Research Study for Children With Salt Wasting Congenital Adrenal Hyperplasia

Adrenal Hyperplasia, Congenital Clinical Trial

Official title:

A Novel Therapeutic Modality for Congenital Adrenal Hyperplasia

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00529841
• Other study ID #: H-19704
• Secondary ID: GCRC # 0962
• Status: Completed
• Phase: N/A
• First received: September 12, 2007
• Last updated: September 10, 2015
• Start date: January 2007
• Est. completion date: September 2008

Study information

• Verified date: September 2015
• Source: Baylor College of Medicine
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to develop a more physiological approach to the management of children and adolescents with salt wasting Congenital Adrenal Hyperplasia.

We will administer the glucocorticosteroid via insulin infusion pump to see whether this treatment will improve the serum hormone concentrations.

Description:

The adrenal gland is a small organ of the body. It produces very important chemicals called hormones. One of these hormones, cortisol (the stress hormone) helps the body fight diseases. The other hormone is the aldosterone helps to maintain the normal amount of salt and water in the body. The third type of hormones are the androgens or male hormones, which cause some of the changes during puberty, like the growth of body hair and pimples.

The salt wasting Congenital Adrenal Hyperplasia or CAH disease is a disease of the adrenal gland. Patients with this disease cannot make cortisol or the aldosterone. As a result, their body cannot fight diseases and cannot keep normal amounts of salt and water in the body. At the same time, the gland makes too much of the male hormones, which is bad for the body because too much male hormone slows down growth, increases the growth of body hair, and causes pimples and abnormal period in girls.

Patients with this disease have to take medications every day. However, the treatment does not work very well, because usually the patients do not have the right amount of hormone in their body. Usually the body gets too much hormone right after taking the pills. A couple of hours later the body has too little of the hormones, because in the meantime the body gets rid of the medication.The healthy adrenal gland makes the hormones throughout the day in different amounts. The patients with this disease take the medication only a couple of times a day. They take the Florinef tablet once a day and the Cortisol tablet two or three times a day. The treatment that we use today by mouth cannot copy the natural hormone productions of the adrenal gland. Because of this it does not make much of a difference in the patient's life.

We would like to improve the treatment and find out the effect of a new treatment. In this study we will try to imitate the body's normal hormone production and will give the medication via an insulin pump to see if this treatment method will decrease the male hormones in the blood. This study will help us to develop a new and better treatment for children and adolescents.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 7
• Est. completion date: September 2008
• Est. primary completion date: September 2008
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 3 Years to 18 Years

Eligibility

Inclusion Criteria:

• Children with salt wasting CAH otherwise healthy without other chronic disease

• Age: between 3 and 18 years of age

• Body weight 23 kg (50 lbs) or above

• Hemoglobin equal to or higher than 12 g/dl before the study

• Supportive family environment

Exclusion Criteria:

• Age less than 3 or older than 18 years at the time of study

• Other chronic disease

• Hemoglobin less than 12 g/dl

• Non-supportive family

• Allergy to local anesthetics

Criteria for study termination: If the subject's parents are unable to manage/operate the pump, the subject will be withdrawn from the study.

Study Design

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Adrenal Hyperplasia, Congenital
• Adrenocortical Hyperfunction
• Adrenogenital Syndrome
• Hyperplasia

Intervention

Drug:
• Hydrocortisone sodium acetate
Subcutaneous administration of medication via insulin pump

Locations

Country	Name	City	State
United States	BCM, Texas Children's Hospital Clinic and General Clinical Research Center	Houston	Texas

Sponsors (1)

Lead Sponsor	Collaborator
Baylor College of Medicine

Country where clinical trial is conducted

United States, 

References & Publications (12)

Claude J.Migeon. Can the Long Range Results of the Treatment of Congenital Adrenal Hyperplasia be improved? JCEM 1996 Vol 81, No 9 3187-3189

Cutler GB Jr, Laue L. Congenital adrenal hyperplasia due to 21-hydroxylase deficiency. N Engl J Med. 1990 Dec 27;323(26):1806-13. Review. — View Citation

Esteban NV, Loughlin T, Yergey AL, Zawadzki JK, Booth JD, Winterer JC, Loriaux DL. Daily cortisol production rate in man determined by stable isotope dilution/mass spectrometry. J Clin Endocrinol Metab. 1991 Jan;72(1):39-45. — View Citation

Kerrigan JR, Veldhuis JD, Leyo SA, Iranmanesh A, Rogol AD. Estimation of daily cortisol production and clearance rates in normal pubertal males by deconvolution analysis. J Clin Endocrinol Metab. 1993 Jun;76(6):1505-10. — View Citation

Laue L, Merke DP, Jones JV, Barnes KM, Hill S, Cutler GB Jr. A preliminary study of flutamide, testolactone, and reduced hydrocortisone dose in the treatment of congenital adrenal hyperplasia. J Clin Endocrinol Metab. 1996 Oct;81(10):3535-9. — View Citation

Lukert BP. Editorial: glucocorticoid replacement--how much is enough? J Clin Endocrinol Metab. 2006 Mar;91(3):793-4. Erratum in: J Clin Endocrinol Metab. 2006 Jun;91(6):2073. — View Citation

Mah PM, Jenkins RC, Rostami-Hodjegan A, Newell-Price J, Doane A, Ibbotson V, Tucker GT, Ross RJ. Weight-related dosing, timing and monitoring hydrocortisone replacement therapy in patients with adrenal insufficiency. Clin Endocrinol (Oxf). 2004 Sep;61(3):367-75. — View Citation

Merza Z, Rostami-Hodjegan A, Memmott A, Doane A, Ibbotson V, Newell-Price J, Tucker GT, Ross RJ. Circadian hydrocortisone infusions in patients with adrenal insufficiency and congenital adrenal hyperplasia. Clin Endocrinol (Oxf). 2006 Jul;65(1):45-50. — View Citation

Sheila K.Gunn et al Subcutaneous Hydrocortisone Delivery mimics Physiologic Cortisol Concentrations, Poster, Endocrine Society Meeting 2000

Speiser PW. Toward better treatment of congenital adrenal hyperplasia. Clin Endocrinol (Oxf). 1999 Sep;51(3):273-4. — View Citation

Wallace WH, Crowne EC, Shalet SM, Moore C, Gibson S, Littley MD, White A. Episodic ACTH and cortisol secretion in normal children. Clin Endocrinol (Oxf). 1991 Mar;34(3):215-21. — View Citation

Winterer J, Chrousos GP, Loriaux DL, Cutler GB Jr. Effect of hydrocortisone dose schedule on adrenal steroid secretion in congenital adrenal hyperplasia. J Pediatr. 1985 Jan;106(1):137-42. — View Citation

* Note: There are 12 references in all — Click here to view all references

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Serum 17-OHP concentration in the morning		11 days	No
Secondary	serum steroid hormone profiles		11 days	No
Secondary	serum blood glucose		study days 2,3 and 11	Yes
Secondary	serum sodium		study days 2,3 and 11	Yes