Three Drug Combination Therapy Versus Conventional Treatment of Children With Congenital Adrenal Hyperplasia

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ClinicalTrials.gov Identifier: NCT00001521

Recruitment Status : Completed

First Posted : November 4, 1999

Last Update Posted : August 9, 2018

Sponsor:

Information provided by:

National Institutes of Health Clinical Center (CC)

Study Description

Brief Summary:

This study was developed to determine if a combination of four drugs (flutamide, testolactone, reduced hydrocortisone dose, and fludrocortisone) can normalize growth in children with congenital adrenal hyperplasia.

The study will take 60 children, boys and girls and divide them into 2 groups based on the medications given. Group one will receive the new four- drug combination. Group two will receive the standard treatment for congenital adrenal hyperplasia (hydrocortisone and fludrocortisone).

The boys in group one will take the medication until the age of 14 at which time they will stop taking the four drug combination and begin receiving the standard treatment for congenital adrenal hyperplasia. Girls in group one will take the four drug combination until the age of 13, at which time they will stop and begin receiving the standard treatment for congenital adrenal hyperplasia plus flutamide. Flutamide will be given to the girls until six months after their first menstrual period.

All of the children will be followed until they reach their final adult height. The effectiveness of the treatment will be determined by measuring the patient's adult height, body mass index, and bone density. <TAB>...

Condition or disease	Intervention/treatment	Phase
Congenital Adrenal Hyperplasia Growth Disorder	Drug: Flutamide and Testolactone Drug: Deslorelin	Phase 1

Detailed Description:

To test the hypothesis that the regimen of flutamide (an antiandrogen), testolactone or letrozole (an inhibitor of androgen-to-estrogen conversion), and reduced hydrocortisone dose can normalize the growth and adult stature of children with congenital adrenal hyperplasia, and can avoid the complications of supraphysiologic glucocorticoid dosage, 60 children with this disorder will be randomized to receive either the above regimen or conventional treatment until they have reached age 13 years in a girl or age 14 in a boy. After these ages boys will receive the conventional treatment and girls will receive conventional treatment plus flutamide. In girls, flutamide will be continued until 6 months after menarche. All children will be followed until they have attained final adult height. The principal outcome measures will be adult height, body mass index, and bone density.

Study Design


Study Type :	Interventional (Clinical Trial)
Actual Enrollment :	62 participants
Primary Purpose:	Treatment
Official Title:	An Open, Randomized, Long-Term Clinical Trial of Flutamide, Testolactone, and Reduced Hydrocortisone Dose vs. Conventional Treatment of Children With Congenital Adrenal Hyperplasia
Study Start Date :	February 2, 1996
Actual Study Completion Date :	January 11, 2017

Resource links provided by the National Library of Medicine

Genetics Home Reference related topics: 17 alpha-hydroxylase/17,20-lyase deficiency 21-hydroxylase deficiency 3-beta-hydroxysteroid dehydrogenase deficiency Congenital adrenal hyperplasia due to 11-beta-hydroxylase deficiency

Drug Information available for: Flutamide Deslorelin

Genetic and Rare Diseases Information Center resources: 21-hydroxylase Deficiency Congenital Adrenal Hyperplasia Hyperadrenalism

U.S. FDA Resources

Arms and Interventions

Outcome Measures

Eligibility Criteria

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Ages Eligible for Study:	2 Years to 20 Years (Child, Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

INCLUSION CRITERIA:

Subjects will be boys with bone ages 2 to 13 years and girls with bone ages 2 to 11 years with classic 21-hydroxylase.

Subjects must either not yet have undergone pubertal activation of the hypothalamic-pituitary-gonadal axis, or, if pubertal activation has occurred, must be receiving an LHRH agonist to suppress secondary central precocious puberty.

Children with a bone age of 1 to 2 years may enroll in the protocol for optimization of conventional therapy, but will not be randomized to a study arm until the bone age reaches 2.

EXCLUSION CRITERIA:

Children who have concurrent illnesses requiring glucocorticoid treatment (such as severe asthma), or requiring drugs that markedly alter hydrocortisone metabolism (such as anticonvulsants), and children who cannot be brought into reasonable control with conventional treatment (an unusual occurrence).

Contacts and Locations

Information from the National Library of Medicine

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 ClinicalTrials.gov identifier (NCT number): NCT00001521

Locations


United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892

Sponsors and Collaborators

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Investigators


Principal Investigator:	Deborah P Merke, M.D.	National Institutes of Health Clinical Center (CC)

More Information

Additional Information:

NIH Clinical Center Detailed Web Page This link exits the ClinicalTrials.gov site

Publications:

Cutler GB Jr, Laue L. Congenital adrenal hyperplasia due to 21-hydroxylase deficiency. N Engl J Med. 1990 Dec 27;323(26):1806-13. Review.

Laue L, Merke DP, Jones JV, Barnes KM, Hill S, Cutler GB Jr. A preliminary study of flutamide, testolactone, and reduced hydrocortisone dose in the treatment of congenital adrenal hyperplasia. J Clin Endocrinol Metab. 1996 Oct;81(10):3535-9.

Merke DP, Cutler GB Jr. New approaches to the treatment of congenital adrenal hyperplasia. JAMA. 1997 Apr 2;277(13):1073-6.


ClinicalTrials.gov Identifier:	NCT00001521 History of Changes
Other Study ID Numbers:	960033 96-CH-0033
First Posted:	November 4, 1999 Key Record Dates
Last Update Posted:	August 9, 2018
Last Verified:	January 17, 2018

Keywords provided by National Institutes of Health Clinical Center (CC):


21-Hydroxylase Deficiency 11-Hydroxylase Deficiency Antiandrogen	Aromatase Inhibitor Growth Disorder Congenital Adrenal Hyperplasia (CAH)

Additional relevant MeSH terms:


Hyperplasia Adrenal Hyperplasia, Congenital Adrenogenital Syndrome Adrenocortical Hyperfunction Growth Disorders 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 Epinephrine Racepinephrine Epinephryl borate Flutamide Testolactone Deslorelin Adrenergic alpha-Agonists Adrenergic Agonists Adrenergic Agents Neurotransmitter Agents Molecular Mechanisms of Pharmacological Action Physiological Effects of Drugs Adrenergic beta-Agonists Bronchodilator Agents

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