Acute Myeloid LeukemiaPhase 2NCT02684162
Guadecitabine and Donor Lymphocyte Infusion in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome Relapsing After Allogeneic Stem Cell Transplant
A Phase 2 study testing Donor Lymphocytes, Guadecitabine and Laboratory Biomarker Analysis in people with Acute Myeloid Leukemia and Chronic Myelomonocytic Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
This phase IIa trial studies how well guadecitabine works in treating patients with acute myelogenous leukemia and myelodysplastic syndrome that has returned after a period of improvement after allogeneic stem cell transplant. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, Myelodysplastic Syndrome
- What is being tested?
- Donor Lymphocytes, Guadecitabine and Laboratory Biomarker Analysis
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 55 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Jun 2016to 8 Oct 2024
- 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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of acute myeloid leukemia (AML) and myelodysplastic syndrome (including chronic myelomonocytic leukemia [CMML]) according to WHO classification that underwent first allogeneic hematopoietic cell transplant (HSCT) with either peripheral blood or bone marrow as the source of the hematopoietic stem cells
- No more than 1 antigen mismatch at human leukocyte antigen (HLA)-A, B, C, DRB1 and DQB1 locus for either related or unrelated donor
- High risk AML and MDS patients will be included
- Cohort 1: morphological relapse after stem cell transplant:
- MDS patients: re-appearance of dysplastic changes in the bone marrow, with or without increase in bone marrow last count, which is pathologically consistent with myelodysplastic syndrome;
- AML patients: bone marrow blast count >= 5%
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- Cohort 2: Persistence or reappearance of minimal residual disease by flow cytometry or cytogenetic or molecular testing while being in morphological remission after allogeneic stem cell transplantation
- Cohort 3: High risk AML and MDS patients who are in complete remission morphologically with no evidence of minimal residual disease by flow cytometry or cytogenetic or molecular testing after allogeneic stem cell transplantation
- MDS patients:
- Cytogenetics consistent with poor or very poor risk group by 5-risk classification;
- Cytogenetics consistent with monosomal karyotype
- Bone marrow blast count > 5% but less than 20% at any time during their disease course before HSCT
- Peripheral blood blast =< 5% at HSCT
- Therapy-related MDS
- AML patients:
- Cytogenetics and molecular features consistent with adverse risk group by European LeukemiaNet classification for AML;
- Presence of minimal residual disease by multi-color flow cytometry or cytogenetics or molecular studies at the time of HSCT;
- Presence of active disease defined as bone marrow blast count > 5% but less than =< 10% at the time of HSCT
- Peripheral blood blast count =< 5% at HSCT
- Therapy-related AML
- Be able to start the drug therapy between 42 to 100 days following allogeneic SCT;
- No more than 1 prior allogeneic SCT
- Post-transplant bone marrow consistent with complete remission with no evidence of minimal residual disease by flow-cytometry or cytogenetics or molecular testing
- Adequate engraftment within 14 days prior to starting study drug: absolute neutrophil count (ANC) >= 1.0 x 10^9/L without daily use of myeloid growth factor; and, platelet >= 50 x 10^9/L without platelet transfusion within 1 week
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2
- Serum creatinine =< 1.5 mg/dL or creatinine clearance greater or equal than 40 cc/min as defined by the Cockcroft-Gault equation
- Serum bilirubin =< 1.5 x upper limit of normal (ULN)
- Aspartate transaminase (aspartate aminotransferase [AST]) or alanine transaminase (alanine aminotransferase [ALT]) =< 2.5 x ULN
- Alkaline phosphatase =< 2.5 x upper limit (UL)
- No active bleeding
- No uncontrolled graft versus host disease (GVHD)
- No clinical evidence of life-threatening infection
- Capable of understanding the investigational nature, potential risks and benefits of the study, and able to provide valid informed consent
- Human immunodeficiency virus (HIV) negative and hepatitis B surface antigen (HBs-Ag) negative
- Negative serum or urine pregnancy test for women with reproductive potential; the only subjects who will be exempt from this criterion are postmenopausal women (defined as women who have been amenorrheic for > 12 months) or subjects who have been surgically sterilized or otherwise proven sterile
Who cannot join
- Use of any anti-leukemic agents after relapse is documented (note that the use of these anti-leukemic agents given as post-transplant maintenance therapy is allowed in this study, e.g., subcutaneous or oral 5-azacytidine or FLT3 inhibitors for maintenance) for cohorts 1 and 2
- Bone marrow blast count > 60% for cohort 1
- Use of any of the following after transplantation and prior to starting study therapy for cohort 3:
- Investigational agents/therapies
- Anti-leukemic agents given as post-transplant maintenance therapy (e.g., subcutaneous or oral 5-azacytidine or FLT3 inhibitors for maintenance)
- Active acute graft versus host disease (GVHD) grade II or higher
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- Active chronic GVHD that is extensive
- Concurrent use of systemic immune suppressive other than calcineurin inhibitors and sirolimus
- Active uncontrolled systemic fungal, bacterial or viral infection
- Symptomatic or uncontrolled arrhythmias
- Significant active cardiac disease within the previous 6 months, including: New York Heart Association (NYHA) class III or IV congestive heart failure; unstable angina or angina requiring surgical or medical intervention, and/or; myocardial infarction
- Known active viral infection with human immunodeficiency virus (HIV), hepatitis B virus (HBV) or hepatitis C virus (HCV)
- Prior history of solid tumor unless the subject has been free of the disease for >= 1 year; however, subjects with the following history/concurrent conditions are allowed: basal or squamous cell carcinoma of the skin; carcinoma in situ of the cervix; carcinoma in situ of the breast; incidental histologic finding of prostate cancer (T1a or T1b using the tumor, node, metastasis [TNM] clinical staging system)
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase II Trial to Assess the Efficacy and Toxicity of SGI-110 With DLI for the Treatment of AML or MDS Relapsing After Allogeneic Stem Cell Transplantation
This phase IIa trial studies how well guadecitabine works in treating patients with acute myelogenous leukemia and myelodysplastic syndrome that has returned after a period of improvement after allogeneic stem cell transplant. Guadecitabine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft-versus-host disease). Giving guadecitabine before the transplant may stop this from happening. Once the donated stem cells begin working, the patient's immune system may see the remaining cancer cells as not belonging in the patient's body and destroy them. Giving an infusion of the donor's white blood cells (donor lymphocyte infusion) may boost this effect.
Read the detailed description
PRIMARY OBJECTIVES:
- To determine the complete response (CR) rate of guadecitabine (SGI-110) with or without donor lymphocyte infusion (DLI) either for the treatment of morphologic relapse or the presence of minimal residual disease (MRD) in patients with acute myeloid leukemia or myelodysplastic syndrome after hematopoietic stem cell transplantation in patients with AML and MDS (cohort 1 and 2).
II. The relapse-free survival with the use of SGI-110 as maintenance therapy in patients with high risk acute myeloid leukemia or myelodysplastic syndrome after hematopoietic stem cell transplantation (cohort 3).
SECONDARY OBJECTIVES:
- To determine the safety and toxicity of SGI-110 with or without DLI in this subject population.
II. To evaluate overall response and survival.
OUTLINE:
Patients receive guadecitabine subcutaneously (SC) once daily (QD) on days 1-5. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Patients also receive DLI intravenously (IV) over 10-30 minutes on day 6 of cycles 2, 4, and 6 in the absence of disease progression or unacceptable toxicity.
- Conditions
- Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, Myelodysplastic Syndrome, Recurrent Acute Myeloid Leukemia, Recurrent Myelodysplastic Syndrome
- Being tested
- Donor Lymphocytes, Guadecitabine, Laboratory Biomarker Analysis
- Medical subject headings
- Leukemia, Myeloid, Acute, Leukemia, Myelomonocytic, Chronic, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Donor LymphocytesBiological
Given IV
GuadecitabineDrug
Also called DNMT inhibitor SGI-110, S110, SGI-110
Given SC
Laboratory Biomarker AnalysisOther
Correlative studies
| Study group | Role | What they receive |
|---|---|---|
| Treatment (guadecitabine, DLI) | Experimental | Patients receive guadecitabine SC QD on days 1-5. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Patients also receive DLI IV over 10-30 minutes on day 6 of cycles 2, 4, and 6 in the absence of disease progression or unacceptable toxicity. Receives: Biological: Donor Lymphocytes; Drug: Guadecitabine; Other: Laboratory Biomarker Analysis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Achieved Complete Response (CR)
Complete remission (CR): Bone marrow with </= 5% bone marrow blasts with normal maturation of all cell lines in the absence of extramedullary disease in addition to a peripheral blood granulocyte count >/= 1 X 10^9/L and a platelet count >/= 100 x 10^9 /L.
Measured: Within the first 6 cycles, up to 168 days.
Secondary outcomes (2)
Overall Survival (OS)
Number of participants that are alive 1 year after study enrollment.
Measured: At 1 year
Disease-free Survival
Number of participants that are disease free and alive 1 year after study enrollment.
Measured: At 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2016 (estimated)
- Study start22 Jun 2016
- Primary completion8 Oct 2024
- Study completion8 Oct 2024
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.
- 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
- 55 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Betul Oran, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02684162
- Sponsor's protocol ID
- 2015-0117
- Other IDs
- NCI-2016-00343, 2015-0117
- Status last verified
- Oct 2025
- First submitted
- 12 Feb 2016
- Last updated
- 6 Nov 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Trial to Assess the Efficacy and Toxicity of SGI-110 With DLI for the Treatment of AML or MDS Relapsing After Allogeneic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT02684162
- Acronym / sponsor ID
- 2015-0117
- Conditions
- Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, Myelodysplastic Syndrome, Recurrent Acute Myeloid Leukemia, Recurrent Myelodysplastic Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 55 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 22 Jun 2016 (actual)
- Primary completion
- 8 Oct 2024 (actual)
- Study completion
- 8 Oct 2024 (actual)
- First posted
- 17 Feb 2016 (estimated)
- Results
- Posted 6 Nov 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase IIa trial studies how well guadecitabine works in treating patients with acute myelogenous leukemia and myelodysplastic syndrome that has returned after a period of improvement after allogeneic stem cell transplant. Guadecitabine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft-versus-host disease). Giving guadecitabine before the transplant may stop this from happening. Once the donated stem cells begin working, the patient's immune system may see the remaining cancer cells as not belonging in the patient's body and destroy them. Giving an infusion of the donor's white blood cells (donor lymphocyte infusion) may boost this effect.
Detailed description
PRIMARY OBJECTIVES:
- To determine the complete response (CR) rate of guadecitabine (SGI-110) with or without donor lymphocyte infusion (DLI) either for the treatment of morphologic relapse or the presence of minimal residual disease (MRD) in patients with acute myeloid leukemia or myelodysplastic syndrome after hematopoietic stem cell transplantation in patients with AML and MDS (cohort 1 and 2).
II. The relapse-free survival with the use of SGI-110 as maintenance therapy in patients with high risk acute myeloid leukemia or myelodysplastic syndrome after hematopoietic stem cell transplantation (cohort 3).
SECONDARY OBJECTIVES:
- To determine the safety and toxicity of SGI-110 with or without DLI in this subject population.
II. To evaluate overall response and survival.
OUTLINE:
Patients receive guadecitabine subcutaneously (SC) once daily (QD) on days 1-5. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Patients also receive DLI intravenously (IV) over 10-30 minutes on day 6 of cycles 2, 4, and 6 in the absence of disease progression or unacceptable toxicity.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (guadecitabine, DLI) | Experimental | Biological: Donor Lymphocytes, Drug: Guadecitabine, Other: Laboratory Biomarker AnalysisPatients receive guadecitabine SC QD on days 1-5. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Patients also receive DLI IV over 10-30 minutes on day 6 of cycles 2, 4, and 6 in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (35 listed)
- Diagnosis of acute myeloid leukemia (AML) and myelodysplastic syndrome (including chronic myelomonocytic leukemia [CMML]) according to WHO classification that underwent first allogeneic hematopoietic cell transplant (HSCT) with either peripheral blood or bone marrow as the source of the hematopoietic stem cells
- No more than 1 antigen mismatch at human leukocyte antigen (HLA)-A, B, C, DRB1 and DQB1 locus for either related or unrelated donor
- High risk AML and MDS patients will be included
- Cohort 1: morphological relapse after stem cell transplant:
- Cohort 2: Persistence or reappearance of minimal residual disease by flow cytometry or cytogenetic or molecular testing while being in morphological remission after allogeneic stem cell transplantation
Key exclusion criteria (13 listed)
- Use of any anti-leukemic agents after relapse is documented (note that the use of these anti-leukemic agents given as post-transplant maintenance therapy is allowed in this study, e.g., subcutaneous or oral 5-azacytidine or FLT3 inhibitors for maintenance) for cohorts 1 and 2
- Bone marrow blast count > 60% for cohort 1
- Use of any of the following after transplantation and prior to starting study therapy for cohort 3:
- Active acute graft versus host disease (GVHD) grade II or higher
- Active chronic GVHD that is extensive
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 |
|---|---|---|
| Donor LymphocytesGiven IV | Biological | Treatment (guadecitabine, DLI) |
| GuadecitabineAlso: DNMT inhibitor SGI-110, S110, SGI-110Given SC | Drug | Treatment (guadecitabine, DLI) |
| Laboratory Biomarker AnalysisCorrelative studies | Other | Treatment (guadecitabine, DLI) |
Primary outcome
Number of Participants Achieved Complete Response (CR)
Time frame: Within the first 6 cycles, up to 168 days.
Complete remission (CR): Bone marrow with </= 5% bone marrow blasts with normal maturation of all cell lines in the absence of extramedullary disease in addition to a peripheral blood granulocyte count >/= 1 X 10^9/L and a platelet count >/= 100 x 10^9 /L.
2 secondary outcomes
Overall Survival (OS)
Time frame: At 1 year
Disease-free Survival
Time frame: At 1 year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Nov 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- MD Anderson Cancer CenterLink provided by the sponsor.
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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.
Betul Oran, 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 Donor Lymphocytes, Guadecitabine and Laboratory Biomarker Analysis in people with Acute Myeloid Leukemia 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 35 inclusion and 13 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 6 Nov 2025. 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 6 Nov 2025). 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?
Number of Participants Achieved Complete Response (CR) (time frame: Within the first 6 cycles, up to 168 days.). 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 6 Nov 2025.