Trial Outcomes & Findings for Azacitidine After Allo Blood And Marrow Transplantation (BMT) for Chronic Myelogenous Leukemia (CML) (NCT NCT00813124)
NCT ID: NCT00813124
Last Updated: 2021-02-11
Results Overview
Molecular Remission defined as two consecutive bone marrow samples done one month apart with negative PCR( polymerase chain reaction) tor CML.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
24 participants
Primary outcome timeframe
12 month post BMT
Results posted on
2021-02-11
Participant Flow
Recruitment Period: December 17, 2008 to February 27, 2014. All recruitment done at The University of Texas (UT) MD Anderson Cancer Center.
Participant milestones
| Measure |
Azacitidine Post Transplant
Fludarabine 40 mg/m\^2 intravenous (IV) Day -5 to Day -2; Busulfan dose calculated to achieve area under curve (AUC) of 4000 µMol-min + 12% based on pharmacokinetic studies (days -5, -4, -3, and -2); Thymoglobulin: 2.5 mg/kg IV on Day -3 to Day -1; Azacitidine 32 mg/m\^2 subcutaneous daily for 5 days starting 5 weeks after transplant, repeated monthly up to 4 months. Allogeneic stem cell infusion (allotx) on day 0.
|
|---|---|
|
Overall Study
STARTED
|
24
|
|
Overall Study
COMPLETED
|
12
|
|
Overall Study
NOT COMPLETED
|
12
|
Reasons for withdrawal
| Measure |
Azacitidine Post Transplant
Fludarabine 40 mg/m\^2 intravenous (IV) Day -5 to Day -2; Busulfan dose calculated to achieve area under curve (AUC) of 4000 µMol-min + 12% based on pharmacokinetic studies (days -5, -4, -3, and -2); Thymoglobulin: 2.5 mg/kg IV on Day -3 to Day -1; Azacitidine 32 mg/m\^2 subcutaneous daily for 5 days starting 5 weeks after transplant, repeated monthly up to 4 months. Allogeneic stem cell infusion (allotx) on day 0.
|
|---|---|
|
Overall Study
Death
|
2
|
|
Overall Study
Ineligible - Participants did not received Azacytidine
|
9
|
|
Overall Study
Withdrawal by Subject
|
1
|
Baseline Characteristics
Azacitidine After Allo Blood And Marrow Transplantation (BMT) for Chronic Myelogenous Leukemia (CML)
Baseline characteristics by cohort
| Measure |
Azacytidine Maintenance After Allotx
n=24 Participants
Fludarabine 40 mg/m\^2 intravenous (IV) Day -5 to Day -2; Busulfan dose calculated to achieve area under curve (AUC) of 4000 µMol-min + 12% based on pharmacokinetic studies (days -5, -4, -3, and -2); Thymoglobulin: 2.5 mg/kg IV on Day -3 to Day -1; Azacitidine 32 mg/m\^2 subcutaneous daily for 5 days starting 5 weeks after transplant, repeated monthly up to 4 months. Allogeneic stem cell infusion (allotx) on day 0.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
23 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
20 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
24 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: 12 month post BMTMolecular Remission defined as two consecutive bone marrow samples done one month apart with negative PCR( polymerase chain reaction) tor CML.
Outcome measures
| Measure |
Azacytidine Maintenance After Allotx
n=12 Participants
Fludarabine 40 mg/m\^2 intravenous (IV) Day -5 to Day -2; Busulfan dose calculated to achieve area under curve (AUC) of 4000 µMol-min + 12% based on pharmacokinetic studies (days -5, -4, -3, and -2); Thymoglobulin: 2.5 mg/kg IV on Day -3 to Day -1; Azacitidine 32 mg/m\^2 subcutaneous daily for 5 days starting 5 weeks after transplant, repeated monthly up to 4 months. Allogeneic stem cell infusion (allotx) on day 0.
|
|---|---|
|
Number of Participants With Molecular Response
Complete Response
|
8 Participants
|
|
Number of Participants With Molecular Response
Partial Response
|
0 Participants
|
|
Number of Participants With Molecular Response
Progressive Disease
|
4 Participants
|
Adverse Events
Azacytidine Maintenance After Allotx
Serious events: 14 serious events
Other events: 24 other events
Deaths: 2 deaths
Serious adverse events
| Measure |
Azacytidine Maintenance After Allotx
n=24 participants at risk
Fludarabine 40 mg/m\^2 intravenous (IV) Day -5 to Day -2; Busulfan dose calculated to achieve area under curve (AUC) of 4000 µMol-min + 12% based on pharmacokinetic studies (days -5, -4, -3, and -2); Thymoglobulin: 2.5 mg/kg IV on Day -3 to Day -1; Azacitidine 32 mg/m\^2 subcutaneous daily for 5 days starting 5 weeks after transplant, repeated monthly up to 4 months. Allogeneic stem cell infusion (allotx) on day 0.
|
|---|---|
|
Investigations
ALT increased
|
8.3%
2/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Infections and infestations
Bacterial
|
25.0%
6/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Psychiatric disorders
Confusion
|
4.2%
1/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Blood and lymphatic system disorders
Delayed engraftment
|
4.2%
1/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
4.2%
1/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Infections and infestations
Fungal
|
4.2%
1/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
8.3%
2/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Renal and urinary disorders
Hemorrhagic Cystitis
|
4.2%
1/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
4.2%
1/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Blood and lymphatic system disorders
Low granulocyte
|
4.2%
1/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Investigations
Low platelet
|
8.3%
2/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Skin and subcutaneous tissue disorders
Rash
|
8.3%
2/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Vascular disorders
Thromboembolic event
|
8.3%
2/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Infections and infestations
Viral
|
20.8%
5/24 • Adverse event data collected through four treatment cycles of 28 days.
|
Other adverse events
| Measure |
Azacytidine Maintenance After Allotx
n=24 participants at risk
Fludarabine 40 mg/m\^2 intravenous (IV) Day -5 to Day -2; Busulfan dose calculated to achieve area under curve (AUC) of 4000 µMol-min + 12% based on pharmacokinetic studies (days -5, -4, -3, and -2); Thymoglobulin: 2.5 mg/kg IV on Day -3 to Day -1; Azacitidine 32 mg/m\^2 subcutaneous daily for 5 days starting 5 weeks after transplant, repeated monthly up to 4 months. Allogeneic stem cell infusion (allotx) on day 0.
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
8.3%
2/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Investigations
ALT increased
|
29.2%
7/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Metabolism and nutrition disorders
Anorexia
|
4.2%
1/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Infections and infestations
Bacterial
|
16.7%
4/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Eye disorders
Blurred vision
|
4.2%
1/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
12.5%
3/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Respiratory, thoracic and mediastinal disorders
Broncholitis obliterans
|
4.2%
1/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Injury, poisoning and procedural complications
Bruising
|
4.2%
1/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Investigations
Creatinine increased
|
12.5%
3/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Gastrointestinal disorders
Diarrhea
|
29.2%
7/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Nervous system disorders
Dizziness
|
4.2%
1/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Eye disorders
Dry eye
|
8.3%
2/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
General disorders
Fever
|
75.0%
18/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
General disorders
Flu like syndrome
|
4.2%
1/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
General disorders
Fluid overload
|
20.8%
5/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Nervous system disorders
Headache
|
20.8%
5/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Renal and urinary disorders
Hemorrhagic Cystitis
|
8.3%
2/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
4.2%
1/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Vascular disorders
Hypertension
|
8.3%
2/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Blood and lymphatic system disorders
Low granulocyte
|
4.2%
1/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Investigations
Low platelet
|
8.3%
2/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Gastrointestinal disorders
Nausea
|
91.7%
22/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Gastrointestinal disorders
Oral mucositis
|
58.3%
14/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Skin and subcutaneous tissue disorders
Rash
|
41.7%
10/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Investigations
T bilirubin increased
|
20.8%
5/24 • Adverse event data collected through four treatment cycles of 28 days.
|
|
Infections and infestations
Viral
|
50.0%
12/24 • Adverse event data collected through four treatment cycles of 28 days.
|
Additional Information
Richard E. Champlin, MD/Chair, Stem Cell Transplantation
University of Texas (UT) MD Anderson Cancer Center
Phone: 713-792-3618
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place