Myelodysplastic SyndromePhase 2NCT00113321
Low-Dose Decitabine in Myelodysplastic Syndrome Post Azacytidine Failure
A Phase 2 study testing Decitabine in people with Myelodysplastic Syndrome and Chronic Myelomonocytic Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
To study if decitabine can help to control Myelodysplastic Syndrome (MDS) in patients who have failed on therapy with azacytidine, the current standard of therapy. 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?
- Decitabine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2005to Nov 2008
- 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
- MDS and 5% or more marrow blasts, or IPSS risk intermediate 1-2 or high risk; or chronic myelomonocytic leukemia. Patients must have failed therapy with azacytidine.
- Performance status 0-2 (ECOG scale); adequate hepatic (bilirubin < 2 mg/dl) and renal functions (creatinine <2mg/dl); New York Heart Association (NYHA)cardiac status III-IV excluded.
- Signed informed consent.
- No prior intensive combination chemotherapy or high-dose ara-C (>/= 1g/m*2 per dose). Prior biologic therapies, targeted therapies and 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. Use of hydroxyurea for patients with rapidly proliferative disease is allowed for the first two weeks on therapy.
Who cannot join
- Nursing and pregnant females are excluded. Patients of childbearing potential should practice effective methods of contraception. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
- Patients with 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase II Study of Low-Dose Decitabine (5-AZA-2'-Deoxycytidine) in Myelodysplastic Syndrome (MDS) Post Azacytidine (AZA) Failure
To study if decitabine can help to control Myelodysplastic Syndrome (MDS) in patients who have failed on therapy with azacytidine, the current standard of therapy.
Read the detailed description
Methylation is a change that occurs to Deoxyribonucleic acid (DNA) that affects gene usage in human cells. Abnormal methylation is very common in leukemias, which is a related disease to MDS. Decitabine is a new drug that blocks DNA methylation. Researchers want to find out if blocking methylation will help control MDS.
Before you can start treatment on this study, you will have what are called "screening tests". These tests will help the doctor decide if you are eligible to take part in the study. You will have a physical exam, routine blood tests (between 4-6 tablespoons), and a bone marrow aspirate. To collect a bone marrow aspirate, an area of the hip or chest bone is numbed with anesthetic and a small amount of bone marrow is withdrawn through a large needle. Women who are able to have children must have a negative blood or urine pregnancy test.
If you are found to be eligible to take part in this study, you will receive decitabine by vein over one hour, once a day, for 5 days (1 course). If this is not possible due to complications, you will receive the drug as an injection under the skin twice a day for 5 days (1 course). Treatment will be given every 4 to 8 weeks depending on how well your blood counts recover.
After completing 8-12 weeks of therapy, response will be evaluated. If the response to treatment is good, treatment with decitabine will continue. Decitabine treatment may be continued for up to 12 courses, or as long as it is judged best to control the leukemia.
During this study, you will need to visit your doctor periodically for physical exams and measurement of vital signs. The frequency of doctor visits will vary depending on your physical condition, but will be required at least once a month.
Blood tests (about 2 teaspoons) will be done about every week during the first 6-8 weeks of treatment, then every 1 to 2 weeks for the length of the study. The blood samples will be used for routine lab tests. Every 1-3 courses, bone marrow samples will also be taken to check cells related to the disease before, during (every 1-3 courses), and after completion of this study.
You will be taken off study if the disease gets worse or intolerable side effects occur.
This is an investigational study. Decitabine is not yet Food and Drug Administration (FDA)approved. It will be provided free of charge by MGI Pharma. Up to 40 patients will take part in this study. All will be enrolled at M. D. Anderson.
- Being tested
- Decitabine
- Keywords
- Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia, Azacytidine Failure, Decitabine
- 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.
DecitabineDrug
Also called Dacogen®
20 mg/m2 IV over 1 hour daily x 5 days.
| Study group | Role | What they receive |
|---|---|---|
| Decitabine | Experimental | 20 mg/m2 by vein (IV) over 1 hour daily x 5 days. Receives: Drug: Decitabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response
Participants with Overall Response, categorized as 'Complete Response' to represent remission or 'No Complete Response' for lack of remission. Response evaluation after completing one course of therapy (8-12 weeks), then bone marrow aspiration to document remission every 1-3 courses.
Measured: Blood tests baseline and after completing 8-12 weeks of therapy
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2005 (estimated)
- Study startMar 2005
- Primary completionNov 2008
- Study completionNov 2008
Why it stopped: Low Accrual
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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Eisai Inc.
- Responsible party
- Sponsor
- Investigators
- Hagop Kantarjian, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00113321
- Sponsor's protocol ID
- 2004-0468
- Status last verified
- Aug 2012
- First submitted
- 7 Jun 2005
- Last updated
- 7 Aug 2012
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Low-Dose Decitabine (5-AZA-2'-Deoxycytidine) in Myelodysplastic Syndrome (MDS) Post Azacytidine (AZA) Failure
- ClinicalTrials.gov ID
- NCT00113321
- Acronym / sponsor ID
- 2004-0468
- Keywords
- Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia, Azacytidine Failure, Decitabine
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Low Accrual
- Enrolment
- 16 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- Eisai Inc.
- Study start
- Mar 2005
- Primary completion
- Nov 2008 (actual)
- Study completion
- Nov 2008 (actual)
- First posted
- 8 Jun 2005 (estimated)
- Results
- Posted 21 Feb 2011
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To study if decitabine can help to control Myelodysplastic Syndrome (MDS) in patients who have failed on therapy with azacytidine, the current standard of therapy.
Detailed description
Methylation is a change that occurs to Deoxyribonucleic acid (DNA) that affects gene usage in human cells. Abnormal methylation is very common in leukemias, which is a related disease to MDS. Decitabine is a new drug that blocks DNA methylation. Researchers want to find out if blocking methylation will help control MDS.
Before you can start treatment on this study, you will have what are called "screening tests". These tests will help the doctor decide if you are eligible to take part in the study. You will have a physical exam, routine blood tests (between 4-6 tablespoons), and a bone marrow aspirate. To collect a bone marrow aspirate, an area of the hip or chest bone is numbed with anesthetic and a small amount of bone marrow is withdrawn through a large needle. Women who are able to have children must have a negative blood or urine pregnancy test.
If you are found to be eligible to take part in this study, you will receive decitabine by vein over one hour, once a day, for 5 days (1 course). If this is not possible due to complications, you will receive the drug as an injection under the skin twice a day for 5 days (1 course). Treatment will be given every 4 to 8 weeks depending on how well your blood counts recover.
After completing 8-12 weeks of therapy, response will be evaluated. If the response to treatment is good, treatment with decitabine will continue. Decitabine treatment may be continued for up to 12 courses, or as long as it is judged best to control the leukemia.
During this study, you will need to visit your doctor periodically for physical exams and measurement of vital signs. The frequency of doctor visits will vary depending on your physical condition, but will be required at least once a month.
Blood tests (about 2 teaspoons) will be done about every week during the first 6-8 weeks of treatment, then every 1 to 2 weeks for the length of the study. The blood samples will be used for routine lab tests. Every 1-3 courses, bone marrow samples will also be taken to check cells related to the disease before, during (every 1-3 courses), and after completion of this study.
You will be taken off study if the disease gets worse or intolerable side effects occur.
This is an investigational study. Decitabine is not yet Food and Drug Administration (FDA)approved. It will be provided free of charge by MGI Pharma. Up to 40 patients will take part in this study. All will be enrolled at M. D. Anderson.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Decitabine | Experimental | Drug: Decitabine20 mg/m2 by vein (IV) over 1 hour daily x 5 days. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- MDS and 5% or more marrow blasts, or IPSS risk intermediate 1-2 or high risk; or chronic myelomonocytic leukemia. Patients must have failed therapy with azacytidine.
- Performance status 0-2 (ECOG scale); adequate hepatic (bilirubin < 2 mg/dl) and renal functions (creatinine <2mg/dl); New York Heart Association (NYHA)cardiac status III-IV excluded.
- Signed informed consent.
- No prior intensive combination chemotherapy or high-dose ara-C (>/= 1g/m*2 per dose). Prior biologic therapies, targeted therapies and 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. Use of hydroxyurea for patients with rapidly proliferative disease is allowed for the first two weeks on therapy.
Key exclusion criteria (3 listed)
- Nursing and pregnant females are excluded. Patients of childbearing potential should practice effective methods of contraception. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
- Patients with 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.
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 |
|---|---|---|
| DecitabineAlso: Dacogen®20 mg/m2 IV over 1 hour daily x 5 days. | Drug | Decitabine |
Primary outcome
Overall Response
Time frame: Blood tests baseline and after completing 8-12 weeks of therapy
Participants with Overall Response, categorized as 'Complete Response' to represent remission or 'No Complete Response' for lack of remission. Response evaluation after completing one course of therapy (8-12 weeks), then bone marrow aspiration to document remission every 1-3 courses.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Aug 2012.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- UT MD Anderson Cancer CenterLink provided by the sponsor.
Related trials for Myelodysplastic Syndrome
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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 Decitabine 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 5 inclusion and 3 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: stopped early, as of the sponsor's last update on 7 Aug 2012. 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 21 Feb 2011). 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?
Overall Response (time frame: Blood tests baseline and after completing 8-12 weeks of therapy). 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 7 Aug 2012.