Trial Outcomes & Findings for Guadecitabine and Donor Lymphocyte Infusion in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome Relapsing After Allogeneic Stem Cell Transplant (NCT NCT02684162)
NCT ID: NCT02684162
Last Updated: 2025-11-06
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
55 participants
Primary outcome timeframe
Within the first 6 cycles, up to 168 days.
Results posted on
2025-11-06
Participant Flow
All participants were registered at MD Anderson Cancer Center.
Participant milestones
| Measure |
Cohort 1: Relapsed Leukemia Post-transplant
Patients with morphological relapse for AML and MDS at least 90 days post-transplant.
MDS patients: Re-appearance of dysplastic changes in the bone marrow, with or without increase in bone marrow blast count, which is pathologically consistent with myelodysplastic syndrome.
AML patients:
Bone marrow blast count \>/= 5%.
|
Cohort 2: MRD(Minimal Residual Disease) Post-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.
|
Cohort 3: Maintenance Treatment Post-transplant
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.
|
|---|---|---|---|
|
Overall Study
STARTED
|
13
|
18
|
24
|
|
Overall Study
COMPLETED
|
13
|
17
|
24
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
0
|
Reasons for withdrawal
| Measure |
Cohort 1: Relapsed Leukemia Post-transplant
Patients with morphological relapse for AML and MDS at least 90 days post-transplant.
MDS patients: Re-appearance of dysplastic changes in the bone marrow, with or without increase in bone marrow blast count, which is pathologically consistent with myelodysplastic syndrome.
AML patients:
Bone marrow blast count \>/= 5%.
|
Cohort 2: MRD(Minimal Residual Disease) Post-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.
|
Cohort 3: Maintenance Treatment Post-transplant
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.
|
|---|---|---|---|
|
Overall Study
Physician Decision
|
0
|
1
|
0
|
Baseline Characteristics
Guadecitabine and Donor Lymphocyte Infusion in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome Relapsing After Allogeneic Stem Cell Transplant
Baseline characteristics by cohort
| Measure |
Cohort 1: Relapsed Leukemia Post-transplant
n=13 Participants
Patients with morphological relapse for AML and MDS at least 90 days post-transplant.
MDS patients: Re-appearance of dysplastic changes in the bone marrow, with or without increase in bone marrow blast count, which is pathologically consistent with myelodysplastic syndrome.
AML patients:
Bone marrow blast count \>/= 5%.
|
Cohort 2: MRD(Minimal Residual Disease) Post-transplant
n=18 Participants
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: Maintenance Treatment Post-transplant
n=24 Participants
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.
|
Total
n=55 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=49 Participants
|
0 Participants
n=50 Participants
|
0 Participants
n=50 Participants
|
0 Participants
n=50 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
8 Participants
n=49 Participants
|
10 Participants
n=50 Participants
|
13 Participants
n=50 Participants
|
31 Participants
n=50 Participants
|
|
Age, Categorical
>=65 years
|
5 Participants
n=49 Participants
|
8 Participants
n=50 Participants
|
11 Participants
n=50 Participants
|
24 Participants
n=50 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=49 Participants
|
11 Participants
n=50 Participants
|
11 Participants
n=50 Participants
|
27 Participants
n=50 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=49 Participants
|
7 Participants
n=50 Participants
|
13 Participants
n=50 Participants
|
28 Participants
n=50 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
4 Participants
n=49 Participants
|
1 Participants
n=50 Participants
|
2 Participants
n=50 Participants
|
7 Participants
n=50 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
9 Participants
n=49 Participants
|
17 Participants
n=50 Participants
|
22 Participants
n=50 Participants
|
48 Participants
n=50 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=49 Participants
|
0 Participants
n=50 Participants
|
0 Participants
n=50 Participants
|
0 Participants
n=50 Participants
|
|
Region of Enrollment
United States
|
13 participants
n=49 Participants
|
18 participants
n=50 Participants
|
24 participants
n=50 Participants
|
55 participants
n=50 Participants
|
PRIMARY outcome
Timeframe: 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.
Outcome measures
| Measure |
Cohort 1: Relapsed Leukemia Post-transplant
n=13 Participants
Patients with morphological relapse for AML and MDS at least 90 days post-transplant.
MDS patients: Re-appearance of dysplastic changes in the bone marrow, with or without increase in bone marrow blast count, which is pathologically consistent with myelodysplastic syndrome.
AML patients:
Bone marrow blast count \>/= 5%.
|
Cohort 2: MRD(Minimal Residual Disease) Post-transplant
n=18 Participants
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: Maintenance Treatment Post-transplant
n=24 Participants
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.
|
|---|---|---|---|
|
Number of Participants Achieved Complete Response (CR)
|
3 Participants
|
9 Participants
|
16 Participants
|
SECONDARY outcome
Timeframe: At 1 yearNumber of participants that are alive 1 year after study enrollment.
Outcome measures
| Measure |
Cohort 1: Relapsed Leukemia Post-transplant
n=13 Participants
Patients with morphological relapse for AML and MDS at least 90 days post-transplant.
MDS patients: Re-appearance of dysplastic changes in the bone marrow, with or without increase in bone marrow blast count, which is pathologically consistent with myelodysplastic syndrome.
AML patients:
Bone marrow blast count \>/= 5%.
|
Cohort 2: MRD(Minimal Residual Disease) Post-transplant
n=18 Participants
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: Maintenance Treatment Post-transplant
n=24 Participants
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.
|
|---|---|---|---|
|
Overall Survival (OS)
|
12 Participants
|
12 Participants
|
10 Participants
|
SECONDARY outcome
Timeframe: At 1 yearNumber of participants that are disease free and alive 1 year after study enrollment.
Outcome measures
| Measure |
Cohort 1: Relapsed Leukemia Post-transplant
n=13 Participants
Patients with morphological relapse for AML and MDS at least 90 days post-transplant.
MDS patients: Re-appearance of dysplastic changes in the bone marrow, with or without increase in bone marrow blast count, which is pathologically consistent with myelodysplastic syndrome.
AML patients:
Bone marrow blast count \>/= 5%.
|
Cohort 2: MRD(Minimal Residual Disease) Post-transplant
n=18 Participants
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: Maintenance Treatment Post-transplant
n=24 Participants
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.
|
|---|---|---|---|
|
Disease-free Survival
|
5 Participants
|
7 Participants
|
20 Participants
|
Adverse Events
Cohort 1: Relapsed Leukemia Post-transplant
Serious events: 9 serious events
Other events: 3 other events
Deaths: 1 deaths
Cohort 2: MRD(Minimal Residual Disease) Post-transplant
Serious events: 11 serious events
Other events: 8 other events
Deaths: 6 deaths
Cohort 3: Maintenance Treatment Post-transplant
Serious events: 12 serious events
Other events: 10 other events
Deaths: 14 deaths
Serious adverse events
| Measure |
Cohort 1: Relapsed Leukemia Post-transplant
n=13 participants at risk
Patients with morphological relapse for AML and MDS at least 90 days post-transplant.
MDS patients: Re-appearance of dysplastic changes in the bone marrow, with or without increase in bone marrow blast count, which is pathologically consistent with myelodysplastic syndrome.
AML patients:
Bone marrow blast count \>/= 5%.
|
Cohort 2: MRD(Minimal Residual Disease) Post-transplant
n=18 participants at risk
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: Maintenance Treatment Post-transplant
n=24 participants at risk
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.
|
|---|---|---|---|
|
Gastrointestinal disorders
Diarrhea
|
7.7%
1/13 • Number of events 1 • 1 year
|
0.00%
0/18 • 1 year
|
0.00%
0/24 • 1 year
|
|
Investigations
Neutrophil count decreased
|
69.2%
9/13 • Number of events 10 • 1 year
|
61.1%
11/18 • Number of events 41 • 1 year
|
50.0%
12/24 • Number of events 50 • 1 year
|
|
Investigations
Platelet count decreased
|
46.2%
6/13 • Number of events 6 • 1 year
|
33.3%
6/18 • Number of events 13 • 1 year
|
25.0%
6/24 • Number of events 6 • 1 year
|
|
Infections and infestations
Sepsis
|
7.7%
1/13 • Number of events 1 • 1 year
|
11.1%
2/18 • Number of events 2 • 1 year
|
0.00%
0/24 • 1 year
|
|
Investigations
White blood cell decreased
|
46.2%
6/13 • Number of events 6 • 1 year
|
55.6%
10/18 • Number of events 13 • 1 year
|
37.5%
9/24 • Number of events 12 • 1 year
|
|
Blood and lymphatic system disorders
Anemia
|
0.00%
0/13 • 1 year
|
5.6%
1/18 • Number of events 2 • 1 year
|
0.00%
0/24 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
0.00%
0/13 • 1 year
|
5.6%
1/18 • Number of events 1 • 1 year
|
0.00%
0/24 • 1 year
|
|
Infections and infestations
Upper Respiratory Infection
|
0.00%
0/13 • 1 year
|
5.6%
1/18 • Number of events 1 • 1 year
|
0.00%
0/24 • 1 year
|
Other adverse events
| Measure |
Cohort 1: Relapsed Leukemia Post-transplant
n=13 participants at risk
Patients with morphological relapse for AML and MDS at least 90 days post-transplant.
MDS patients: Re-appearance of dysplastic changes in the bone marrow, with or without increase in bone marrow blast count, which is pathologically consistent with myelodysplastic syndrome.
AML patients:
Bone marrow blast count \>/= 5%.
|
Cohort 2: MRD(Minimal Residual Disease) Post-transplant
n=18 participants at risk
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: Maintenance Treatment Post-transplant
n=24 participants at risk
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.
|
|---|---|---|---|
|
Investigations
Alanine aminotransferase increased
|
7.7%
1/13 • Number of events 1 • 1 year
|
11.1%
2/18 • Number of events 2 • 1 year
|
8.3%
2/24 • Number of events 7 • 1 year
|
|
Blood and lymphatic system disorders
Anemia
|
23.1%
3/13 • Number of events 6 • 1 year
|
38.9%
7/18 • Number of events 14 • 1 year
|
20.8%
5/24 • Number of events 7 • 1 year
|
|
Investigations
Blood bilirubin increased
|
7.7%
1/13 • Number of events 1 • 1 year
|
0.00%
0/18 • 1 year
|
0.00%
0/24 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
7.7%
1/13 • Number of events 1 • 1 year
|
16.7%
3/18 • Number of events 5 • 1 year
|
4.2%
1/24 • Number of events 1 • 1 year
|
|
General disorders
Fatigue
|
7.7%
1/13 • Number of events 1 • 1 year
|
16.7%
3/18 • Number of events 3 • 1 year
|
12.5%
3/24 • Number of events 3 • 1 year
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
15.4%
2/13 • Number of events 7 • 1 year
|
16.7%
3/18 • Number of events 3 • 1 year
|
8.3%
2/24 • Number of events 2 • 1 year
|
|
Infections and infestations
Gallbladder infection
|
7.7%
1/13 • Number of events 1 • 1 year
|
0.00%
0/18 • 1 year
|
0.00%
0/24 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
7.7%
1/13 • Number of events 1 • 1 year
|
0.00%
0/18 • 1 year
|
0.00%
0/24 • 1 year
|
|
Vascular disorders
Hypotension
|
7.7%
1/13 • Number of events 1 • 1 year
|
5.6%
1/18 • Number of events 1 • 1 year
|
0.00%
0/24 • 1 year
|
|
General disorders
Injection site reaction
|
7.7%
1/13 • Number of events 1 • 1 year
|
0.00%
0/18 • 1 year
|
0.00%
0/24 • 1 year
|
|
Infections and infestations
Lung infection
|
7.7%
1/13 • Number of events 1 • 1 year
|
5.6%
1/18 • Number of events 1 • 1 year
|
20.8%
5/24 • Number of events 7 • 1 year
|
|
Gastrointestinal disorders
Nausea
|
15.4%
2/13 • Number of events 5 • 1 year
|
27.8%
5/18 • Number of events 5 • 1 year
|
8.3%
2/24 • Number of events 2 • 1 year
|
|
Investigations
Neutrophil count decreased
|
15.4%
2/13 • Number of events 2 • 1 year
|
27.8%
5/18 • Number of events 48 • 1 year
|
37.5%
9/24 • Number of events 56 • 1 year
|
|
Investigations
Platelet count decreased
|
15.4%
2/13 • Number of events 8 • 1 year
|
44.4%
8/18 • Number of events 70 • 1 year
|
41.7%
10/24 • Number of events 76 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary edema
|
7.7%
1/13 • Number of events 1 • 1 year
|
0.00%
0/18 • 1 year
|
0.00%
0/24 • 1 year
|
|
Nervous system disorders
Syncope
|
7.7%
1/13 • Number of events 1 • 1 year
|
0.00%
0/18 • 1 year
|
0.00%
0/24 • 1 year
|
|
Infections and infestations
Upper respiratory infection
|
7.7%
1/13 • Number of events 1 • 1 year
|
11.1%
2/18 • Number of events 3 • 1 year
|
16.7%
4/24 • Number of events 5 • 1 year
|
|
Infections and infestations
Urinary tract infection
|
7.7%
1/13 • Number of events 1 • 1 year
|
5.6%
1/18 • Number of events 1 • 1 year
|
4.2%
1/24 • Number of events 1 • 1 year
|
|
Investigations
White blood cell decreased
|
7.7%
1/13 • Number of events 6 • 1 year
|
38.9%
7/18 • Number of events 65 • 1 year
|
37.5%
9/24 • Number of events 164 • 1 year
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/13 • 1 year
|
5.6%
1/18 • Number of events 1 • 1 year
|
0.00%
0/24 • 1 year
|
|
Metabolism and nutrition disorders
Anorexia
|
0.00%
0/13 • 1 year
|
5.6%
1/18 • Number of events 1 • 1 year
|
0.00%
0/24 • 1 year
|
|
Eye disorders
Blurred Vision
|
0.00%
0/13 • 1 year
|
11.1%
2/18 • Number of events 2 • 1 year
|
0.00%
0/24 • 1 year
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/13 • 1 year
|
5.6%
1/18 • Number of events 1 • 1 year
|
8.3%
2/24 • Number of events 2 • 1 year
|
|
General disorders
Edema limbs
|
0.00%
0/13 • 1 year
|
5.6%
1/18 • Number of events 1 • 1 year
|
0.00%
0/24 • 1 year
|
|
General disorders
Fever
|
0.00%
0/13 • 1 year
|
16.7%
3/18 • Number of events 3 • 1 year
|
0.00%
0/24 • 1 year
|
|
Gastrointestinal disorders
Gastroparesis
|
0.00%
0/13 • 1 year
|
5.6%
1/18 • Number of events 1 • 1 year
|
0.00%
0/24 • 1 year
|
|
Nervous system disorders
Headache
|
0.00%
0/13 • 1 year
|
5.6%
1/18 • Number of events 1 • 1 year
|
0.00%
0/24 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
0.00%
0/13 • 1 year
|
5.6%
1/18 • Number of events 1 • 1 year
|
0.00%
0/24 • 1 year
|
|
Cardiac disorders
Sinus tachycardia
|
0.00%
0/13 • 1 year
|
5.6%
1/18 • Number of events 1 • 1 year
|
0.00%
0/24 • 1 year
|
|
Gastrointestinal disorders
Vomitting
|
0.00%
0/13 • 1 year
|
11.1%
2/18 • Number of events 3 • 1 year
|
4.2%
1/24 • Number of events 1 • 1 year
|
|
Investigations
Creatinine increased
|
0.00%
0/13 • 1 year
|
0.00%
0/18 • 1 year
|
8.3%
2/24 • Number of events 9 • 1 year
|
|
Nervous system disorders
Dizziness
|
0.00%
0/13 • 1 year
|
0.00%
0/18 • 1 year
|
4.2%
1/24 • Number of events 1 • 1 year
|
|
Infections and infestations
Infections and infestations
|
0.00%
0/13 • 1 year
|
0.00%
0/18 • 1 year
|
12.5%
3/24 • Number of events 3 • 1 year
|
|
General disorders
Localized edema
|
0.00%
0/13 • 1 year
|
0.00%
0/18 • 1 year
|
4.2%
1/24 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
0.00%
0/13 • 1 year
|
0.00%
0/18 • 1 year
|
4.2%
1/24 • Number of events 1 • 1 year
|
|
Infections and infestations
Otitis externa
|
0.00%
0/13 • 1 year
|
0.00%
0/18 • 1 year
|
4.2%
1/24 • Number of events 1 • 1 year
|
|
Infections and infestations
Otitis media
|
0.00%
0/13 • 1 year
|
0.00%
0/18 • 1 year
|
4.2%
1/24 • Number of events 1 • 1 year
|
|
Skin and subcutaneous tissue disorders
Pain of skin
|
0.00%
0/13 • 1 year
|
0.00%
0/18 • 1 year
|
4.2%
1/24 • Number of events 1 • 1 year
|
|
Cardiac disorders
Pericardial effusion
|
0.00%
0/13 • 1 year
|
0.00%
0/18 • 1 year
|
4.2%
1/24 • Number of events 1 • 1 year
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
0.00%
0/13 • 1 year
|
0.00%
0/18 • 1 year
|
4.2%
1/24 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
0.00%
0/13 • 1 year
|
0.00%
0/18 • 1 year
|
4.2%
1/24 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
Stomach pain
|
0.00%
0/13 • 1 year
|
0.00%
0/18 • 1 year
|
4.2%
1/24 • Number of events 1 • 1 year
|
Additional Information
Betul Oran, MD / Stem Cell Transplantation
The University of Texas MD Anderson Cancer Center
Phone: 713-745-2820
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place