Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Celgene Corporation | INDUSTRY |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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).
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.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Arm A: 5-azacytidine | Experimental | 5-azacytidine as pre-transplant cytoreduction prior to allogeneic stem cell transplantation for High Risk Myelodysplatic Syndromes. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 5-azacytidine | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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%. | 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. | 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%. | 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%. | 2 years |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
Not provided
Inclusion Criteria:
Patients fulfilling the following criteria will be eligible for study entry:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| John M. McCarty, MD | Virginia Commonwealth University | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Massey Cancer Center / Virginia Commonwealth University | Richmond | Virginia | 23298 | United States |
Not provided
This study plans to accrue over one year with a planned 2 year subsequent follow-up. Virginia Commonwealth Unvierstiy (VCU) will be the sole site for this project.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Arm A: 5-azacytidine | 5-azacytidine as pre-transplant cytoreduction prior to allogeneic stem cell transplantation for High Risk Myelodysplatic Syndromes. 5-azacytidine: 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. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Out of the 16 evaluable patients, 12 were able to proceed to transplant.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Arm A: 5-azacytidine | 5-azacytidine as pre-transplant cytoreduction prior to allogeneic stem cell transplantation for High Risk Myelodysplatic Syndromes. 5-azacytidine: 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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%. | Patients surviving after stem cell infusion will be considered in the transplantation cohort; those dying or relapsing prior to this event are considered to have progressed. Outcomes of 5-Axacytidine responders and non-responders will be compared. Patients alive at the time of last observation will be censored. | Posted | Number | 95% Confidence Interval | percentage of participants | 1 year |
|
3 years
Reportable adverse events include relapse, graft failure, any other unexpected grade 3 or 4 that in the opinion of investigator may be related to protocol treatment or death from any cause. All hospitalizations and serious infections (requiring IV antibiotics) will be reported on regular follow-up forms. All other AEs not reportable per protocol.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Arm A: 5-azacytidine | 5-azacytidine as pre-transplant cytoreduction prior to allogeneic stem cell transplantation for High Risk Myelodysplatic Syndromes. 5-azacytidine: 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. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Blood/Bone Marrow - Other (Specify, death-died of progressive MDS) | Blood and lymphatic system disorders | CTCAE 4.0 | Systematic Assessment | Death- died of progressive MDS |
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| John M. McCarty, MD | Massey Cancer Center | 804-828-4360 | jmccarty@vcu.edu |
Not provided
| ID | Term |
|---|---|
| D009190 | Myelodysplastic Syndromes |
| ID | Term |
|---|---|
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D001374 | Azacitidine |
| ID | Term |
|---|---|
| D001372 | Aza Compounds |
| D009930 | Organic Chemicals |
| D003562 | Cytidine |
| D011741 | Pyrimidine Nucleosides |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
| 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. | 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%). | 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%). | 2 years |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
|
|
| Secondary | 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. | Intent to treat analysis including patients who consented and were eligible. Patients enrolled in the study having received at least one 28 day cycle of 5-Azacytidine. As well as engraftment of white blood cells and platelets, graft failure, relapse and Graft Versus Host Disease (GVHD) will be considered in the intent-to-treat analysis. | Posted | Number | 95% Confidence Interval | percentage of participants | 1 year |
|
|
|
| Primary | 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. | Patients surviving after stem cell infusion will be considered in the transplantation cohort; those dying or relapsing prior to this event are considered to have progressed. Outcomes of 5-Axacytidine responders and non-responders will be compared. Patients alive at the time of last observation will be censored. | Posted | Number | 95% Confidence Interval | percentage of participants | 2 years |
|
|
|
| Primary | 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%. | Patients surviving after stem cell infusion will be considered in the transplantation cohort; those dying or relapsing prior to this event are considered to have progressed. Outcomes of 5-Axacytidine responders and non-responders will be compared. Patients alive at the time of last observation will be censored. | Posted | Number | 95% Confidence Interval | percentage of participants | 1 year |
|
|
|
| Primary | 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%. | Patients surviving after stem cell infusion will be considered in the transplantation cohort; those dying or relapsing prior to this event are considered to have progressed. Outcomes of 5-Axacytidine responders and non-responders will be compared. Patients alive at the time of last observation will be censored. | Posted | Number | 95% Confidence Interval | percentage of participants | 2 years |
|
|
|
| Secondary | 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. | Intent to treat analysis including patients who consented and were eligible. Patients enrolled in the study having received at least one 28 day cycle of 5-Azacytidine. As well as engraftment of white blood cells and platelets, graft failure, relapse and Graft Versus Host Disease (GVHD) will be considered in the intent-to-treat analysis. | Posted | Number | 95% Confidence Interval | percentage of participants | 2 years |
|
|
|
| Secondary | 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%). | Intent to treat analysis including patients who consented and were eligible. Patients enrolled in the study having received at least one 28 day cycle of 5-Azacytidine. As well as engraftment of white blood cells and platelets, graft failure, relapse and Graft Versus Host Disease (GVHD) will be considered in the intent-to-treat analysis. | Posted | Number | 95% Confidence Interval | percentage of participants | 1 year |
|
|
|
| Secondary | 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%). | Intent to treat analysis including patients who consented and were eligible. Patients enrolled in the study having received at least one 28 day cycle of 5-Azacytidine. As well as engraftment of white blood cells and platelets, graft failure, relapse and Graft Versus Host Disease (GVHD) will be considered in the intent-to-treat analysis. | Posted | Number | 95% Confidence Interval | percentage of participants | 2 years |
|
|
|
| 13 |
| 16 |
| 0 |
| 16 |
|
| Blood/Bone Marrow- Other | Blood and lymphatic system disorders | CTCAE 4.0 | Systematic Assessment | progression/relapse of MDS/AML |
|
| Seizure | Nervous system disorders | CTCAE 4.0 | Systematic Assessment | Primary Attribution: Unlikely Detailed Attribution: Other Pt with long history of seizure disorder had single seizure. Was seen in ED and discharged to home |
|
| Cholecystitis | Hepatobiliary disorders | CTCAE 4.0 | Systematic Assessment | Primary Attribution: Unrelated Detailed Attribution: Other Drug: 5-Azacytidine Admitted for abdominal pain, found to have acute cholecystitis and had lap cholycystectomy. |
|
| Pain - Abdomen NOS | Gastrointestinal disorders | CTCAE 4.0 | Systematic Assessment | Primary Attribution: Unrelated Detailed Attribution: Other Drug: 5-Azacytidine antibiotics, supportive care Grade 2 |
|
| Nausea | Gastrointestinal disorders | CTCAE 4.0 | Systematic Assessment | Primary Attribution: Unrelated Detailed Attribution: Other Drug: 5-Azacytidine |
|
| Fatigue | Investigations | CTCAE 4.0 | Systematic Assessment | Fatigue (asthenia, lethargy, malaise) Primary Attribution: Unrelated Detailed Attribution: Other Drug: 5-Azacytidine |
|
| Dyspnea | Respiratory, thoracic and mediastinal disorders | CTCAE 4.0 | Systematic Assessment | Dyspnea (shortness of breath) Primary Attribution: Possible Detailed Attribution: Investigational Therapy Drug: 5-Azacytidine |
|
| Infection | Blood and lymphatic system disorders | CTCAE 4.0 | Systematic Assessment | Infection (documented clinically or microbiologically) with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L) - Skin (cellulitis) Primary Attribution:Possible Detailed Attribution:Other Drug: 5-Azacytidine |
|
| Infection | Blood and lymphatic system disorders | CTCAE 4.0 | Systematic Assessment | Infection with unknown ANC |
|
| Hemorrhage | Respiratory, thoracic and mediastinal disorders | CTCAE 4.0 | Systematic Assessment | Hemorrhage/Bleeding |
|
| Opportunistic infection associated with >=Grade 2 Lymphopenia | Blood and lymphatic system disorders | CTCAE 4.0 | Systematic Assessment | patient admitted 3/12/12 for fever and mental status changes, confusion worsened and patient found to have basal ganglia hemorrhage secondary to toxoplasmosis, died in paliative care unit 4/17/12 |
|
| Died of Sepsis | Infections and infestations | CTCAE 4.0 | Systematic Assessment |
|
| Perforation | Gastrointestinal disorders | CTCAE 4.0 | Systematic Assessment | Perforation, GI - Colon |
|
| Febrile Neutropenia | Infections and infestations | CTCAE 4.0 | Systematic Assessment | Febrile neutropenia (fever of unknown origin without clinically or microbiologically documented infection)(ANC <1.0 x 10e9/L, fever >=38.5 degrees C) |
|
| Myositis | Skin and subcutaneous tissue disorders | CTCAE 4.0 | Systematic Assessment | Myositis (inflammation/damage of muscle) |
|
Not provided
Not provided
| D011743 |
| Pyrimidines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D009705 | Nucleosides |
| D009706 | Nucleic Acids, Nucleotides, and Nucleosides |
| D012263 | Ribonucleosides |