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 : June 24, 2022
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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 Drug: Hydrocortisone Drug: Fludrocortisone | Phase 2 |
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 |
Actual Study Start Date : | June 8, 1995 |
Estimated Primary Completion Date : | December 31, 2022 |
Estimated Study Completion Date : | December 31, 2022 |

Arm | Intervention/treatment |
---|---|
Experimental: Investigational 1
boys/flutamide, letrozole, and reduced hydrocortisone dose or conventional treatment (with hydrocortisone and fludrocortisone ) until the age of 14
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Drug: Flutamide
Non steroidal anti-androgen that prevents the action of androgens by blocking receptor sites in target tissue. It may also produce changes in testosterone and estradiol Drug: Letrozole Armomatase inhibitor. Drug: Hydrocortisone reduced hydrocortisone dose can normalize the growth and adult stature of children with congenital adrenal hyperplasia Drug: Fludrocortisone Since hydrocortisone dosage will be reduced , some patients may develop symptoms or signs of adrenal insufficiency. Patients will continue to receive an optimal fludrocortisone dose |
Experimental: Investigational 2
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
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Drug: Flutamide
Non steroidal anti-androgen that prevents the action of androgens by blocking receptor sites in target tissue. It may also produce changes in testosterone and estradiol Drug: Letrozole Armomatase inhibitor. Drug: Hydrocortisone reduced hydrocortisone dose can normalize the growth and adult stature of children with congenital adrenal hyperplasia |
- Adult height [ Time Frame: at 13 for boys; 14 for girls ]The primary outcome variable is adult height which will be expressed in SD units relative to the normal population.
- hormone levels (plasma, urine) [ Time Frame: at study conclusion and analysis ]hormone levels
- Predicted adult height (Bayley-Pinneau) [ Time Frame: at study conclusion and analysis ]adult height
- Weight velocity (SD units) [ Time Frame: at study conclusion and analysis ]body mass index, and bone density
- Growth velocity (SD units) [ Time Frame: at study conclusion and analysis ]adult height, body mass index, and bone density

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Ages Eligible for Study: | 2 Years to 18 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
- 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).

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
United States, Maryland | |
National Institutes of Health Clinical Center, 9000 Rockville Pike | |
Bethesda, Maryland, United States, 20892 |
Principal Investigator: | Deborah P Merke, M.D. | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) |
Publications:
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: | June 24, 2022 |
Last Verified: | September 29, 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Undecided |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Congenital Adrenal Hyperplasia (CAH) Intervention Children |
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 |
Hydrocortisone Fludrocortisone 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 Anti-Inflammatory Agents Androgen Antagonists |