Acute Myeloid LeukemiaPhase 2NCT01700673
Phase II Study of Azacitidine and Sargramostim as Maintenance Treatment for Poor-Risk AML or MDS
A Phase 2 study testing Azacitidine and Sargramostim in people with Acute Myeloid Leukemia and Myelodysplastic Syndrome, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins at 1 site in United States.
Trial summary
To determine the impact of maintenance therapy in patients with MDS/AML in remission. Read more about this study
- Who is it for?
- Ages 6 months and older
- Conditions the study is for
- Acute Myeloid Leukemia, Myelodysplastic Syndrome
- What is being tested?
- Azacitidine and Sargramostim
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2013to Jun 2020
- 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 6 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age > 6 months
- Initial diagnosis of poor -risk AML or MDS (defined in section 3.2), treated with either stem cell transplant or cytarabine-based consolidation chemotherapy, within the past 60-185 days
- ECOG performance status 0-2
- No morphologic evidence of leukemia or active MDS as determined by JHH Hematopathologist independent review of a bone marrow aspirate and biopsy done following the completion of therapy and within 14 days prior to enrollment
- Peripheral blood count recovery: Neutrophil count ≥ 1000 /µL, platelet count ≥ 50x 109 /µL without platelet transfusions, and adequate hematocrit independent of red cell transfusions .
- No evidence of extramedullary leukemia, such as CNS or soft tissue involvement
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- Adequate end organ function as measured by the following: AST and ALT < 4 x normal, total serum bilirubin < 2 x upper limit normal (unless due to hemolysis, Gilbert's syndrome, or ineffective erythropoiesis), creatinine < 2 x upper limit of normal
- Ability to give informed consent
- In agreement to use an effective barrier method of birth control to avoid pregnancy during the study and for a minimum of 30 days after study treatment, for all male and female patients who are fertile
Who cannot join
- Patients with untreated or uncontrolled infections
- Patients with untreated or uncontrolled grade 3 or 4 GVHD
- Pregnancy and lactation
- Concurrent use of any other investigational agents.
- Known HIV-positive patients.
- Known hypersensitivity to 5AC or GM-CSF
Trial locations and contacts
1 site in 1 country.
United States1 site
The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore, Maryland, 21287
About this study
Official title: A Phase II Study of 5-Azacitidine (5AC) in Combination With Sargramostim (GM-CSF) as Maintenance Treatment, After Definitive Therapy With Either Stem Cell Transplant (SCT) or Cytarabine-based Chemotherapy, in Patients With Poor-risk Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)
To determine the impact of maintenance therapy in patients with MDS/AML in remission.
Read the detailed description
We propose a phase II study to determine the impact of maintenance therapy with 5-azacytidine and GM-CSF in patients with poor-risk AML or MDS, who are in remission after definitive treatment with either stem cell transplant or cytarabine-based consolidation chemotherapy.
In order to precede relapse and to avoid lead time bias, treatment would need to commence within 185 days of definitive therapy. Furthermore, approximately 50% of relapses occur within the first year and up to 80% within two years after SCT, therefore we would limit the duration of maintenance therapy to one year, followed by two years of follow-up.
- Conditions
- Acute Myeloid Leukemia, Myelodysplastic Syndrome
- Being tested
- Azacitidine, Sargramostim
- Keywords
- maintenance treatment, stem cell transplant, cytarabine-based chemotherapy
- Medical subject headings
- Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AzacitidineDrug
Also called Vidaza, 5-azacytidine, Azacytidine
Azacitidine will be administered days 1-5 of a 28 day cycle. Treatment is planned for a total of 12 cycles.
SargramostimBiological
Also called GM-CSF
Sargramostim will be administered days 1-10 of a 28 day cycle. Treatment is planned for a total of 12 cycles.
| Study group | Role | What they receive |
|---|---|---|
| Myeloablative BMT | Experimental | Azacitidine and sargramostim after myeloablative stem cell transplant Receives: Drug: Azacitidine; Biological: Sargramostim |
| Non-myeloablative BMT | Experimental | Azacitidine and sargramostim after non-myeloablative stem cell transplant Receives: Drug: Azacitidine; Biological: Sargramostim |
| Standard consolidation | Experimental | Azacitidine and sargramostim after standard consolidation Receives: Drug: Azacitidine; Biological: Sargramostim |
What is being measured
The primary outcome is the main question the study is designed to answer.
Two-year Relapse Free Survival of Patients
To evaluate the two-year relapse-free survival (RFS) of patients with poor-risk Acute Myeloid Leukemia (AML) or Myelodysplasia (MDS), who receive maintenance treatment with 5-Azacytidine (5AC) in combination with sargramostim (GM-CSF) during remission, following definitive therapy with either a stem cell transplant (SCT) or cytarabine-based consolidation chemotherapy.
Measured: 2 year
Secondary outcomes (3)
Hematologic Toxicity as Determined by Anemia
Percentage of patients with anemia, the most commonly reported hematologic toxicity, after receiving the combination of Azacitidine and sargramostim
Measured: 1 year
One-year RFS
We will report the number of participants with one year RFS
Measured: 1 year
Overall Survival
Percentage of participants with overall survival at 2 years.
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 posted4 Oct 2012 (estimated)
- Study startJun 2013
- Primary completionJun 2020
- Study completionJun 2020
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 Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- 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
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Margaret Showel, MD, Study chair, JHU
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01700673
- Sponsor's protocol ID
- J1240
- Other IDs
- P01CA015396, NA_00072223
- Status last verified
- Aug 2021
- First submitted
- 2 Oct 2012
- Last updated
- 18 Oct 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of 5-Azacitidine (5AC) in Combination With Sargramostim (GM-CSF) as Maintenance Treatment, After Definitive Therapy With Either Stem Cell Transplant (SCT) or Cytarabine-based Chemotherapy, in Patients With Poor-risk Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)
- ClinicalTrials.gov ID
- NCT01700673
- Acronym / sponsor ID
- J1240
- Conditions
- Acute Myeloid Leukemia, Myelodysplastic Syndrome
- Keywords
- maintenance treatment, stem cell transplant, cytarabine-based chemotherapy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Jun 2013 (actual)
- Primary completion
- Jun 2020 (actual)
- Study completion
- Jun 2020 (actual)
- First posted
- 4 Oct 2012 (estimated)
- Results
- Posted 2 Sep 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To determine the impact of maintenance therapy in patients with MDS/AML in remission.
Detailed description
We propose a phase II study to determine the impact of maintenance therapy with 5-azacytidine and GM-CSF in patients with poor-risk AML or MDS, who are in remission after definitive treatment with either stem cell transplant or cytarabine-based consolidation chemotherapy.
In order to precede relapse and to avoid lead time bias, treatment would need to commence within 185 days of definitive therapy. Furthermore, approximately 50% of relapses occur within the first year and up to 80% within two years after SCT, therefore we would limit the duration of maintenance therapy to one year, followed by two years of follow-up.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Myeloablative BMT | Experimental | Drug: Azacitidine, Biological: SargramostimAzacitidine and sargramostim after myeloablative stem cell transplant |
| Non-myeloablative BMT | Experimental | Drug: Azacitidine, Biological: SargramostimAzacitidine and sargramostim after non-myeloablative stem cell transplant |
| Standard consolidation | Experimental | Drug: Azacitidine, Biological: SargramostimAzacitidine and sargramostim after standard consolidation |
Who may qualify
- Ages
- 6 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Age > 6 months
- Initial diagnosis of poor -risk AML or MDS (defined in section 3.2), treated with either stem cell transplant or cytarabine-based consolidation chemotherapy, within the past 60-185 days
- ECOG performance status 0-2
- No morphologic evidence of leukemia or active MDS as determined by JHH Hematopathologist independent review of a bone marrow aspirate and biopsy done following the completion of therapy and within 14 days prior to enrollment
- Peripheral blood count recovery: Neutrophil count ≥ 1000 /µL, platelet count ≥ 50x 109 /µL without platelet transfusions, and adequate hematocrit independent of red cell transfusions .
Key exclusion criteria (6 listed)
- Patients with untreated or uncontrolled infections
- Patients with untreated or uncontrolled grade 3 or 4 GVHD
- Pregnancy and lactation
- Concurrent use of any other investigational agents.
- Known HIV-positive patients.
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 |
|---|---|---|
| AzacitidineAlso: Vidaza, 5-azacytidine, AzacytidineAzacitidine will be administered days 1-5 of a 28 day cycle. Treatment is planned for a total of 12 cycles. | Drug | Myeloablative BMT, Non-myeloablative BMT, Standard consolidation |
| SargramostimAlso: GM-CSFSargramostim will be administered days 1-10 of a 28 day cycle. Treatment is planned for a total of 12 cycles. | Biological | Myeloablative BMT, Non-myeloablative BMT, Standard consolidation |
Primary outcome
Two-year Relapse Free Survival of Patients
Time frame: 2 year
To evaluate the two-year relapse-free survival (RFS) of patients with poor-risk Acute Myeloid Leukemia (AML) or Myelodysplasia (MDS), who receive maintenance treatment with 5-Azacytidine (5AC) in combination with sargramostim (GM-CSF) during remission, following definitive therapy with either a stem cell transplant (SCT) or cytarabine-based consolidation chemotherapy.
3 secondary outcomes
Hematologic Toxicity as Determined by Anemia
Time frame: 1 year
One-year RFS
Time frame: 1 year
Overall Survival
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 18 Oct 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Margaret Showel, MD study chair
JHU
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 Azacitidine and Sargramostim in people with Acute Myeloid Leukemia and Myelodysplastic Syndrome, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins 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 9 inclusion and 6 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 18 Oct 2022. 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 2 Sep 2021). 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?
Two-year Relapse Free Survival of Patients (time frame: 2 year). 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 18 Oct 2022; AzTrials last refreshed its copy on 26 Oct 2026.