Trial Outcomes & Findings for Steroid Treatment for Sickle Cell Pain Crisis (NCT NCT00263562)
NCT ID: NCT00263562
Last Updated: 2020-07-07
Results Overview
Severity of pain using a 10-point scale ranging from 1-10, with higher numbers corresponding to worsening pain.
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
18 participants
Primary outcome timeframe
30 days
Results posted on
2020-07-07
Participant Flow
Participant milestones
| Measure |
Intervention (Steroids)
Day 1: Solumedrol 15 mg/kg (maximum 1 gram) Day 2: Prednisone 2mg/kg PO BID Day 3: Prednisone 2mg/kg PO daily Day 4: Prednisone 1mg/kg PO daily Day 5: Prednisone 1mg/kg PO daily
|
Placebo
a comparison group who received standard of care therapy for acute chest syndrome, with normal saline substituted in lieu of IV solumedrol (methylprednisolone) and placebo pills in lieu of prednisone tablets (same # of tablets of placebo given as would have been administered to the intervention arm).
|
|---|---|---|
|
Overall Study
STARTED
|
9
|
9
|
|
Overall Study
COMPLETED
|
6
|
9
|
|
Overall Study
NOT COMPLETED
|
3
|
0
|
Reasons for withdrawal
| Measure |
Intervention (Steroids)
Day 1: Solumedrol 15 mg/kg (maximum 1 gram) Day 2: Prednisone 2mg/kg PO BID Day 3: Prednisone 2mg/kg PO daily Day 4: Prednisone 1mg/kg PO daily Day 5: Prednisone 1mg/kg PO daily
|
Placebo
a comparison group who received standard of care therapy for acute chest syndrome, with normal saline substituted in lieu of IV solumedrol (methylprednisolone) and placebo pills in lieu of prednisone tablets (same # of tablets of placebo given as would have been administered to the intervention arm).
|
|---|---|---|
|
Overall Study
Protocol Violation
|
3
|
0
|
Baseline Characteristics
Steroid Treatment for Sickle Cell Pain Crisis
Baseline characteristics by cohort
| Measure |
Intervention Arm
n=9 Participants
Receipt of IV pulse of steroids (methylprednisolone) followed by a steroid taper on the subsequent days: Day 2: Prednisone 2mg/kg PO BID Day 3: Prednisone 2mg/kg PO daily Day 4: Prednisone 1mg/kg PO daily Day 5: Prednisone 1mg/kg PO daily
|
Placebo Arm
n=9 Participants
Receipt of usual care, with administration of placebo: 1) normal saline in lieu of IV methylprednisolone followed by 2) placebo tablets in lieu of prednisone tablets.
|
Total
n=18 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
9 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 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
|
6.5 years
n=5 Participants
|
10.6 years
n=7 Participants
|
9.6 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
8 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 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
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 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
|
8 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 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
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
9 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 30 daysPopulation: Analyzed by intent to treat (ITT), with the 3 protocol deviations in the intervention arm (all 3 received placebo) counting toward the intervention arm.
Severity of pain using a 10-point scale ranging from 1-10, with higher numbers corresponding to worsening pain.
Outcome measures
| Measure |
Intervention (Steroids)
n=9 Participants
Receipt of pulse IV dose of methylprednisolone followed by a tapering dose of systemic oral corticosteroids (prednisone).
|
Placebo Group
n=9 Participants
Receipt of usual care, with normal saline in lieu of IV methylprednisolone and placebo tablets in lieu of prednisone tablets.
|
|---|---|---|
|
Pain Scores
|
4.3 score on a scale
Interval 2.5 to 6.7
|
4.5 score on a scale
Interval 2.1 to 7.1
|
SECONDARY outcome
Timeframe: Through hospitalization, up to 15 daysPopulation: Intent to treat
Length of stay (from emergency department arrival to discharge from the inpatient unit), in days, through hospitalization, up to 15 days
Outcome measures
| Measure |
Intervention (Steroids)
n=9 Participants
Receipt of pulse IV dose of methylprednisolone followed by a tapering dose of systemic oral corticosteroids (prednisone).
|
Placebo Group
n=9 Participants
Receipt of usual care, with normal saline in lieu of IV methylprednisolone and placebo tablets in lieu of prednisone tablets.
|
|---|---|---|
|
Duration of Hospitalization
|
6.5 days
Interval 4.9 to 8.2
|
10.6 days
Interval 7.3 to 13.2
|
SECONDARY outcome
Timeframe: 30 daysPopulation: Intent to treat
To describe adverse events, including infection, hypertension, and/or GI bleeding
Outcome measures
| Measure |
Intervention (Steroids)
n=9 Participants
Receipt of pulse IV dose of methylprednisolone followed by a tapering dose of systemic oral corticosteroids (prednisone).
|
Placebo Group
n=9 Participants
Receipt of usual care, with normal saline in lieu of IV methylprednisolone and placebo tablets in lieu of prednisone tablets.
|
|---|---|---|
|
Number of Participants With Complications and Adverse Events
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 30 daysPopulation: Intent to treat
Number of recurrent episodes of pain within 1 month of treatment
Outcome measures
| Measure |
Intervention (Steroids)
n=9 Participants
Receipt of pulse IV dose of methylprednisolone followed by a tapering dose of systemic oral corticosteroids (prednisone).
|
Placebo Group
n=9 Participants
Receipt of usual care, with normal saline in lieu of IV methylprednisolone and placebo tablets in lieu of prednisone tablets.
|
|---|---|---|
|
Number of Participants With Recurrent Episodes of Pain Within 1 Month of Treatment
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Through hospitalization, up to 15 daysPopulation: Intent to treat
\# of days during which the child received parenterally-administered opioids through hospitalization, up to 15 days.
Outcome measures
| Measure |
Intervention (Steroids)
n=9 Participants
Receipt of pulse IV dose of methylprednisolone followed by a tapering dose of systemic oral corticosteroids (prednisone).
|
Placebo Group
n=9 Participants
Receipt of usual care, with normal saline in lieu of IV methylprednisolone and placebo tablets in lieu of prednisone tablets.
|
|---|---|---|
|
Number of Days Analgesia Used
|
4 days
Interval 1.0 to 7.0
|
3 days
Interval 1.0 to 8.0
|
Adverse Events
Intervention Arm (Steroids)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Control Group
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place