Three Drug Combination Therapy Versus Conventional Treatment of Children With Congenital Adrenal Hyperplasia
Three Drug Combination Therapy Versus Conventional Treatment of Children With Congenital Adrenal Hyperplasia
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>
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.
-  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).