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

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United States Department of Health and Human Services (HHS)

Status and phase

Completed
Phase 4

Conditions

Congenital Adrenal Hyperplasia (CAH)

Treatments

Drug: Hydrocortisone
Drug: Letrozole
Drug: Flutamide
Drug: Fludrocortisone

Study type

Interventional

Funder types

NIH

Identifiers

NCT00001521
96-CH-0033
960033

Details and patient eligibility

About

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\>...

Full 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.

Enrollment

62 patients

Sex

All

Ages

2 to 18 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion 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).

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Parallel Assignment

Masking

None (Open label)

62 participants in 2 patient groups

Investigational 1
Experimental group
Description:
boys/flutamide, letrozole, and reduced hydrocortisone dose or conventional treatment (with hydrocortisone and fludrocortisone ) until the age of 14
Treatment:
Drug: Fludrocortisone
Drug: Flutamide
Drug: Letrozole
Drug: Hydrocortisone
Investigational 2
Experimental group
Description:
girls/flutamide, letrozole, and reduced hydrocortisone dose, the Letrozole will discontinue at 13 y.o. and continue flutamide until 2 years after menarche or when final height is reached, whichever occurs first
Treatment:
Drug: Flutamide
Drug: Letrozole
Drug: Hydrocortisone

Trial contacts and locations

1

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Data sourced from clinicaltrials.gov

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