Dexamethasone Treatment of Congenital Adrenal Hyperplasia

NCT00621985 | Completed Phase 2 Results

• 1 other identifier: 08-01-0025
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

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.

Conditions

Adrenal Hyperplasia, Congenital

Outcome Measures

Primary Outcomes (1)

• Percent Difference in the Mean Log Transformed Area Under the Curve of 17-hydroxyprogesterone Between Regimens

  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.

  23 hours

Study Arms (1)

Experimental

EXPERIMENTAL

Experimental therapy with nocturnal dexamethasone.

Drug: dexamethasone; Drug: Hydrocortisone

Interventions

dexamethasone DRUG

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.

Also known as: Decadron

Experimental

Hydrocortisone DRUG

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.

Experimental

Eligibility Criteria

• Age: 2 Years - 9 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Boston Children's Hospital: lead

Study Sites (1)

Children's Hospital Boston

Boston, Massachusetts, 02115, United States

Location

Related Publications (1)

• Dauber A, Feldman HA, Majzoub JA. Nocturnal Dexamethasone versus Hydrocortisone for the Treatment of Children with Congenital Adrenal Hyperplasia. Int J Pediatr Endocrinol. 2010;2010:347636. doi: 10.1155/2010/347636. Epub 2010 Sep 14.

  PMID: 20871859 RESULT

MeSH Terms

Conditions

Adrenal Hyperplasia, Congenital

Interventions

Dexamethasone; Calcium Dobesilate; Hydrocortisone

Condition Hierarchy (Ancestors)

Adrenogenital Syndrome; Disorders of Sex Development; Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Steroid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Adrenal Gland Diseases; Endocrine System Diseases; Gonadal Disorders

Intervention Hierarchy (Ancestors)

Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Steroids, Fluorinated; Benzenesulfonates; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Arylsulfonates; Arylsulfonic Acids; Sulfonic Acids; Sulfur Acids; Sulfur Compounds; Pregnenediones; Pregnenes; 11-Hydroxycorticosteroids; Hydroxycorticosteroids; Adrenal Cortex Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; 17-Hydroxycorticosteroids

Results Point of Contact

• Title: Joseph Majzoub
• Organization: Children's Hospital Boston

joseph.majzoub@childrens.harvard.edu

617-355-7476

Study Officials

• Joseph Majzoub, MD

  Boston Children's Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: February 11, 2008
• First Posted: February 22, 2008
• Study Start: April 1, 2008
• Primary Completion: June 1, 2009
• Study Completion: June 1, 2009
• Last Updated: February 23, 2011
• Results First Posted: February 23, 2011

Record last verified: 2011-01

Locations

US (1)