Flutamide for Adrenal Hyperplasia, Congenital

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, MD
Adrenal Hyperplasia, Congenital+4 More
Flutamide - Drug
Eligibility
< 65
All Sexes
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Study Summary

This study is evaluating whether a combination of four drugs can help children with congenital adrenal hyperplasia grow normally.

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Eligible Conditions

  • Adrenal Hyperplasia, Congenital
  • Congenital Adrenal Hyperplasia (CAH)

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Adrenal Hyperplasia, Congenital

Study Objectives

This trial is evaluating whether Flutamide will improve 1 primary outcome and 4 secondary outcomes in patients with Adrenal Hyperplasia, Congenital. Measurement will happen over the course of at 13 for boys; 14 for girls.

at 13 for boys; 14 for girls
Adult height
at study conclusion and analysis
Growth velocity (SD units)
Predicted adult height (Bayley-Pinneau)
Weight velocity (SD units)
hormone levels (plasma, urine)

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for Adrenal Hyperplasia, Congenital

Trial Design

2 Treatment Groups

Investigational 1
1 of 2
Investigational 2
1 of 2
Experimental Treatment

This trial requires 62 total participants across 2 different treatment groups

This trial involves 2 different treatments. Flutamide is the primary treatment being studied. Participants will be divided into 2 treatment groups. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Investigational 1boys/flutamide, letrozole, and reduced hydrocortisone dose or conventional treatment (with hydrocortisone and fludrocortisone ) until the age of 14
Investigational 2girls/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
First Studied
Drug Approval Stage
How many patients have taken this drug
Hydrocortisone
FDA approved
Letrozole
FDA approved
Fludrocortisone
FDA approved
Flutamide
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: at study conclusion and analysis
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly at study conclusion and analysis for reporting.

Closest Location

National Institutes of Health Clinical Center, 9000 Rockville Pike - Bethesda, MD

Eligibility Criteria

This trial is for patients born any sex aged 65 and younger. There are 3 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
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.

Patient Q&A Section

Can adrenal hyperplasia, congenital be cured?

"A combination of metyrapone, fludrocortisone, and chlorotrianisene is effective in the treatment of familial and neonatal cases of primary aldosteronism, but the role of surgery to treat disease in other ages can be doubtful. The exact role and timing of treatment may need to be individualized in order to maximize the extent of tumor reduction while maintaining the patient's life and preserving the adrenal gland from further damage." - Anonymous Online Contributor

Unverified Answer

Is flutamide typically used in combination with any other treatments?

"Flutamide has been the only drug used or proposed for use on a regular basis for the treatment of hirsutism for a number of years. These data are similar to those published by the United States Food and Drug Administration in the absence of safety monitoring. The inclusion of flutamide in the treatment of hirsutism on a more regular basis may be a cost-effective solution to a problem which is significant to several societies." - Anonymous Online Contributor

Unverified Answer

What is adrenal hyperplasia, congenital?

"Adrenal hyperplasia, congenital can be described as an accumulation or overactivity of an adrenal gland that releases certain hormones during infancy or early childhood (e.g., cortisol in the blood). The symptoms and signs of adrenal hyperplasia, congenital can vary, depending on which of the adrenocortical glands is affected." - Anonymous Online Contributor

Unverified Answer

What causes adrenal hyperplasia, congenital?

"There is a high percentage of adrenal hyperplasia, congenital in patients with isolated growth hormone deficient dwarfism. The clinical picture, in other words the clinical picture of the disease, and the hormonal assay can suggest a diagnosis, but a precise demonstration of a specific gene for a case of isolated growth hormone-deficient dwarfism needs to be made." - Anonymous Online Contributor

Unverified Answer

How many people get adrenal hyperplasia, congenital a year in the United States?

"1.3 million individuals in the United States are diagnosed with adrenal hyperplasia. This makes up 21% of the total population of pediatric endocrinology in the United States." - Anonymous Online Contributor

Unverified Answer

What are common treatments for adrenal hyperplasia, congenital?

"Corticosteroids are most effective in the treatment of congenital adrenal hyperplasia, and often reduce the need for surgical correction. Corticosteroids are often prescribed with anabolic steroid and mineralocorticoid blockade. Steroids are often used to treat hyperprostasemia, with or without adrenal atrophy. Mineralocorticoid blockers may be used alone and in combinations with other drugs." - Anonymous Online Contributor

Unverified Answer

What are the signs of adrenal hyperplasia, congenital?

"Signs of adrenal insufficiency may include weakness, pale skin, pallor and increased thirst, urination, fatigue, lack of appetite, diarrhea, constipation and vomiting. In severe cases, shock may occur.\n" - Anonymous Online Contributor

Unverified Answer

Have there been other clinical trials involving flutamide?

"There have been few other clinical trials involving flutamide. In those trials, the results suggest that it has been well tolerated but does not seem to improve outcomes. However, because we do not have a large body of clinical studies, we cannot assess the potential effects of flutamide in terms of safety. This question will have to be asked in future studies using flutamide." - Anonymous Online Contributor

Unverified Answer

How does flutamide work?

"The present study demonstrates that flutamide causes a decrease in sex hormone levels. In addition, it reduces gonadal and adrenal androgen levels, while there is an increment in the levels of cortisol. flutamide also reverses hyperprolactinemia in male rats. Results from a recent paper also show that flutamide has a direct negative effect on prolactin secretion in the rat anterior pituitary gland. Taken together, these results indicate that flutamide causes an androgen depletion in the gonads and adrenals and is also able to reduce prolactin production. Moreover, it seems that flutamide influences the dopaminergic neuron, possibly mediated by its action on the gonadal and adrenal steroid levels." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating adrenal hyperplasia, congenital?

"[Adrenal hyperplasia, congenital is the most common type of hyperfunction and it is also more serious as the defect in adrenal secretion is congenital or in part. Patients should be diagnosed early to give them effective treatment and prevention of complications] according to previous study [(3)3] answer: None of our recent findings is new for treating adrenal hyperplasia, congenital. It is important to note that adrenal hyperplasia, congenital patients could also have other problems besides adrenal hyperplasia, congenital such as primary or secondary hyperaldosteronism, and Addison's disease." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets adrenal hyperplasia, congenital?

"Adrenal hyperplasia presents on average for younger children (about 1 year old). Symptoms usually appear within the first year of life. More information is available on adrenal hyperplasia at [U.S. National Cancer Institute] (http://www.hbpcdc.nih.gov/about/about/hbpcdc/about/hbpcdc/about/about_pregnancy.htm) and [U.S. National Library of Medicine's] (http://www.nlm.nih.gov/about/medical/conditions/about_adrenal_hyp.html)." - Anonymous Online Contributor

Unverified Answer

Does adrenal hyperplasia, congenital run in families?

"Adrenal hyperplasia, congenital has a autosomal dominant inheritance pattern. We suggest that adrenal hyperplasia, congenital (ADHOC) run in families warrants further investigation involving other siblings of the probands as to whether this is a common occurrence in adrenal hyperplasia, congenital." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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