Myelodysplastic SyndromePhase 2NCT00299156
Oral Clofarabine Study in Patients With Myelodysplastic Syndrome
A Phase 2 study testing Clofarabine in people with Myelodysplastic Syndrome and Chronic Myelomonocytic Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia
- What is being tested?
- Clofarabine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 65 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2006to Dec 2012
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
U.T.M.D. Anderson Cancer Center
Houston, Texas, 77030
About this study
Official title: Phase II Study of Oral Clofarabine in Myelodysplastic Syndrome (MDS)
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.
Read the 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.
- Being tested
- Clofarabine
- Keywords
- Myelodysplastic Syndrome, MDS, Chronic Myelomonocytic Leukemia, CMML, Clofarabine, Clofarex, Clolar, Chemotherapy
- Medical subject headings
- Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ClofarabineDrug
Also called Clolar®, Clorafex
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.
| Study group | Role | What they receive |
|---|---|---|
| Oral Clofarabine | Experimental | 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. Receives: Drug: Clofarabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: After 3 courses of treatment, up to 24 weeks.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Mar 2006 (estimated)
- Study startMar 2006
- Primary completionDec 2012
- Study completionDec 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Myelodysplastic SyndromeStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 65 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Genzyme, a Sanofi Company
- Responsible party
- Sponsor
- Investigators
- Hagop Kantarjian, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00299156
- Sponsor's protocol ID
- 2005-0536
- Status last verified
- Aug 2015
- First submitted
- 3 Mar 2006
- Last updated
- 3 Nov 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Oral Clofarabine in Myelodysplastic Syndrome (MDS)
- ClinicalTrials.gov ID
- NCT00299156
- Acronym / sponsor ID
- 2005-0536
- Keywords
- Myelodysplastic Syndrome, MDS, Chronic Myelomonocytic Leukemia, CMML, Clofarabine, Clofarex, Clolar, Chemotherapy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 65 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- Genzyme, a Sanofi Company
- Study start
- Mar 2006
- Primary completion
- Dec 2012 (actual)
- Study completion
- Dec 2012 (actual)
- First posted
- 6 Mar 2006 (estimated)
- Results
- Posted 18 Sep 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Oral Clofarabine | Experimental | Drug: Clofarabine10 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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ClofarabineAlso: Clolar®, ClorafexStarting 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. | Drug | Oral Clofarabine |
Primary outcome
Participants With a Complete Remission (CR)
Time frame: After 3 courses of treatment, up to 24 weeks.
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Nov 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- UT MD Anderson Cancer Center WebsiteLink provided by the sponsor.
Related trials for Myelodysplastic Syndrome
RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Active, not recruitingPhase 1/2
Omacetaxine + Azacitidine in Untreated Patients With High Grade MDS
High Grade Myelodysplastic Syndromes
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Hagop Kantarjian, MD principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Clofarabine in people with Myelodysplastic Syndrome and Chronic Myelomonocytic Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Nov 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 18 Sep 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Participants With a Complete Remission (CR) (time frame: After 3 courses of treatment, up to 24 weeks.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Nov 2015.