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Oral Clofarabine Study in Patients With Myelodysplastic Syndrome

Primary Purpose

Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Clofarabine
Sponsored by
M.D. Anderson Cancer Center
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Myelodysplastic Syndrome focused on measuring Myelodysplastic Syndrome, MDS, Chronic Myelomonocytic Leukemia, CMML, Clofarabine, Clofarex, Clolar, Chemotherapy

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Patients with MDS and >/= 5% blasts or IPSS risk intermediate or high; patients with Chronic myelomonocytic leukemia (CMML). No prior intensive chemotherapy or high-dose ara-C (>/= 1g/m2). Prior biologic therapies, targeted therapies, or single agent chemotherapy allowed. Patients must have been off chemotherapy for 2 weeks prior to entering this study and recovered from the toxic effects of that therapy, unless there is evidence of rapidly progressive disease. Hydroxyurea is permitted for control of counts prior to treatment. Procrit, GCSF are allowed before therapy. Performance 0-2 (ECOG). Adequate organ function including the following:Adequate liver function (bilirubin of < 2mg/dl), and renal function (creatinine < 2mg/dl), and SGPT (ALT) < 3 X ULN. Adequate cardiac functions (NYHA cardiac III-IV excluded). Signed informed consent. Exclusion Criteria: Nursing and pregnant females. Patients of childbearing potential should practice effective methods of contraception. Child bearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately. Active and uncontrolled infections. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, or psychiatric illness/social situations that would limit compliance with study requirements. Prior clofarabine treatment.

Sites / Locations

  • U.T.M.D. Anderson Cancer Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Oral Clofarabine

Arm Description

10 mg (Group 1) or 20 mg (Group 2) tablets once a day for 5 days in a row and repeated every 4-8 week cycle.

Outcomes

Primary Outcome Measures

Participants With a Complete Remission (CR)
Complete Remission (CR): Normalization of the peripheral blood and bone marrow with <5% bone marrow blasts, a peripheral blood granulocyte count > (1.0 x 109/ L, and a platelet count > 100 x 109/L). Partial Remission: as above except for the presence of 6-15% marrow blasts, or 50% reduction if <15% at start of treatment. Hematologic Improvement: meets all criteria for CR except for platelet recovery to >100 x 109/L. Clinical Benefit: Platelets increase by 50% and to above 30 x 109/L untransfused (if lower than that pretherapy); or granulocytes increase by 100% and to above 109/L (if lower than that pretherapy); or hemoglobin increase by 2 g/dl; or transfusion independent; or splenomegaly reduction by > 50%; or monocytosis reduction by > 50% if pretreatment > 5 x 109/L.

Secondary Outcome Measures

Full Information

First Posted
March 3, 2006
Last Updated
October 8, 2015
Sponsor
M.D. Anderson Cancer Center
Collaborators
Genzyme, a Sanofi Company
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1. Study Identification

Unique Protocol Identification Number
NCT00299156
Brief Title
Oral Clofarabine Study in Patients With Myelodysplastic Syndrome
Official Title
Phase II Study of Oral Clofarabine in Myelodysplastic Syndrome (MDS)
Study Type
Interventional

2. Study Status

Record Verification Date
August 2015
Overall Recruitment Status
Completed
Study Start Date
March 2006 (undefined)
Primary Completion Date
December 2012 (Actual)
Study Completion Date
December 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
M.D. Anderson Cancer Center
Collaborators
Genzyme, a Sanofi Company

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
The goal of this clinical research study is to learn if clofarabine given by mouth on a weekly schedule can help to control MDS. The safety of clofarabine given by mouth will also be studied.
Detailed Description
Clofarabine is a new chemotherapy drug that is designed to interfere with the growth and development of cancer cells. If you are found to be eligible, you will be randomly assigned (as in the roll of dice) to one of 2 treatment groups. Participants in Group 1 will take a lower dose of clofarabine than Participants in Group 2. You have an equal chance of being assigned to either of the 2 treatment groups. Neither you nor your study doctor can choose your assignment. Later (after the first 40 patients), the assignment will favor the better treatment arm until one arm is selected as the significantly better one, or until 80 patients are treated in total. When you have been assigned to a treatment group, you will receive clofarabine as tablets once a day for 5 days in a row. This will be repeated every 4-8 weeks. Each 4-8 week period is considered 1 cycle of treatment. Original patients who are still on study (original patients who received or are still receiving 30 mg/m2) have already been reduced to lower doses on subsequent courses or had treatment discontinued/completed. These patients will be asked to sign a new informed consent document to be made aware of the new developments in this study. Each dose of clofarabine should be taken with 4 ounces of water in the morning, on an empty stomach. You must not eat or drink anything besides water from midnight the night before you take the study drug until 1 hour after taking the morning dose. You should take clofarabine every morning at about the same time. If vomiting occurs within 15 minutes of taking clofarabine, the dose may be repeated. If vomiting occurs more than 15 minutes after taking clofarabine, the dose cannot be replaced or made up. Coffee and other caffeinated liquids cannot be taken before dosing and for 1 hour after dosing. All clofarabine doses should be taken either in the outpatient or inpatient setting by qualified and trained site personnel or given with appropriate instructions to you and/or your care provider to take at home. At the start of each cycle, you will receive enough clofarabine for 1 cycle of therapy. You should store the clofarabine in its original container at room temperature. You should keep the container closed when not in use, and out of the reach of children. After each cycle of therapy, you will not receive the next cycle of chemotherapy until your blood counts have recovered and any possible side effects have gone away (for around 4 to 8 weeks). You must stay in Houston for the first treatment cycle (about 4 to 8 weeks) and will be required to return to Houston before receiving each additional cycle of chemotherapy (up to 6 days each cycle). Before every treatment cycle, your doctor will perform a physical exam, including measurement of your weight and vital signs (blood pressure, heart rate, temperature, and breathing rate). You will be asked about the level of your daily activities and how you are feeling. You will have blood samples (about 1-2 teaspoons) collected for routine lab tests 1-2 times a week for the first cycle, then every 2-4 weeks while on therapy. Repeat bone marrow samples will be collected every 1-3 cycles. However, if you complete the study before the third cycle, the bone marrow may be taken then. You may choose to have check-up visits and blood tests with your local doctor. If you show a response and do not experience any severe side effects, you can receive up to a total of 12 cycles of therapy. During each cycle, clofarabine will be given the same way as during the first cycle. However, the dose of clofarabine may be lowered during later cycles to decrease the risk of side effects that may have occurred in previous cycles. If the disease gets worse or you experience any intolerable side effects, you will be taken off the study and your doctor will discuss other treatment options with you. This is an investigational study. Clofarabine given by vein is approved by the FDA for treatment of pediatric acute lymphoblastic leukemia. Its use in this study is experimental. Up to 80 patients will take part in this study. All will be enrolled at MD Anderson.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia
Keywords
Myelodysplastic Syndrome, MDS, Chronic Myelomonocytic Leukemia, CMML, Clofarabine, Clofarex, Clolar, Chemotherapy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
65 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Oral Clofarabine
Arm Type
Experimental
Arm Description
10 mg (Group 1) or 20 mg (Group 2) tablets once a day for 5 days in a row and repeated every 4-8 week cycle.
Intervention Type
Drug
Intervention Name(s)
Clofarabine
Other Intervention Name(s)
Clolar®, Clorafex
Intervention Description
Starting dose 10 mg (Group 1) or 20 mg (Group 2) as tablets once a day for 5 days in a row and repeated every 4-8 weeks. Each 4-8 week period is a cycle.
Primary Outcome Measure Information:
Title
Participants With a Complete Remission (CR)
Description
Complete Remission (CR): Normalization of the peripheral blood and bone marrow with <5% bone marrow blasts, a peripheral blood granulocyte count > (1.0 x 109/ L, and a platelet count > 100 x 109/L). Partial Remission: as above except for the presence of 6-15% marrow blasts, or 50% reduction if <15% at start of treatment. Hematologic Improvement: meets all criteria for CR except for platelet recovery to >100 x 109/L. Clinical Benefit: Platelets increase by 50% and to above 30 x 109/L untransfused (if lower than that pretherapy); or granulocytes increase by 100% and to above 109/L (if lower than that pretherapy); or hemoglobin increase by 2 g/dl; or transfusion independent; or splenomegaly reduction by > 50%; or monocytosis reduction by > 50% if pretreatment > 5 x 109/L.
Time Frame
After 3 courses of treatment, up to 24 weeks.

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patients with MDS and >/= 5% blasts or IPSS risk intermediate or high; patients with Chronic myelomonocytic leukemia (CMML). No prior intensive chemotherapy or high-dose ara-C (>/= 1g/m2). Prior biologic therapies, targeted therapies, or single agent chemotherapy allowed. Patients must have been off chemotherapy for 2 weeks prior to entering this study and recovered from the toxic effects of that therapy, unless there is evidence of rapidly progressive disease. Hydroxyurea is permitted for control of counts prior to treatment. Procrit, GCSF are allowed before therapy. Performance 0-2 (ECOG). Adequate organ function including the following:Adequate liver function (bilirubin of < 2mg/dl), and renal function (creatinine < 2mg/dl), and SGPT (ALT) < 3 X ULN. Adequate cardiac functions (NYHA cardiac III-IV excluded). Signed informed consent. Exclusion Criteria: Nursing and pregnant females. Patients of childbearing potential should practice effective methods of contraception. Child bearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately. Active and uncontrolled infections. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, or psychiatric illness/social situations that would limit compliance with study requirements. Prior clofarabine treatment.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Hagop Kantarjian, MD
Organizational Affiliation
M.D. Anderson Cancer Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
U.T.M.D. Anderson Cancer Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States

12. IPD Sharing Statement

Links:
URL
http://www.mdanderson.org
Description
UT MD Anderson Cancer Center Website

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Oral Clofarabine Study in Patients With Myelodysplastic Syndrome

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