Dexamethasone Treatment of Congenital Adrenal Hyperplasia

Phase 2

Completed

• Conditions: Adrenal Hyperplasia, Congenital
• Interventions: Drug: dexamethasone; Drug: Hydrocortisone

• Registration Number: NCT00621985
• Lead Sponsor: Boston Children's Hospital

Brief Summary

The purpose of this study is to determine if dexamethasone given at night is a more effective treatment for congenital adrenal hyperplasia in young children than standard three times per day hydrocortisone. Our hypothesis is that nocturnal dexamethasone will lead to more efficient suppression of the hypothalamic-pituitary-adrenal axis. We performed a cross-over trial comparing hormonal control during two 24-hour hospitalizations, one on hydrocortisone and one on nocturnal dexamethasone.

Detailed Description

This is a Phase II clinical trial, intended to estimate the effect of instituting Dexamethasone therapy in comparison to prior standard therapy. Each subject provides his own baseline data. There is no control group. Patients with CAH who meet inclusion criteria will be admitted to the clinical research center for two 24 hour hospitalizations. Adrenal hormone profiles will be measured during each hospitalization. The patient will take his or her baseline hydrocortisone regimen during one hospitalization and a new regimen consisting of a single daily nocturnal dose of Dexamethasone during the second hospitalization.

Study Timeline

• Study First Submitted: 2008-02-11
• Study First Submitted QC: 2008-02-11
• Study First Posted: 2008-02-22
• Study Start: 2008-04
• Primary Completion: 2009-06
• Study Completion: 2009-06
• Results First Submitted: 2010-09-17
• Status Verified: 2011-01
• Results First Submitted QC: 2011-01-26
• Last Update Submitted: 2011-01-26
• Results First Posted: 2011-02-23
• Last Update Posted: 2011-02-23

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 5

Inclusion Criteria

• Classic salt-wasting 21-hydroxylase deficient congenital adrenal hyperplasia
• Pre-pubertal children with bone ages below 8 years

Exclusion Criteria

• Age less than 2 years
• Patients with additional medical conditions necessitating glucocorticoid therapy.
• Patients on phenytoin, barbiturates, and rifampin as these medications accelerate the metabolism of glucocorticoids.
• Patients on ketoconazole as this medication increases the bioavailability of glucocorticoids.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
Experimental	dexamethasone	Experimental therapy with nocturnal dexamethasone.
Experimental	Hydrocortisone	Experimental therapy with nocturnal dexamethasone.

Primary Outcome Measures

Name	Time	Method
Percent Difference in the Mean Log Transformed Area Under the Curve of 17-hydroxyprogesterone Between Regimens	23 hours	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.

Trial Locations

Locations (1)

Children's Hospital Boston; 🇺🇸 Boston, Massachusetts, United States