Trial Outcomes & Findings for Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia (NCT NCT03718234)
NCT ID: NCT03718234
Last Updated: 2025-06-25
Results Overview
24-hour area under the curve (AUC) following oral hydrocortisone dosing (admission 1).
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
11 participants
Primary outcome timeframe
Week 1
Results posted on
2025-06-25
Participant Flow
Participant milestones
| Measure |
Subcutaneous Hydrocortisone Via Infusion Pump
Patients will receive a subcutaneous injection of hydrocortisone (HC). Each patient's total daily dose (TDD) of oral tablet hydrocortisone to determine the doses to be delivered of the study drug. The 24-hr schedule and percentage of the TDD of HC will be as follows: approximately 60% of the TDD of HC will be delivered in 3 equal pulses at 0300, 0600 and 0900. Another 35% will be delivered in 3 equal pulses at 1200, 1500 and 1800 and the remaining 5% at 2100 and 2400.
Subcutaneous hydrocortisone: Patients will be monitored and receive an interval bolus SQHC dosing regimen that more closely mimics cortisol, 17OHP and D4A circadian and ultradian rhythms than conventional oral HC dosing.
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|---|---|
|
Overall Study
STARTED
|
11
|
|
Overall Study
COMPLETED
|
8
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia
Baseline characteristics by cohort
| Measure |
Subcutaneous Hydrocortisone Via Infusion Pump
n=11 Participants
Patients will receive a subcutaneous injection of hydrocortisone (HC). Each patient's total daily dose (TDD) of oral tablet hydrocortisone to determine the doses to be delivered of the study drug. The 24-hr schedule and percentage of the TDD of HC will be as follows: approximately 60% of the TDD of HC will be delivered in 3 equal pulses at 0300, 0600 and 0900. Another 35% will be delivered in 3 equal pulses at 1200, 1500 and 1800 and the remaining 5% at 2100 and 2400.
Subcutaneous hydrocortisone: Patients will be monitored and receive an interval bolus SQHC dosing regimen that more closely mimics cortisol, 17OHP and D4A circadian and ultradian rhythms than conventional oral HC dosing.
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|---|---|
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Age, Categorical
<=18 years
|
11 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 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
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Week 124-hour area under the curve (AUC) following oral hydrocortisone dosing (admission 1).
Outcome measures
| Measure |
Subcutaneous Hydrocortisone Via Infusion Pump
n=8 Participants
Patients will receive a subcutaneous injection of hydrocortisone (HC). Each patient's total daily dose (TDD) of oral tablet hydrocortisone to determine the doses to be delivered of the study drug. The 24-hr schedule and percentage of the TDD of HC will be as follows: approximately 60% of the TDD of HC will be delivered in 3 equal pulses at 0300, 0600 and 0900. Another 35% will be delivered in 3 equal pulses at 1200, 1500 and 1800 and the remaining 5% at 2100 and 2400.
Subcutaneous hydrocortisone: Patients will be monitored and receive an interval bolus SQHC dosing regimen that more closely mimics cortisol, 17OHP and D4A circadian and ultradian rhythms than conventional oral HC dosing.
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|---|---|
|
Cortisol Exposure
|
110 hr*mcg/dL
Standard Error 9.96
|
PRIMARY outcome
Timeframe: Week 14The 24-hour cortisol area-under-the curve (AUC) while on the interval bolus delivery (pulsatile) of SQHC (admission 3)
Outcome measures
| Measure |
Subcutaneous Hydrocortisone Via Infusion Pump
n=8 Participants
Patients will receive a subcutaneous injection of hydrocortisone (HC). Each patient's total daily dose (TDD) of oral tablet hydrocortisone to determine the doses to be delivered of the study drug. The 24-hr schedule and percentage of the TDD of HC will be as follows: approximately 60% of the TDD of HC will be delivered in 3 equal pulses at 0300, 0600 and 0900. Another 35% will be delivered in 3 equal pulses at 1200, 1500 and 1800 and the remaining 5% at 2100 and 2400.
Subcutaneous hydrocortisone: Patients will be monitored and receive an interval bolus SQHC dosing regimen that more closely mimics cortisol, 17OHP and D4A circadian and ultradian rhythms than conventional oral HC dosing.
|
|---|---|
|
Cortisol Exposure
|
118 hr*mcg/dL
Standard Error 9.10
|
PRIMARY outcome
Timeframe: Week 124-hour area under the curve (AUC) following oral hydrocortisone dosing (admission 1).
Outcome measures
| Measure |
Subcutaneous Hydrocortisone Via Infusion Pump
n=8 Participants
Patients will receive a subcutaneous injection of hydrocortisone (HC). Each patient's total daily dose (TDD) of oral tablet hydrocortisone to determine the doses to be delivered of the study drug. The 24-hr schedule and percentage of the TDD of HC will be as follows: approximately 60% of the TDD of HC will be delivered in 3 equal pulses at 0300, 0600 and 0900. Another 35% will be delivered in 3 equal pulses at 1200, 1500 and 1800 and the remaining 5% at 2100 and 2400.
Subcutaneous hydrocortisone: Patients will be monitored and receive an interval bolus SQHC dosing regimen that more closely mimics cortisol, 17OHP and D4A circadian and ultradian rhythms than conventional oral HC dosing.
|
|---|---|
|
17-OHP Exposure
|
46700 hr*ng/dL
Standard Error 19200
|
PRIMARY outcome
Timeframe: Week 14The 24-hour cortisol area-under-the curve (AUC) while on the interval bolus delivery (pulsatile) of SQHC (admission 3)
Outcome measures
| Measure |
Subcutaneous Hydrocortisone Via Infusion Pump
n=8 Participants
Patients will receive a subcutaneous injection of hydrocortisone (HC). Each patient's total daily dose (TDD) of oral tablet hydrocortisone to determine the doses to be delivered of the study drug. The 24-hr schedule and percentage of the TDD of HC will be as follows: approximately 60% of the TDD of HC will be delivered in 3 equal pulses at 0300, 0600 and 0900. Another 35% will be delivered in 3 equal pulses at 1200, 1500 and 1800 and the remaining 5% at 2100 and 2400.
Subcutaneous hydrocortisone: Patients will be monitored and receive an interval bolus SQHC dosing regimen that more closely mimics cortisol, 17OHP and D4A circadian and ultradian rhythms than conventional oral HC dosing.
|
|---|---|
|
17-OHP Exposure
|
26900 hr*ng/dL
Standard Error 16200
|
PRIMARY outcome
Timeframe: Week 124-hour area under the curve (AUC) following oral hydrocortisone dosing (admission 1).
Outcome measures
| Measure |
Subcutaneous Hydrocortisone Via Infusion Pump
n=8 Participants
Patients will receive a subcutaneous injection of hydrocortisone (HC). Each patient's total daily dose (TDD) of oral tablet hydrocortisone to determine the doses to be delivered of the study drug. The 24-hr schedule and percentage of the TDD of HC will be as follows: approximately 60% of the TDD of HC will be delivered in 3 equal pulses at 0300, 0600 and 0900. Another 35% will be delivered in 3 equal pulses at 1200, 1500 and 1800 and the remaining 5% at 2100 and 2400.
Subcutaneous hydrocortisone: Patients will be monitored and receive an interval bolus SQHC dosing regimen that more closely mimics cortisol, 17OHP and D4A circadian and ultradian rhythms than conventional oral HC dosing.
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|---|---|
|
Androstenedione (A4) Exposure
|
3350 hr*ng/dL
Standard Error 2410
|
PRIMARY outcome
Timeframe: Week 14The 24-hour cortisol area-under-the curve (AUC) while on the interval bolus delivery (pulsatile) of SQHC (admission 3)
Outcome measures
| Measure |
Subcutaneous Hydrocortisone Via Infusion Pump
n=8 Participants
Patients will receive a subcutaneous injection of hydrocortisone (HC). Each patient's total daily dose (TDD) of oral tablet hydrocortisone to determine the doses to be delivered of the study drug. The 24-hr schedule and percentage of the TDD of HC will be as follows: approximately 60% of the TDD of HC will be delivered in 3 equal pulses at 0300, 0600 and 0900. Another 35% will be delivered in 3 equal pulses at 1200, 1500 and 1800 and the remaining 5% at 2100 and 2400.
Subcutaneous hydrocortisone: Patients will be monitored and receive an interval bolus SQHC dosing regimen that more closely mimics cortisol, 17OHP and D4A circadian and ultradian rhythms than conventional oral HC dosing.
|
|---|---|
|
Androstenedione (A4) Exposure
|
1420 hr*ng/dL
Standard Error 968
|
Adverse Events
Subcutaneous Hydrocortisone Via Infusion Pump
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Standard Glucocorticoid Therapy
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Subcutaneous Hydrocortisone Via Infusion Pump
n=11 participants at risk
Patients will receive a subcutaneous injection of hydrocortisone (HC). Each patient's total daily dose (TDD) of oral tablet hydrocortisone to determine the doses to be delivered of the study drug. The 24-hr schedule and percentage of the TDD of HC will be as follows: approximately 60% of the TDD of HC will be delivered in 3 equal pulses at 0300, 0600 and 0900. Another 35% will be delivered in 3 equal pulses at 1200, 1500 and 1800 and the remaining 5% at 2100 and 2400.
Subcutaneous hydrocortisone: Patients will be monitored and receive an interval bolus SQHC dosing regimen that more closely mimics cortisol, 17OHP and D4A circadian and ultradian rhythms than conventional oral HC dosing.
|
Standard Glucocorticoid Therapy
n=11 participants at risk
Subjects in this arm will continue on standard oral hydrocortisone therapy
Standard glucocorticoid therapy: Subjects in this arm will continue on standard oral hydrocortisone therapy
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|---|---|---|
|
General disorders
Stinging/Burning sensation
|
72.7%
8/11 • Number of events 11 • 20 weeks
|
72.7%
8/11 • Number of events 11 • 20 weeks
|
|
General disorders
Erythema
|
36.4%
4/11 • Number of events 4 • 20 weeks
|
36.4%
4/11 • Number of events 4 • 20 weeks
|
|
General disorders
Itching
|
45.5%
5/11 • Number of events 6 • 20 weeks
|
45.5%
5/11 • Number of events 6 • 20 weeks
|
|
General disorders
Skin rash
|
9.1%
1/11 • Number of events 1 • 20 weeks
|
9.1%
1/11 • Number of events 1 • 20 weeks
|
|
General disorders
Excoriation
|
18.2%
2/11 • Number of events 2 • 20 weeks
|
18.2%
2/11 • Number of events 2 • 20 weeks
|
|
General disorders
Hives
|
9.1%
1/11 • Number of events 1 • 20 weeks
|
9.1%
1/11 • Number of events 1 • 20 weeks
|
|
General disorders
Fatigue
|
9.1%
1/11 • Number of events 1 • 20 weeks
|
9.1%
1/11 • Number of events 1 • 20 weeks
|
|
General disorders
Fever
|
18.2%
2/11 • Number of events 4 • 20 weeks
|
18.2%
2/11 • Number of events 4 • 20 weeks
|
|
General disorders
Headache
|
9.1%
1/11 • Number of events 1 • 20 weeks
|
9.1%
1/11 • Number of events 1 • 20 weeks
|
|
General disorders
Nasal congestion
|
9.1%
1/11 • Number of events 1 • 20 weeks
|
9.1%
1/11 • Number of events 1 • 20 weeks
|
|
General disorders
covid
|
9.1%
1/11 • Number of events 1 • 20 weeks
|
9.1%
1/11 • Number of events 1 • 20 weeks
|
|
General disorders
Heartburn
|
9.1%
1/11 • Number of events 2 • 20 weeks
|
9.1%
1/11 • Number of events 2 • 20 weeks
|
|
General disorders
Ear infection
|
9.1%
1/11 • Number of events 1 • 20 weeks
|
9.1%
1/11 • Number of events 1 • 20 weeks
|
|
General disorders
Streptococcal pharyngitis
|
9.1%
1/11 • Number of events 1 • 20 weeks
|
9.1%
1/11 • Number of events 1 • 20 weeks
|
|
General disorders
Respiratory virus (RSV)
|
9.1%
1/11 • Number of events 1 • 20 weeks
|
9.1%
1/11 • Number of events 1 • 20 weeks
|
|
General disorders
stomach ache
|
9.1%
1/11 • Number of events 1 • 20 weeks
|
9.1%
1/11 • Number of events 1 • 20 weeks
|
|
General disorders
vasovagal syndrome
|
9.1%
1/11 • Number of events 2 • 20 weeks
|
9.1%
1/11 • Number of events 2 • 20 weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place