Dexamethasone Treatment of Congenital Adrenal Hyperplasia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00621985
Recruitment Status : Completed
First Posted : February 22, 2008
Results First Posted : February 23, 2011
Last Update Posted : February 23, 2011
Information provided by:
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.

Condition or disease Intervention/treatment Phase
Adrenal Hyperplasia, Congenital Drug: dexamethasone Drug: Hydrocortisone Phase 2

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 Type : Interventional  (Clinical Trial)
Actual Enrollment : 5 participants
Allocation: Non-Randomized
Intervention Model: Crossover Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Dexamethasone Treatment of Congenital Adrenal Hyperplasia
Study Start Date : April 2008
Primary Completion Date : June 2009
Study Completion Date : June 2009

Arm Intervention/treatment
Experimental: Experimental
Experimental therapy with nocturnal dexamethasone.
Drug: dexamethasone
Dexamethasone will be given at a dose that equals 1/50 of the total daily hydrocortisone dose of the patient. It will be given in solution form at 10 PM for 3 days.
Other Name: Decadron
Drug: Hydrocortisone
Subjects were given their baseline hydrocortisone regimen which was three times daily for 4 of the subjects and twice daily for one subject. Doses were given at 8 AM, 2 PM, and 8 PM. The 2 PM time point was skipped for the subject who received hydrocortisone twice daily. Doses ranged from 6.9 to 18.5 milligrams per meter squared per day and were based on each individual's baseline regimen.

Primary Outcome Measures :
  1. Percent Difference in the Mean Log Transformed Area Under the Curve of 17-hydroxyprogesterone Between Regimens [ Time Frame: 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.

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Ages Eligible for Study:   2 Years to 9 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

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To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00621985

United States, Massachusetts
Children's Hospital Boston
Boston, Massachusetts, United States, 02115
Sponsors and Collaborators
Boston Children’s Hospital
Principal Investigator: Joseph Majzoub, MD Boston Children’s Hospital

Publications of Results:
Responsible Party: Joseph Majzoub, Children's Hospital Boston Identifier: NCT00621985     History of Changes
Other Study ID Numbers: 08-01-0025
First Posted: February 22, 2008    Key Record Dates
Results First Posted: February 23, 2011
Last Update Posted: February 23, 2011
Last Verified: January 2011

Additional relevant MeSH terms:
Adrenal Hyperplasia, Congenital
Adrenogenital Syndrome
Adrenocortical Hyperfunction
Pathologic Processes
Disorders of Sex Development
Urogenital Abnormalities
Congenital Abnormalities
Genetic Diseases, Inborn
Steroid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Metabolic Diseases
Adrenal Gland Diseases
Endocrine System Diseases
Gonadal Disorders
Dexamethasone acetate
Hydrocortisone 17-butyrate 21-propionate
Hydrocortisone acetate
Cortisol succinate
BB 1101
Anti-Inflammatory Agents
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Gastrointestinal Agents