Trial Outcomes & Findings for Dexamethasone to Treat Acute Chest Syndrome in People With Sickle Cell Disease (NCT NCT00530270)
NCT ID: NCT00530270
Last Updated: 2013-04-09
Results Overview
Resolution of symptoms of ACS includes respiratory rate \<= upper limit of normal +2, no work of breathing (retractions, nasal flaring, and use of accessory muscles), thoracic pain \<= 4, no use of supplemental oxygen, no use of ventilary support, and saturation of peripheral oxygen (Sp02) \>= steady state value -2. Symptoms were measured every 4 hours from the first dose of study drug to resolution of symptoms or hospital discharge.
TERMINATED
PHASE3
12 participants
Measured from first dose to end of the hospital stay, no maximum number of days
2013-04-09
Participant Flow
Subjects were recruited from October 2006 through June 2008 at 10 sites across the United States. Due to the acute nature of the disease under study, subjects were recruited in a hospital, frequently in the Emergency Department.
No events excluded patients following enrollment, but prior to group assignment. Group assignments were made at enrollment.
Participant milestones
| Measure |
Dexamethasone
0.3 mg/kg (12 mg maximum single dose) every 12 hours until discharge from the hospital or for a maximum of 4 doses, whichever comes first. Thereafter, study drug will be tapered over 6 days (not to exceed 8 days).
|
Placebo
0.3 mg/kg (12 mg maximum single dose) every 12 hours until discharge from the hospital or for a maximum of 4 doses, whichever comes first. Thereafter, study drug will be tapered over 6 days (not to exceed 8 days).
|
|---|---|---|
|
Overall Study
STARTED
|
6
|
6
|
|
Overall Study
COMPLETED
|
5
|
6
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
| Measure |
Dexamethasone
0.3 mg/kg (12 mg maximum single dose) every 12 hours until discharge from the hospital or for a maximum of 4 doses, whichever comes first. Thereafter, study drug will be tapered over 6 days (not to exceed 8 days).
|
Placebo
0.3 mg/kg (12 mg maximum single dose) every 12 hours until discharge from the hospital or for a maximum of 4 doses, whichever comes first. Thereafter, study drug will be tapered over 6 days (not to exceed 8 days).
|
|---|---|---|
|
Overall Study
Protocol Violation
|
1
|
0
|
Baseline Characteristics
Dexamethasone to Treat Acute Chest Syndrome in People With Sickle Cell Disease
Baseline characteristics by cohort
| Measure |
Dexamethasone
n=6 Participants
0.3 mg/kg (12 mg maximum single dose) every 12 hours until discharge from the hospital or for a maximum of 4 doses, whichever comes first. Thereafter, study drug will be tapered over 6 days (not to exceed 8 days).
|
Placebo
n=6 Participants
0.3 mg/kg (12 mg maximum single dose) every 12 hours until discharge from the hospital or for a maximum of 4 doses, whichever comes first. Thereafter, study drug will be tapered over 6 days (not to exceed 8 days).
|
Total
n=12 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
4 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
9 Participants
n=206 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age Continuous
|
18.8 years
STANDARD_DEVIATION 14.08 • n=99 Participants
|
15.8 years
STANDARD_DEVIATION 12.81 • n=107 Participants
|
17.3 years
STANDARD_DEVIATION 12.93 • n=206 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
7 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
6 participants
n=99 Participants
|
6 participants
n=107 Participants
|
12 participants
n=206 Participants
|
PRIMARY outcome
Timeframe: Measured from first dose to end of the hospital stay, no maximum number of daysPopulation: Subject without major protocol violation and received treatment.
Resolution of symptoms of ACS includes respiratory rate \<= upper limit of normal +2, no work of breathing (retractions, nasal flaring, and use of accessory muscles), thoracic pain \<= 4, no use of supplemental oxygen, no use of ventilary support, and saturation of peripheral oxygen (Sp02) \>= steady state value -2. Symptoms were measured every 4 hours from the first dose of study drug to resolution of symptoms or hospital discharge.
Outcome measures
| Measure |
Dexamethasone
n=5 Participants
0.3 mg/kg (12 mg maximum single dose) every 12 hours until discharge from the hospital or for a maximum of 4 doses, whichever comes first. Thereafter, study drug will be tapered over 6 days (not to exceed 8 days).
|
Placebo
n=6 Participants
0.3 mg/kg (12 mg maximum single dose) every 12 hours until discharge from the hospital or for a maximum of 4 doses, whichever comes first. Thereafter, study drug will be tapered over 6 days (not to exceed 8 days).
|
|---|---|---|
|
Log (Natural) of Duration of Signs and Symptoms of Acute Chest Syndrome (ACS) or Duration of Hospitalization, Whichever is Less
|
2.4 hours (log transformed)
Standard Deviation 1.23
|
3.5 hours (log transformed)
Standard Deviation 0.60
|
SECONDARY outcome
Timeframe: Measured at the end of the hospital stayPopulation: Subject without major protocol violation and received treatment.
Change from baseline rating of pain from randomization (baseline) to discharge from the hospital, evaluated every 4 hours. Pain was rated on the Oucher Scale for the pediatric population or numeric rating scale for the adult population, both 0 to 10 with 0 indicating no pain and 10 indicating severe pain.
Outcome measures
| Measure |
Dexamethasone
n=5 Participants
0.3 mg/kg (12 mg maximum single dose) every 12 hours until discharge from the hospital or for a maximum of 4 doses, whichever comes first. Thereafter, study drug will be tapered over 6 days (not to exceed 8 days).
|
Placebo
n=6 Participants
0.3 mg/kg (12 mg maximum single dose) every 12 hours until discharge from the hospital or for a maximum of 4 doses, whichever comes first. Thereafter, study drug will be tapered over 6 days (not to exceed 8 days).
|
|---|---|---|
|
Rating of Pain
Thoracic
|
-3.8 Units on a scale
Standard Error 1.34
|
-4.1 Units on a scale
Standard Error 1.26
|
|
Rating of Pain
Non-thoracic
|
-2.0 Units on a scale
Standard Error 1.35
|
-1.4 Units on a scale
Standard Error 1.28
|
SECONDARY outcome
Timeframe: Measured at the end of hospital stay, no maximum number of daysPopulation: Subject without major protocol violation and received treatment.
Duration in hours from treatment start time to hospital discharge.
Outcome measures
| Measure |
Dexamethasone
n=5 Participants
0.3 mg/kg (12 mg maximum single dose) every 12 hours until discharge from the hospital or for a maximum of 4 doses, whichever comes first. Thereafter, study drug will be tapered over 6 days (not to exceed 8 days).
|
Placebo
n=6 Participants
0.3 mg/kg (12 mg maximum single dose) every 12 hours until discharge from the hospital or for a maximum of 4 doses, whichever comes first. Thereafter, study drug will be tapered over 6 days (not to exceed 8 days).
|
|---|---|---|
|
Duration of Hospitalization
|
41.5 Hours
Standard Deviation 9.03
|
62.3 Hours
Standard Deviation 14.27
|
SECONDARY outcome
Timeframe: Measured at the end of hospital stayPopulation: Subject without major protocol violation and received treatment.
Time period between the supplemental oxygen start date/time and first dose date/time, whichever is later, and the supplemental oxygen stop date/time
Outcome measures
| Measure |
Dexamethasone
n=5 Participants
0.3 mg/kg (12 mg maximum single dose) every 12 hours until discharge from the hospital or for a maximum of 4 doses, whichever comes first. Thereafter, study drug will be tapered over 6 days (not to exceed 8 days).
|
Placebo
n=6 Participants
0.3 mg/kg (12 mg maximum single dose) every 12 hours until discharge from the hospital or for a maximum of 4 doses, whichever comes first. Thereafter, study drug will be tapered over 6 days (not to exceed 8 days).
|
|---|---|---|
|
Duration of Supplemental Oxygen
|
17.5 Hours
Standard Deviation 0
|
41.2 Hours
Standard Deviation 22.22
|
SECONDARY outcome
Timeframe: Measured at the end of hospital stayPopulation: Subject without major protocol violation and received treatment.
Sum of time periods when subject was hypoxemic (Sp02 value less than 92%) since the first dose date/time
Outcome measures
| Measure |
Dexamethasone
n=5 Participants
0.3 mg/kg (12 mg maximum single dose) every 12 hours until discharge from the hospital or for a maximum of 4 doses, whichever comes first. Thereafter, study drug will be tapered over 6 days (not to exceed 8 days).
|
Placebo
n=6 Participants
0.3 mg/kg (12 mg maximum single dose) every 12 hours until discharge from the hospital or for a maximum of 4 doses, whichever comes first. Thereafter, study drug will be tapered over 6 days (not to exceed 8 days).
|
|---|---|---|
|
Duration of Hypoxemia (Low Blood Oxygen)
|
13.8 Hours
Standard Deviation 0
|
38.4 Hours
Standard Deviation 22.18
|
Adverse Events
Dexamethasone
Placebo
Serious adverse events
| Measure |
Dexamethasone
n=6 participants at risk
0.3 mg/kg (12 mg maximum single dose) every 12 hours until discharge from the hospital or for a maximum of 4 doses, whichever comes first. Thereafter, study drug will be tapered over 6 days (not to exceed 8 days).
|
Placebo
n=6 participants at risk
0.3 mg/kg (12 mg maximum single dose) every 12 hours until discharge from the hospital or for a maximum of 4 doses, whichever comes first. Thereafter, study drug will be tapered over 6 days (not to exceed 8 days).
|
|---|---|---|
|
Congenital, familial and genetic disorders
Sickle cell anemia with crisis
|
16.7%
1/6 • Number of events 1
|
0.00%
0/6
|
|
Infections and infestations
Gastroenteritis
|
16.7%
1/6 • Number of events 1
|
0.00%
0/6
|
Other adverse events
| Measure |
Dexamethasone
n=6 participants at risk
0.3 mg/kg (12 mg maximum single dose) every 12 hours until discharge from the hospital or for a maximum of 4 doses, whichever comes first. Thereafter, study drug will be tapered over 6 days (not to exceed 8 days).
|
Placebo
n=6 participants at risk
0.3 mg/kg (12 mg maximum single dose) every 12 hours until discharge from the hospital or for a maximum of 4 doses, whichever comes first. Thereafter, study drug will be tapered over 6 days (not to exceed 8 days).
|
|---|---|---|
|
Congenital, familial and genetic disorders
Sickle cell anemia with crisis
|
50.0%
3/6 • Number of events 3
|
0.00%
0/6
|
|
General disorders
Pyrexia
|
33.3%
2/6 • Number of events 2
|
0.00%
0/6
|
|
General disorders
Pain
|
16.7%
1/6 • Number of events 1
|
0.00%
0/6
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/6
|
16.7%
1/6 • Number of events 1
|
|
Psychiatric disorders
Insomnia
|
16.7%
1/6 • Number of events 1
|
0.00%
0/6
|
|
Vascular disorders
Hypertension
|
16.7%
1/6 • Number of events 1
|
0.00%
0/6
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/6
|
16.7%
1/6 • Number of events 1
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/6
|
16.7%
1/6 • Number of events 1
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place