ):
Oral Clofarabine,
relapsed/refractory MDS,

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents