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Research Study for Children With Salt Wasting Congenital Adrenal Hyperplasia

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ClinicalTrials.gov Identifier: NCT00529841
Recruitment Status : Completed
First Posted : September 14, 2007
Last Update Posted : September 14, 2015
Information provided by (Responsible Party):
Baylor College of Medicine

Brief 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.

Condition or disease Intervention/treatment Phase
Adrenal Hyperplasia, Congenital Drug: Hydrocortisone sodium acetate Not Applicable

Detailed 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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 7 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Novel Therapeutic Modality for Congenital Adrenal Hyperplasia
Study Start Date : January 2007
Actual Primary Completion Date : September 2008
Actual Study Completion Date : September 2008

Arm Intervention/treatment
Experimental: 1 (Hydrocortisone sodium acetate)
Subcutaneous administration of Hydrocortisone sodium acetate via insulin pump
Drug: Hydrocortisone sodium acetate
Subcutaneous administration of medication via insulin pump
Other Name: Solu-Cortef injection

Primary Outcome Measures :
  1. Serum 17-OHP concentration in the morning [ Time Frame: 11 days ]

Secondary Outcome Measures :
  1. serum steroid hormone profiles [ Time Frame: 11 days ]
  2. serum blood glucose [ Time Frame: study days 2,3 and 11 ]
  3. serum sodium [ Time Frame: study days 2,3 and 11 ]

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Ages Eligible for Study:   3 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

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): NCT00529841

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United States, Texas
BCM, Texas Children's Hospital Clinic and General Clinical Research Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
Baylor College of Medicine
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Principal Investigator: Morey W Haymond, MD Baylor College of Medicine
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
Sheila K.Gunn et al Subcutaneous Hydrocortisone Delivery mimics Physiologic Cortisol Concentrations, Poster, Endocrine Society Meeting 2000

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Responsible Party: Baylor College of Medicine
ClinicalTrials.gov Identifier: NCT00529841    
Other Study ID Numbers: H-19704
GCRC # 0962 ( Other Identifier: Baylor College of Medicine )
First Posted: September 14, 2007    Key Record Dates
Last Update Posted: September 14, 2015
Last Verified: September 2015
Keywords provided by Baylor College of Medicine:
Salt Wasting Congenital Adrenal Hyperplasia
Subcutaneous Hydrocortisone
Additional relevant MeSH terms:
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Adrenal Hyperplasia, Congenital
Adrenogenital Syndrome
Adrenocortical Hyperfunction
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 hemisuccinate
Anti-Inflammatory Agents