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

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ClinicalTrials.gov Identifier: NCT00001521
Recruitment Status : Active, not recruiting
First Posted : November 4, 1999
Last Update Posted : May 29, 2020
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) )

Study Description
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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 (CAH) Drug: Flutamide Drug: Letrozole Phase 2

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
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 62 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
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
Estimated Primary Completion Date : December 31, 2020
Estimated Study Completion Date : December 31, 2020

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Investigational 1
Flutamide, Aromatase inhibitor (testolactone /letrozole) and reduced hydrocortisone dose
 Drug: Letrozole
Armomatase inhibitor.
Experimental: Investigational 2
Flutamide, Aromatase inhibitor (testolactone/letrozole) and reduced hydrocortisone dose
 Drug: Flutamide
Non steroidal antiandrogen that prevents the action of androgens by blocking receptor sites in target tissue. It may also produce changes in testosterone and estradiol


Outcome Measures
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Primary Outcome Measures :
  1. Adult height [ Time Frame: at 13 fpr boys; 14 for girls ]
    Principal outcome variable for long term study will be final adult height, body mass index, and bone mineral density.


Secondary Outcome Measures :
  1. hormone levels (plasma, urine) [ Time Frame: at study conclusion and analysis ]
  2. Predicted adult height (Bayley-Pinneau) [ Time Frame: at study conclusion and analysis ]
  3. Weight velocity (SD units) [ Time Frame: at study conclusion and analysis ]
  4. Growth velocity (SD units) [ Time Frame: at study conclusion and analysis ]

Eligibility Criteria
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Ages Eligible for Study:   2 Years to 18 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
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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
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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
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Principal Investigator: Deborah P Merke, M.D. National Institutes of Health Clinical Center (CC)
More Information
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Additional Information:

Publications:

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Responsible Party: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
ClinicalTrials.gov Identifier: NCT00001521    
Other Study ID Numbers: 960033
96-CH-0033
First Posted: November 4, 1999    Key Record Dates
Last Update Posted: May 29, 2020
Last Verified: December 3, 2019
Keywords provided by National Institutes of Health Clinical Center (CC) ( Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) ):
Congenital Adrenal Hyperplasia (CAH)
Intervention
Children
Additional relevant MeSH terms:
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Adrenal Hyperplasia, Congenital
Adrenogenital Syndrome
Adrenocortical Hyperfunction
Hyperplasia
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
Letrozole
Flutamide
Antineoplastic Agents
Aromatase Inhibitors
Steroid Synthesis Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Estrogen Antagonists
Hormone Antagonists
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Androgen Antagonists
Antineoplastic Agents, Hormonal