Trial Outcomes & Findings for Guadecitabine in Treating Patients With Higher-Risk Myelodysplastic Syndromes (NCT NCT02131597)
NCT ID: NCT02131597
Last Updated: 2025-07-17
Results Overview
Complete Response is Normalization of the peripheral blood and bone marrow with \</= 5% bone marrow blasts, a peripheral blood granulocyte count \>/= 1.0 x 10\^9/L, and a platelet count \>/= 100 x 10\^9/L. Hemoglobin \>/= 11 g/dL at any point while on treatment after the first cycle of therapy.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
100 participants
Primary outcome timeframe
At cycle 3 and cycle 6, cycles are up every 4 to 8 weeks. Up to 9 years, 8 months and 15 days.
Results posted on
2025-07-17
Participant Flow
Participant milestones
| Measure |
Treatment (Guadecitabine)
Patients receive guadecitabine SC on days 1-5. Treatment repeats every 4-8 weeks for up to 24 courses in the absence of disease progression or unacceptable toxicity. Patients with stable disease after 3 courses are taken off therapy after 6 courses. Patients may continue to receive treatment after 24 courses if the investigator determines it is in the patient's best interest.
Guadecitabine: Given SC
|
|---|---|
|
Overall Study
STARTED
|
100
|
|
Overall Study
COMPLETED
|
100
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Guadecitabine in Treating Patients With Higher-Risk Myelodysplastic Syndromes
Baseline characteristics by cohort
| Measure |
Treatment (Guadecitabine)
n=100 Participants
Patients receive guadecitabine SC on days 1-5. Treatment repeats every 4-8 weeks for up to 24 courses in the absence of disease progression or unacceptable toxicity. Patients with stable disease after 3 courses are taken off therapy after 6 courses. Patients may continue to receive treatment after 24 courses if the investigator determines it is in the patient's best interest.
Guadecitabine: Given SC
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
34 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
66 Participants
n=5 Participants
|
|
Age, Continuous
|
69 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
38 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
62 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
87 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
10 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
100 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: At cycle 3 and cycle 6, cycles are up every 4 to 8 weeks. Up to 9 years, 8 months and 15 days.Complete Response is Normalization of the peripheral blood and bone marrow with \</= 5% bone marrow blasts, a peripheral blood granulocyte count \>/= 1.0 x 10\^9/L, and a platelet count \>/= 100 x 10\^9/L. Hemoglobin \>/= 11 g/dL at any point while on treatment after the first cycle of therapy.
Outcome measures
| Measure |
Treatment (Guadecitabine)
n=100 Participants
Patients receive guadecitabine SC on days 1-5. Treatment repeats every 4-8 weeks for up to 24 courses in the absence of disease progression or unacceptable toxicity. Patients with stable disease after 3 courses are taken off therapy after 6 courses. Patients may continue to receive treatment after 24 courses if the investigator determines it is in the patient's best interest.
Guadecitabine: Given SC
|
|---|---|
|
Number of Participants With a Complete Response (CR)
|
25 Participants
|
SECONDARY outcome
Timeframe: Up to 9 years, 8 months and 15 daysTime from date of treatment start until date of death due to any cause or last Follow-up.
Outcome measures
| Measure |
Treatment (Guadecitabine)
n=100 Participants
Patients receive guadecitabine SC on days 1-5. Treatment repeats every 4-8 weeks for up to 24 courses in the absence of disease progression or unacceptable toxicity. Patients with stable disease after 3 courses are taken off therapy after 6 courses. Patients may continue to receive treatment after 24 courses if the investigator determines it is in the patient's best interest.
Guadecitabine: Given SC
|
|---|---|
|
Overall Survival
|
16.9 Months
Interval 1.0 to 59.3
|
SECONDARY outcome
Timeframe: Up to 9 years, 8 months and 15 daysOutcome measures
| Measure |
Treatment (Guadecitabine)
n=100 Participants
Patients receive guadecitabine SC on days 1-5. Treatment repeats every 4-8 weeks for up to 24 courses in the absence of disease progression or unacceptable toxicity. Patients with stable disease after 3 courses are taken off therapy after 6 courses. Patients may continue to receive treatment after 24 courses if the investigator determines it is in the patient's best interest.
Guadecitabine: Given SC
|
|---|---|
|
Time to Acute Myeloid Leukemia (AML) Transformation
|
27.3 Months
Interval 0.96 to 69.9
|
Adverse Events
Treatment (Guadecitabine)
Serious events: 76 serious events
Other events: 93 other events
Deaths: 14 deaths
Serious adverse events
| Measure |
Treatment (Guadecitabine)
n=100 participants at risk
Patients receive guadecitabine SC on days 1-5. Treatment repeats every 4-8 weeks for up to 24 courses in the absence of disease progression or unacceptable toxicity. Patients with stable disease after 3 courses are taken off therapy after 6 courses. Patients may continue to receive treatment after 24 courses if the investigator determines it is in the patient's best interest.
Guadecitabine: Given SC
|
|---|---|
|
General disorders
Abdominal pain
|
3.0%
3/100 • Number of events 3 • Up to 9 years, 8 months and 15 days
|
|
Hepatobiliary disorders
Acute Cholecystitis
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
General disorders
Acute coronary syndrome
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Renal and urinary disorders
Acute kidney injury
|
4.0%
4/100 • Number of events 5 • Up to 9 years, 8 months and 15 days
|
|
Blood and lymphatic system disorders
Anemia
|
2.0%
2/100 • Number of events 2 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Anorectal infection
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Cardiac disorders
Atrial fibrillation
|
2.0%
2/100 • Number of events 3 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Bacteremia
|
10.0%
10/100 • Number of events 12 • Up to 9 years, 8 months and 15 days
|
|
Cardiac disorders
Cardiac arrest
|
2.0%
2/100 • Number of events 2 • Up to 9 years, 8 months and 15 days
|
|
Cardiac disorders
Cardiopulomary Arrest
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Cellulitis
|
6.0%
6/100 • Number of events 6 • Up to 9 years, 8 months and 15 days
|
|
Cardiac disorders
Chest pain - cardiac
|
2.0%
2/100 • Number of events 2 • Up to 9 years, 8 months and 15 days
|
|
Hepatobiliary disorders
Cholecystitis
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Clostridium difficile
|
2.0%
2/100 • Number of events 2 • Up to 9 years, 8 months and 15 days
|
|
Gastrointestinal disorders
Colitis
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Eye disorders
Conjunctivitis
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Metabolism and nutrition disorders
Dehydration
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Device-related infection
|
3.0%
3/100 • Number of events 3 • Up to 9 years, 8 months and 15 days
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
3.0%
3/100 • Number of events 4 • Up to 9 years, 8 months and 15 days
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
2.0%
2/100 • Number of events 2 • Up to 9 years, 8 months and 15 days
|
|
Injury, poisoning and procedural complications
Fall
|
3.0%
3/100 • Number of events 3 • Up to 9 years, 8 months and 15 days
|
|
General disorders
Fatigue
|
2.0%
2/100 • Number of events 2 • Up to 9 years, 8 months and 15 days
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
28.0%
28/100 • Number of events 30 • Up to 9 years, 8 months and 15 days
|
|
General disorders
Flu-like symptoms
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Fungal Pneumonia
|
3.0%
3/100 • Number of events 3 • Up to 9 years, 8 months and 15 days
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Nervous system disorders
Headache
|
3.0%
3/100 • Number of events 3 • Up to 9 years, 8 months and 15 days
|
|
Cardiac disorders
Heart Failure
|
2.0%
2/100 • Number of events 2 • Up to 9 years, 8 months and 15 days
|
|
Vascular disorders
Hematoma
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Renal and urinary disorders
Hematuria
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Hepatic infection
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Injury, poisoning and procedural complications
Hip fracture
|
2.0%
2/100 • Number of events 2 • Up to 9 years, 8 months and 15 days
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Vascular disorders
Hypotension
|
2.0%
2/100 • Number of events 2 • Up to 9 years, 8 months and 15 days
|
|
Injury, poisoning and procedural complications
Infusion related reaction
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Blood and lymphatic system disorders
Leukocytosis
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Lung infection
|
5.0%
5/100 • Number of events 6 • Up to 9 years, 8 months and 15 days
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Myelodysplastic syndrome
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, malignant and unspecified
|
4.0%
4/100 • Number of events 4 • Up to 9 years, 8 months and 15 days
|
|
Cardiac disorders
Non-cardiac chest pain
|
2.0%
2/100 • Number of events 2 • Up to 9 years, 8 months and 15 days
|
|
Gastrointestinal disorders
Oral hemorrhage
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
General disorders
Pain
|
2.0%
2/100 • Number of events 2 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Penumonia
|
10.0%
10/100 • Number of events 11 • Up to 9 years, 8 months and 15 days
|
|
Vascular disorders
Peripheral vascular occlusive disease
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Pharyngitis
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Phlebitis infective
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Investigations
Platelet count decreased
|
2.0%
2/100 • Number of events 2 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Pneumonia
|
9.0%
9/100 • Number of events 12 • Up to 9 years, 8 months and 15 days
|
|
Eye disorders
Retinal vascular disorder
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Sepsis
|
6.0%
6/100 • Number of events 8 • Up to 9 years, 8 months and 15 days
|
|
Cardiac disorders
Sinus bradycardia
|
1.0%
1/100 • Number of events 2 • Up to 9 years, 8 months and 15 days
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Gastrointestinal disorders
Small intestinal obstruction
|
2.0%
2/100 • Number of events 3 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Soft tissue infection
|
3.0%
3/100 • Number of events 3 • Up to 9 years, 8 months and 15 days
|
|
Nervous system disorders
Stroke
|
2.0%
2/100 • Number of events 2 • Up to 9 years, 8 months and 15 days
|
|
Nervous system disorders
Syncope
|
3.0%
3/100 • Number of events 6 • Up to 9 years, 8 months and 15 days
|
|
Vascular disorders
Thromboembolic Event
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Tooth infection
|
2.0%
2/100 • Number of events 2 • Up to 9 years, 8 months and 15 days
|
|
Nervous system disorders
Transient ischemic attacks
|
2.0%
2/100 • Number of events 2 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Upper respiratory infection
|
3.0%
3/100 • Number of events 3 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Urinary tract infection
|
7.0%
7/100 • Number of events 7 • Up to 9 years, 8 months and 15 days
|
|
Renal and urinary disorders
Urinary tract obstruction
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Diverticulitis
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Gastrointestinal disorders
Gastrointestinal hemorrhage
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Gastrointestinal disorders
GI perforation
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
muscle abscess
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Fungal sinusitis
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Bronchiolitis
|
1.0%
1/100 • Number of events 1 • Up to 9 years, 8 months and 15 days
|
Other adverse events
| Measure |
Treatment (Guadecitabine)
n=100 participants at risk
Patients receive guadecitabine SC on days 1-5. Treatment repeats every 4-8 weeks for up to 24 courses in the absence of disease progression or unacceptable toxicity. Patients with stable disease after 3 courses are taken off therapy after 6 courses. Patients may continue to receive treatment after 24 courses if the investigator determines it is in the patient's best interest.
Guadecitabine: Given SC
|
|---|---|
|
Infections and infestations
Upper respiratory infection
|
5.0%
5/100 • Number of events 5 • Up to 9 years, 8 months and 15 days
|
|
Injury, poisoning and procedural complications
Fall
|
5.0%
5/100 • Number of events 6 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Skin infection
|
5.0%
5/100 • Number of events 6 • Up to 9 years, 8 months and 15 days
|
|
Blood and lymphatic system disorders
Anemia
|
7.0%
7/100 • Number of events 7 • Up to 9 years, 8 months and 15 days
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
5.0%
5/100 • Number of events 7 • Up to 9 years, 8 months and 15 days
|
|
General disorders
Fever
|
7.0%
7/100 • Number of events 7 • Up to 9 years, 8 months and 15 days
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
6.0%
6/100 • Number of events 7 • Up to 9 years, 8 months and 15 days
|
|
Vascular disorders
Hypotension
|
7.0%
7/100 • Number of events 7 • Up to 9 years, 8 months and 15 days
|
|
General disorders
Pain
|
5.0%
5/100 • Number of events 8 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Urinary tract infection
|
8.0%
8/100 • Number of events 9 • Up to 9 years, 8 months and 15 days
|
|
General disorders
Edema limbs
|
5.0%
5/100 • Number of events 10 • Up to 9 years, 8 months and 15 days
|
|
Nervous system disorders
Dizziness
|
7.0%
7/100 • Number of events 11 • Up to 9 years, 8 months and 15 days
|
|
Investigations
Neutrophil count decreased
|
9.0%
9/100 • Number of events 11 • Up to 9 years, 8 months and 15 days
|
|
Nervous system disorders
Headache
|
8.0%
8/100 • Number of events 12 • Up to 9 years, 8 months and 15 days
|
|
Gastrointestinal disorders
Vomiting
|
11.0%
11/100 • Number of events 13 • Up to 9 years, 8 months and 15 days
|
|
Gastrointestinal disorders
Diarrhea
|
15.0%
15/100 • Number of events 15 • Up to 9 years, 8 months and 15 days
|
|
Investigations
White blood cell decreased
|
9.0%
9/100 • Number of events 16 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Infections and infestations
|
15.0%
15/100 • Number of events 18 • Up to 9 years, 8 months and 15 days
|
|
Gastrointestinal disorders
Constipation
|
17.0%
17/100 • Number of events 23 • Up to 9 years, 8 months and 15 days
|
|
Gastrointestinal disorders
Mucositis oral
|
17.0%
17/100 • Number of events 25 • Up to 9 years, 8 months and 15 days
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
20.0%
20/100 • Number of events 30 • Up to 9 years, 8 months and 15 days
|
|
General disorders
Injection site reaction
|
19.0%
19/100 • Number of events 30 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Platelet count decreased
|
14.0%
14/100 • Number of events 30 • Up to 9 years, 8 months and 15 days
|
|
Infections and infestations
Lung infection
|
28.0%
28/100 • Number of events 34 • Up to 9 years, 8 months and 15 days
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
28.0%
28/100 • Number of events 35 • Up to 9 years, 8 months and 15 days
|
|
Gastrointestinal disorders
Nausea
|
30.0%
30/100 • Number of events 40 • Up to 9 years, 8 months and 15 days
|
|
General disorders
Fatigue
|
52.0%
52/100 • Number of events 105 • Up to 9 years, 8 months and 15 days
|
Additional Information
Guillermo Garcia-Manero, MD/ Professor
The University of Texas MD Anderson Cancer Center
Phone: 713-745-3428
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place