Myelodysplastic SyndromePhase 2NCT00721214
5-Azacytidine Prior to Allogeneic Stem Cell Transplant in High Risk Myelodysplastic Syndrome
A Phase 2 study testing 5-azacytidine in people with Myelodysplastic Syndrome, run by Virginia Commonwealth University at 1 site in United States.
Trial summary
The purpose of this study is to examine the feasibility and efficacy of using the demethylating agent 5-Azacytidine prior to allogeneic stem cell transplantation in patients with high risk myelodysplastic syndrome (MDS). Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Myelodysplastic Syndrome
- What is being tested?
- 5-azacytidine
- 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
- Jul 2008to Jun 2014
- 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 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients fulfilling the following criteria will be eligible for study entry:
- Diagnosis of MDS according to WHO criteria
- Intermediate-2 or high risk by IPSS score
- Clinically able to receive 5-Azacytidine
- Serum bilirubin levels </=1.5 times the upper limit of the normal range for the laboratory (ULN). Higher levels are acceptable if these can be attributed to active hemolysis or ineffective erythropoiesis
- Serum glutamic-oxaloacetic transaminase (SGOT) or serum glutamic-pyruvic transaminase (SGPT) levels </=2 x ULN
Show all 12Show fewer
- Serum creatinine levels </=1.5 x ULN
- Negative serum pregnancy test prior to 5-Azacytidine treatment for women of childbearing potential
- Women and men of childbearing potential agree to use contraception while receiving treatment with 5-Azacytidine
- Potentially eligible for allogeneic transplantation
- No prior allogeneic transplant
- Age 18 to 70, inclusive.
Who cannot join
- Known or suspected hypersensitivity to 5-azacytidine or mannitol
- Patients previously treated with 5-azacytidine or deoxyazacytidine
- Pregnant or breast feeding
- Patients with advanced malignant hepatic tumors
Trial locations and contacts
1 site in 1 country.
United States1 site
Massey Cancer Center / Virginia Commonwealth University
Richmond, Virginia, 23298
About this study
Official title: A Phase II Study of the Use of 5-Azacytidine as Pre-Transplant Cytoreduction Prior to Allogeneic Stem Cell Transplantation for High Risk Myelodysplastic Syndromes
The purpose of this study is to examine the feasibility and efficacy of using the demethylating agent 5-Azacytidine prior to allogeneic stem cell transplantation in patients with high risk myelodysplastic syndrome (MDS).
Read the detailed description
The study drug, 5-azacytidine, is given daily intravenously for 7 days. After every 2 cycles study participants will have a bone marrow test to evaluate the effect of the 5-azacytidine on the Myelodysplastic Syndrome (MDS). Participants continue to get cycles of 5-Azacytidine until 2 bone marrow tests show the MDS has stopped responding to the treatment. At that time they will undergo a transplant if a donor is available.
- Conditions
- Myelodysplastic Syndrome
- Being tested
- 5-azacytidine
- Keywords
- Myelodysplastic Syndrome, 5-azacytidine, allogeneic stem cell transplantation
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
5-azacytidineDrug
Also called Mylosar, Vidaza, 5-AC, 5-AZC
The recommended starting dose for the first treatment cycle, for all patients regardless of baseline hematology laboratory values, is 75 mg/m2 subcutaneously or intravenously, daily for 7 days.
| Study group | Role | What they receive |
|---|---|---|
| Arm A: 5-azacytidine | Experimental | 5-azacytidine as pre-transplant cytoreduction prior to allogeneic stem cell transplantation for High Risk Myelodysplatic Syndromes. Receives: Drug: 5-azacytidine |
What is being measured
The primary outcome is the main question the study is designed to answer.
One Year Overall Survival of Allogeneic Transplant Recipients After Transplantation
Percentage of patients alive one year after their transplantation, as estimated by the Kaplan-Meier survival curve. The estimated one year survival rate from this curve is 50%, while the estimated two year survival rate is 50%.
Measured: 1 year
Two Year Overall Survival of Allogeneic Transplant Recipients After Transplantation
Percentage of patients alive two years after their transplantation, as estimated by the Kaplan-Meier survival curve. The estimated two year survival rate is 50%, the same as one year survival rate.
Measured: 2 years
One Year Event Free Survival (EFS) for Allogeneic Transplant Recipients After Transplantation
Percentage of participants that received allogeneic transplant and had event free survival, as estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. The estimated one-year event-free survival rate is the same as overall survival, 50%.
Measured: 1 year
Two Year Event Free Survival (EFS) for Allogeneic Transplant Recipients After Transplantation
Percentage of participants that received allogeneic transplant and had event free survival. The percentage of patients was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. The estimated two-year event-free survival rate is the same as overall survival, 50%.
Measured: 2 years
Secondary outcomes (4)
One-year Overall Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts
Percentage of participants alive one year after their first treatment was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. Patients alive at the time of last observation will be censored. The estimated one year overall survival rate from this curve is 47%. The one year overall survival is the same as one year event free survival rate.
Measured: 1 year
Two-year Overall Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts.
Percentage of participants alive two years after their first treatment was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. Patients alive at the time of last observation will be censored.The estimated two year survival rate is 37% .The estimated two-year overall survival rate is the same as two-year event free survival.
Measured: 2 years
One-year Event-free Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts
Percentage of participants with one year event free survival after their first treatment was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. Patients alive at the time of last observation will be censored.The estimated one-year event-free survival rate is the same as for overall survival, 47% (SE = 13.6%).
Measured: 1 year
Two-year Event-free Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts
Percentage of participants with two year event free survival after their first treatment was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. Patients alive at the time of last observation will be censored.The estimated two- year event-free survival rate is the same as for overall survival, 37% (SE = 14.3%).
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jul 2008 (estimated)
- Study startJul 2008
- Primary completionJun 2014
- Study completionJun 2014
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 Virginia Commonwealth UniversityThe 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
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Virginia Commonwealth University
- Sponsor type
- University, hospital or other
- Collaborators
- Celgene Corporation
- Responsible party
- Sponsor
- Investigators
- John M. McCarty, MD, Study chair, Virginia Commonwealth University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00721214
- Sponsor's protocol ID
- MCC-11328
- Status last verified
- Feb 2016
- First submitted
- 22 Jul 2008
- Last updated
- 2 Mar 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 2 Mar 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of the Use of 5-Azacytidine as Pre-Transplant Cytoreduction Prior to Allogeneic Stem Cell Transplantation for High Risk Myelodysplastic Syndromes
- ClinicalTrials.gov ID
- NCT00721214
- Acronym / sponsor ID
- MCC-11328
- Conditions
- Myelodysplastic Syndrome
- Keywords
- Myelodysplastic Syndrome, 5-azacytidine, allogeneic stem cell transplantation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Virginia Commonwealth UniversityUniversity, hospital or other
- Collaborators
- Celgene Corporation
- Study start
- Jul 2008
- Primary completion
- Jun 2014 (actual)
- Study completion
- Jun 2014 (actual)
- First posted
- 24 Jul 2008 (estimated)
- Results
- Posted 3 Aug 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to examine the feasibility and efficacy of using the demethylating agent 5-Azacytidine prior to allogeneic stem cell transplantation in patients with high risk myelodysplastic syndrome (MDS).
Detailed description
The study drug, 5-azacytidine, is given daily intravenously for 7 days. After every 2 cycles study participants will have a bone marrow test to evaluate the effect of the 5-azacytidine on the Myelodysplastic Syndrome (MDS). Participants continue to get cycles of 5-Azacytidine until 2 bone marrow tests show the MDS has stopped responding to the treatment. At that time they will undergo a transplant if a donor is available.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Arm A: 5-azacytidine | Experimental | Drug: 5-azacytidine5-azacytidine as pre-transplant cytoreduction prior to allogeneic stem cell transplantation for High Risk Myelodysplatic Syndromes. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Patients fulfilling the following criteria will be eligible for study entry:
Key exclusion criteria (4 listed)
- Known or suspected hypersensitivity to 5-azacytidine or mannitol
- Patients previously treated with 5-azacytidine or deoxyazacytidine
- Pregnant or breast feeding
- Patients with advanced malignant hepatic tumors
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 |
|---|---|---|
| 5-azacytidineAlso: Mylosar, Vidaza, 5-AC, 5-AZCThe recommended starting dose for the first treatment cycle, for all patients regardless of baseline hematology laboratory values, is 75 mg/m2 subcutaneously or intravenously, daily for 7 days. | Drug | Arm A: 5-azacytidine |
Primary outcome
One Year Overall Survival of Allogeneic Transplant Recipients After Transplantation
Time frame: 1 year
Percentage of patients alive one year after their transplantation, as estimated by the Kaplan-Meier survival curve. The estimated one year survival rate from this curve is 50%, while the estimated two year survival rate is 50%.
Two Year Overall Survival of Allogeneic Transplant Recipients After Transplantation
Time frame: 2 years
Percentage of patients alive two years after their transplantation, as estimated by the Kaplan-Meier survival curve. The estimated two year survival rate is 50%, the same as one year survival rate.
One Year Event Free Survival (EFS) for Allogeneic Transplant Recipients After Transplantation
Time frame: 1 year
Percentage of participants that received allogeneic transplant and had event free survival, as estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. The estimated one-year event-free survival rate is the same as overall survival, 50%.
Two Year Event Free Survival (EFS) for Allogeneic Transplant Recipients After Transplantation
Time frame: 2 years
Percentage of participants that received allogeneic transplant and had event free survival. The percentage of patients was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. The estimated two-year event-free survival rate is the same as overall survival, 50%.
4 secondary outcomes
One-year Overall Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts
Time frame: 1 year
Two-year Overall Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts.
Time frame: 2 years
One-year Event-free Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts
Time frame: 1 year
Two-year Event-free Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts
Time frame: 2 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Mar 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
John M. McCarty, MD study chair
Virginia Commonwealth University
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 5-azacytidine in people with Myelodysplastic Syndrome, run by Virginia Commonwealth University 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 12 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 2 Mar 2016. 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 3 Aug 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?
One Year Overall Survival of Allogeneic Transplant Recipients After Transplantation (time frame: 1 year). The study lists 4 primary outcomes in all. 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 2 Mar 2016.