Trial Outcomes & Findings for PRO#1278: Fludarabine and Busulfan vs. Fludarabine, Busulfan and Total Body Irradiation (NCT NCT01366612)
NCT ID: NCT01366612
Last Updated: 2023-06-13
Results Overview
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
53 participants
Primary outcome timeframe
1 year
Results posted on
2023-06-13
Participant Flow
Of the 53 consented, Forty-Seven patients with myeloid diseases were randomized into Flu/Bu4 (25 patients) or Flu/Bu4/TBI (22 patients)
Participant milestones
| Measure |
Group 1
FLUDARABINE AND BUSULFAN
Fludarabine and Busulfan plus/minus Total Body Irradiation (low dose): Fludarabine 40mg/m2 and Busulfan 130mg/m2 on days -6, -5, -4 and -3 of transplant.
rATG on days -3, -2 and -1
|
Group 2
FLUDARABINE, BUSULFAN AND LOW DOSE TOTAL BODY IRRADIATION
Fludarabine and Busulfan + Low Dose Total Body Irradiation (LD TBI): Fludarabine 40mg/m2 and Busulfan 130mg/m2 on days -6, -5, -4 and -3 of transplant.
rATG on days -3, -2 and -1 TBI 200cGY (as randomized) on day -1
|
|---|---|---|
|
Overall Study
STARTED
|
25
|
22
|
|
Overall Study
COMPLETED
|
25
|
22
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
PRO#1278: Fludarabine and Busulfan vs. Fludarabine, Busulfan and Total Body Irradiation
Baseline characteristics by cohort
| Measure |
Group 1
n=25 Participants
FLUDARABINE AND BUSULFAN
Fludarabine and Busulfan plus/minus Total Body Irradiation (low dose): Fludarabine 40mg/m2 and Busulfan 130mg/m2 on days -6, -5, -4 and -3 of transplant.
rATG on days -3, -2 and -1
|
Group 2
n=22 Participants
FLUDARABINE, BUSULFAN AND LOW DOSE TOTAL BODY IRRADIATION
Fludarabine and Busulfan + Low Dose Total Body Irradiation (LD TBI): Fludarabine 40mg/m2 and Busulfan 130mg/m2 on days -6, -5, -4 and -3 of transplant.
rATG on days -3, -2 and -1 TBI 200cGY (as randomized) on day -1
|
Total
n=47 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
25 Participants
n=5 Participants
|
22 Participants
n=7 Participants
|
47 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
48.4 Years
n=5 Participants
|
48.8 Years
n=7 Participants
|
48.6 Years
n=5 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
19 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
28 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
21 Participants
n=5 Participants
|
19 Participants
n=7 Participants
|
40 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
3 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
18 Participants
n=5 Participants
|
19 Participants
n=7 Participants
|
37 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
25 participants
n=5 Participants
|
22 participants
n=7 Participants
|
47 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 1 yearPopulation: Relapse Rate was analyzed in 34 patients with AML
Outcome measures
| Measure |
Group 1
n=18 Participants
FLUDARABINE AND BUSULFAN
Fludarabine and Busulfan plus/minus Total Body Irradiation (low dose): Fludarabine 40mg/m2 and Busulfan 130mg/m2 on days -6, -5, -4 and -3 of transplant.
rATG on days -3, -2 and -1
|
Group 2
n=16 Participants
FLUDARABINE, BUSULFAN AND LOW DOSE TOTAL BODY IRRADIATION
Fludarabine and Busulfan + Low Dose Total Body Irradiation (LD TBI): Fludarabine 40mg/m2 and Busulfan 130mg/m2 on days -6, -5, -4 and -3 of transplant.
rATG on days -3, -2 and -1 TBI 200cGY (as randomized) on day -1
|
|---|---|---|
|
To Compare the Relapse Rate at 1 Year of Patients With Myeloid Malignancies Receiving Each Treatment
|
38.9 Percent
|
18.8 Percent
|
Adverse Events
Group 1
Serious events: 18 serious events
Other events: 0 other events
Deaths: 6 deaths
Group 2
Serious events: 16 serious events
Other events: 0 other events
Deaths: 8 deaths
Serious adverse events
| Measure |
Group 1
n=25 participants at risk
FLUDARABINE AND BUSULFAN
Fludarabine and Busulfan plus/minus Total Body Irradiation (low dose): Fludarabine 40mg/m2 and Busulfan 130mg/m2 on days -6, -5, -4 and -3 of transplant.
rATG on days -3, -2 and -1
|
Group 2
n=22 participants at risk
FLUDARABINE, BUSULFAN AND LOW DOSE TOTAL BODY IRRADIATION
Fludarabine and Busulfan + Low Dose Total Body Irradiation (LD TBI): Fludarabine 40mg/m2 and Busulfan 130mg/m2 on days -6, -5, -4 and -3 of transplant.
rATG on days -3, -2 and -1 TBI 200cGY (as randomized) on day -1
|
|---|---|---|
|
Investigations
Multi-system failure
|
4.0%
1/25 • Number of events 1 • 1 year
|
9.1%
2/22 • Number of events 3 • 1 year
|
|
Blood and lymphatic system disorders
HUS-TTP
|
4.0%
1/25 • Number of events 1 • 1 year
|
0.00%
0/22 • 1 year
|
|
Vascular disorders
Cerebrovascular Accident
|
0.00%
0/25 • 1 year
|
4.5%
1/22 • Number of events 1 • 1 year
|
|
Vascular disorders
Veno-Occlusive Disease
|
0.00%
0/25 • 1 year
|
4.5%
1/22 • Number of events 1 • 1 year
|
|
Investigations
Fall
|
0.00%
0/25 • 1 year
|
4.5%
1/22 • Number of events 1 • 1 year
|
|
Investigations
Post-Transplant Lymphoproliferative Disease
|
0.00%
0/25 • 1 year
|
4.5%
1/22 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
Colitis and Internal Hemorrhoids
|
4.0%
1/25 • Number of events 1 • 1 year
|
0.00%
0/22 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Shortness of Breath
|
4.0%
1/25 • Number of events 1 • 1 year
|
9.1%
2/22 • Number of events 2 • 1 year
|
|
Infections and infestations
Sapovirus
|
4.0%
1/25 • Number of events 1 • 1 year
|
0.00%
0/22 • 1 year
|
|
Nervous system disorders
Limbic Encephalitis
|
4.0%
1/25 • Number of events 1 • 1 year
|
0.00%
0/22 • 1 year
|
|
Metabolism and nutrition disorders
Hyponatremia
|
4.0%
1/25 • Number of events 1 • 1 year
|
0.00%
0/22 • 1 year
|
|
Nervous system disorders
Headache
|
0.00%
0/25 • 1 year
|
4.5%
1/22 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Acute Asthma
|
0.00%
0/25 • 1 year
|
4.5%
1/22 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
Bowel Obstruction
|
0.00%
0/25 • 1 year
|
9.1%
2/22 • Number of events 2 • 1 year
|
|
Investigations
Chest Pain
|
4.0%
1/25 • Number of events 1 • 1 year
|
0.00%
0/22 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
4.0%
1/25 • Number of events 1 • 1 year
|
0.00%
0/22 • 1 year
|
|
Gastrointestinal disorders
Diarrhea and Vomiting
|
4.0%
1/25 • Number of events 1 • 1 year
|
0.00%
0/22 • 1 year
|
|
Investigations
Elevated LFTs
|
0.00%
0/25 • 1 year
|
4.5%
1/22 • Number of events 1 • 1 year
|
|
Investigations
Fever with Nausea and Vomiting
|
4.0%
1/25 • Number of events 1 • 1 year
|
0.00%
0/22 • 1 year
|
|
Nervous system disorders
Increased Confusion
|
4.0%
1/25 • Number of events 1 • 1 year
|
0.00%
0/22 • 1 year
|
|
General disorders
Left Left Extremity Swelling
|
4.0%
1/25 • Number of events 1 • 1 year
|
0.00%
0/22 • 1 year
|
|
Investigations
Fever
|
4.0%
1/25 • Number of events 1 • 1 year
|
9.1%
2/22 • Number of events 2 • 1 year
|
|
Infections and infestations
Infection/Reactivation
|
20.0%
5/25 • Number of events 5 • 1 year
|
9.1%
2/22 • Number of events 2 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
12.0%
3/25 • Number of events 3 • 1 year
|
9.1%
2/22 • Number of events 3 • 1 year
|
|
Immune system disorders
GvHD
|
36.0%
9/25 • Number of events 12 • 1 year
|
27.3%
6/22 • Number of events 6 • 1 year
|
|
Investigations
Relapse/Disease Progression
|
28.0%
7/25 • Number of events 7 • 1 year
|
22.7%
5/22 • Number of events 5 • 1 year
|
|
Cardiac disorders
Cardiac Tamponade
|
0.00%
0/25 • 1 year
|
4.5%
1/22 • Number of events 1 • 1 year
|
|
Infections and infestations
BK hemorrhagic cystitis
|
0.00%
0/25 • 1 year
|
18.2%
4/22 • Number of events 4 • 1 year
|
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place