Trial Outcomes & Findings for Studying Changes in Physical Function Measures in Stem Cell Transplant Patients at Risk for Steroid Myopathy (NCT NCT04521777)
NCT ID: NCT04521777
Last Updated: 2021-04-20
Results Overview
5xSTS was measured in seconds on Baseline, Day 14, Day 28 and Day 56 and measured the median changes for Day 14 minus Day 0, Day 28 minus Day 0 and Day 56 minus Day 0.
Recruitment status
TERMINATED
Study phase
NA
Target enrollment
24 participants
Primary outcome timeframe
Baseline, Day 14, Day 28 and Day 56
Results posted on
2021-04-20
Participant Flow
Patients who were treated with high dose corticosteroid for Acute Graft Versus Host Disease at MD Anderson Cancer Center between October 2015 and April 2017.
Participant milestones
| Measure |
All Participants
In this single-arm trial, patients who were started on high dose corticosteroids (methylprednisolone or equivalent, 2mg/kg/day) for suspected Acute Graft Versus Host Disease were enrolled on the trial. In order monitor for muscle weakness, serial physical function measures were obtained (baseline, Day 14 after enrollment, Day 28, and Day 56). All enrolled patients were also instructed on a home-based exercise program.
|
|---|---|
|
Overall Study
STARTED
|
24
|
|
Overall Study
COMPLETED
|
20
|
|
Overall Study
NOT COMPLETED
|
4
|
Reasons for withdrawal
| Measure |
All Participants
In this single-arm trial, patients who were started on high dose corticosteroids (methylprednisolone or equivalent, 2mg/kg/day) for suspected Acute Graft Versus Host Disease were enrolled on the trial. In order monitor for muscle weakness, serial physical function measures were obtained (baseline, Day 14 after enrollment, Day 28, and Day 56). All enrolled patients were also instructed on a home-based exercise program.
|
|---|---|
|
Overall Study
Death
|
2
|
|
Overall Study
Withdrawal by Subject
|
2
|
Baseline Characteristics
Studying Changes in Physical Function Measures in Stem Cell Transplant Patients at Risk for Steroid Myopathy
Baseline characteristics by cohort
| Measure |
All Participants
n=23 Participants
In this single-arm trial, patients who were started on high dose corticosteroids (methylprednisolone or equivalent, 2mg/kg/day) for suspected Acute Graft Versus Host Disease were enrolled on the trial. In order monitor for muscle weakness, serial physical function measures were obtained (baseline, Day 14 after enrollment, Day 28, and Day 56). All enrolled patients were also instructed on a home-based exercise program.
|
|---|---|
|
Age, Continuous
|
60 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
21 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
2 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
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
21 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
23 participants
n=5 Participants
|
|
Body Mass Index
|
29 kg/m^2
n=5 Participants
|
|
Primary Cancer
Acute Myeloid Leukemia
|
7 Participants
n=5 Participants
|
|
Primary Cancer
Myelodysplastic Syndrome
|
4 Participants
n=5 Participants
|
|
Primary Cancer
B cell Lymphoma
|
3 Participants
n=5 Participants
|
|
Primary Cancer
Chronic Myeloid Leukemia
|
4 Participants
n=5 Participants
|
|
Primary Cancer
Chronic Lymphocytic Leukemia
|
2 Participants
n=5 Participants
|
|
Primary Cancer
Acute Lymphocytic Leukemia
|
2 Participants
n=5 Participants
|
|
Primary Cancer
Myelofibrosis
|
1 Participants
n=5 Participants
|
|
Cancer Status
Active
|
6 Participants
n=5 Participants
|
|
Cancer Status
Refractory
|
2 Participants
n=5 Participants
|
|
Cancer Status
Remission
|
15 Participants
n=5 Participants
|
|
Graft Source
Bone marrow
|
4 Participants
n=5 Participants
|
|
Graft Source
Peripheral Blood
|
19 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, Day 14, Day 28 and Day 566 minute walk test (measured in meters) was conducted on Baseline, Day 14, Day 28 and Day 56 and measured the median changes for Day 14 minus Day 0, Day 28 minus Day 0, and Day 56 minus Day 0.
Outcome measures
| Measure |
All Participants
n=23 Participants
In this single-arm trial, patients who were started on high dose corticosteroids (methylprednisolone or equivalent, 2mg/kg/day) for suspected Acute Graft Versus Host Disease were enrolled on the trial. In order monitor for muscle weakness, serial physical function measures were obtained (baseline, Day 14 after enrollment, Day 28, and Day 56). All enrolled patients were also instructed on a home-based exercise program.
|
|---|---|
|
6 Minutes Walk Test
Day 14 minus Day 0
|
-9.25 meters
Interval -354.0 to 174.0
|
|
6 Minutes Walk Test
Day 28 minus Day 0
|
-20 meters
Interval -360.0 to 185.0
|
|
6 Minutes Walk Test
Day 56 minus Day 0
|
-23 meters
Interval -360.0 to 263.0
|
PRIMARY outcome
Timeframe: Baseline, Day 14, Day 28 and Day 565xSTS was measured in seconds on Baseline, Day 14, Day 28 and Day 56 and measured the median changes for Day 14 minus Day 0, Day 28 minus Day 0 and Day 56 minus Day 0.
Outcome measures
| Measure |
All Participants
n=23 Participants
In this single-arm trial, patients who were started on high dose corticosteroids (methylprednisolone or equivalent, 2mg/kg/day) for suspected Acute Graft Versus Host Disease were enrolled on the trial. In order monitor for muscle weakness, serial physical function measures were obtained (baseline, Day 14 after enrollment, Day 28, and Day 56). All enrolled patients were also instructed on a home-based exercise program.
|
|---|---|
|
5 Times Sit to Stand (5xSTS)
Day 28 minus Day 0
|
4.0 seconds
Interval -16.0 to 37.0
|
|
5 Times Sit to Stand (5xSTS)
Day 14 minus Day 0
|
1.6 seconds
Interval -11.0 to 37.0
|
|
5 Times Sit to Stand (5xSTS)
Day 56 minus Day 0
|
1.85 seconds
Interval -14.5 to 37.0
|
PRIMARY outcome
Timeframe: Baseline, Day 14, Day 28 and Day 56Brooke Scale for myopathy (a score from 0 to 6, with 6 being the best score with full range of motion of the arms) was measured on Baseline, Day 14, Day 28 and Day 56 and measured the median changes for Day 14 minus Day 0, Day 28 minus Day 0 and Day 56 minus Day 0
Outcome measures
| Measure |
All Participants
n=23 Participants
In this single-arm trial, patients who were started on high dose corticosteroids (methylprednisolone or equivalent, 2mg/kg/day) for suspected Acute Graft Versus Host Disease were enrolled on the trial. In order monitor for muscle weakness, serial physical function measures were obtained (baseline, Day 14 after enrollment, Day 28, and Day 56). All enrolled patients were also instructed on a home-based exercise program.
|
|---|---|
|
Brooke Scale for Myopathy
Day 14 minus Day 0
|
0 score on a scale
Interval -6.0 to 0.0
|
|
Brooke Scale for Myopathy
Day 28 minus Day 0
|
0 score on a scale
Interval -6.0 to 0.0
|
|
Brooke Scale for Myopathy
Day 56 minus Day 0
|
0 score on a scale
Interval -6.0 to 0.0
|
PRIMARY outcome
Timeframe: Baseline, Day 14, Day 28 and Day 56The modified Adult Myopathy Assessment Tool assigns a numerical score to a series of physical tasks (arm raise \[0 to 3\], sustained arm raise \[0 to 4\], single sit-to-stand \[0 to 3\], sustained hip flexion \[0 to 4\], and sustained knee extension \[0 to 4\]). The total score of all test components (0 to 18) is reported, with 18 as the highest score for muscle function and endurance.
Outcome measures
| Measure |
All Participants
n=23 Participants
In this single-arm trial, patients who were started on high dose corticosteroids (methylprednisolone or equivalent, 2mg/kg/day) for suspected Acute Graft Versus Host Disease were enrolled on the trial. In order monitor for muscle weakness, serial physical function measures were obtained (baseline, Day 14 after enrollment, Day 28, and Day 56). All enrolled patients were also instructed on a home-based exercise program.
|
|---|---|
|
Modified Adult Myopathy Assessment Tool
Day 14 minus Day 0
|
0 score on a scale
Interval -18.0 to 3.0
|
|
Modified Adult Myopathy Assessment Tool
Day 28 minus Day 0
|
-1 score on a scale
Interval -18.0 to 3.0
|
|
Modified Adult Myopathy Assessment Tool
Day 56 minus Day 0
|
-3 score on a scale
Interval -18.0 to 3.0
|
PRIMARY outcome
Timeframe: Baseline, Day 14, Day 28 and Day 56Knee Extensors Strength (in pounds) was measured on Baseline, Day 14, Day 28 and Day 56 and measured the median change for Day 14 minus Day 0, Day 28 minus Day 0 and Day 56 minus Day 0.
Outcome measures
| Measure |
All Participants
n=23 Participants
In this single-arm trial, patients who were started on high dose corticosteroids (methylprednisolone or equivalent, 2mg/kg/day) for suspected Acute Graft Versus Host Disease were enrolled on the trial. In order monitor for muscle weakness, serial physical function measures were obtained (baseline, Day 14 after enrollment, Day 28, and Day 56). All enrolled patients were also instructed on a home-based exercise program.
|
|---|---|
|
Knee Extensors Strength
Day 14 minus Day 0
|
-6.43 pounds
Interval -32.2 to 14.25
|
|
Knee Extensors Strength
Day 28 minus Day 0
|
-4.83 pounds
Interval -32.2 to 11.78
|
|
Knee Extensors Strength
Day 56 minus Day 0
|
-14.30 pounds
Interval -32.2 to 12.73
|
PRIMARY outcome
Timeframe: Baseline, Day 14, Day 28 and Day 56Hip Flexors Strength (in pounds) was measured on Baseline, Day 14, Day 28 and Day 56 and measured the median changes for Day 14 minus Day 0, Day 28 minus Day 0 and Day 56 minus Day 0.
Outcome measures
| Measure |
All Participants
n=23 Participants
In this single-arm trial, patients who were started on high dose corticosteroids (methylprednisolone or equivalent, 2mg/kg/day) for suspected Acute Graft Versus Host Disease were enrolled on the trial. In order monitor for muscle weakness, serial physical function measures were obtained (baseline, Day 14 after enrollment, Day 28, and Day 56). All enrolled patients were also instructed on a home-based exercise program.
|
|---|---|
|
Hip Flexors Strength
Day 14 minus Day 0
|
-3.21 pounds
Interval -37.03 to 16.65
|
|
Hip Flexors Strength
Day 28 minus Day 0
|
-4.98 pounds
Interval -37.03 to 20.65
|
|
Hip Flexors Strength
Day 56 minus Day 0
|
-4.90 pounds
Interval -37.03 to 20.78
|
Adverse Events
All Participants
Serious events: 0 serious events
Other events: 0 other events
Deaths: 3 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
An T Ngo-Huang, Assistant Professor, Rehabilitation Medicine
UT MD Anderson Cancer Center
Phone: (713) 745-2327
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place