Trial Outcomes & Findings for Dexamethasone Treatment of Congenital Adrenal Hyperplasia (NCT NCT00621985)

Results Overview

Each subject was admitted for 2 24 hour hospitalizations, one on hydrocortisone and one on dexamethasone. Due to the timing of blood draws, the serum hormonal profile was only measured for 23 hours. The primary outcome was the Percent Difference in the Mean log transformed Area under the curve of 17-hydroxyprogesterone between the two regimens.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

5 participants

Primary outcome timeframe

23 hours

Results posted on

2011-02-23

Participant Flow

Subjects were recruited from the Endocrine Clinic at Children's Hospital Boston from April through November 2008.

This was a cross over study. All subjects were admitted on their baseline hydrocortisone regimen for a 24 hour hospital admission. They then had a second hospital admission within 8 weeks while being administered the dexamethasone therapy.

Participant milestones

Participant milestones
Measure	Experimental Baseline hydrocortisone was given at a dose determined by the subject's primary endocrinologist and was given either 2 or 3 times per day as per their home regimen. Experimental therapy with nocturnal dexamethasone given at a dose equivalent to 1/50th of the total daily hydrocortisone dose. This dose was given at 10 PM for three nights with the admission to the hospital occurring on the 3rd day prior to the 3rd evening dose.
Hydrocortisone Admission STARTED	5
Hydrocortisone Admission COMPLETED	5
Hydrocortisone Admission NOT COMPLETED	0
Dexamethasone Admission STARTED	5
Dexamethasone Admission COMPLETED	4
Dexamethasone Admission NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Experimental Baseline hydrocortisone was given at a dose determined by the subject's primary endocrinologist and was given either 2 or 3 times per day as per their home regimen. Experimental therapy with nocturnal dexamethasone given at a dose equivalent to 1/50th of the total daily hydrocortisone dose. This dose was given at 10 PM for three nights with the admission to the hospital occurring on the 3rd day prior to the 3rd evening dose.
Dexamethasone Admission Adverse Event	1

Baseline Characteristics

Dexamethasone Treatment of Congenital Adrenal Hyperplasia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Experimental Group n=5 Participants These subjects were admitted twice, once while on baseline hydrocortisone and once on dexamethasone.
Age, Categorical <=18 years	5 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	2 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Region of Enrollment United States	5 participants n=5 Participants

PRIMARY outcome

Timeframe: 23 hours

Population: Only four participants completed both the hydrocortisone and dexamethasone admissions.

Each subject was admitted for 2 24 hour hospitalizations, one on hydrocortisone and one on dexamethasone. Due to the timing of blood draws, the serum hormonal profile was only measured for 23 hours. The primary outcome was the Percent Difference in the Mean log transformed Area under the curve of 17-hydroxyprogesterone between the two regimens.

Outcome measures

Outcome measures
Measure	Dexamethasone Admission n=4 Participants This is the second admission of the protocol during which the subject received dexamethasone.	Hydrocortisone Admission n=4 Participants This is the first admission of the protocol during which the subject received hydrocortisone.
Percent Difference in the Mean Log Transformed Area Under the Curve of 17-hydroxyprogesterone Between Regimens	3.16 Log Mean Area Under the Curve Standard Deviation 0.93	3.91 Log Mean Area Under the Curve Standard Deviation 0.66

Adverse Events

Dexamethasone Admission

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Hydrocortisone Admission

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Dexamethasone Admission n=5 participants at risk This is the second admission of the protocol during which the subject received dexamethasone.	Hydrocortisone Admission n=5 participants at risk This is the first admission of the protocol during which the subject received hydrocortisone.
Injury, poisoning and procedural complications IV infiltration	20.0% 1/5 • Number of events 1	0.00% 0/5

Additional Information

Joseph Majzoub

Children's Hospital Boston

Phone: 617-355-7476

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place