Trial record 5 of 50 for:    "Adrenal Hyperplasia, Congenital" [DISEASE]

Research Study for Children With Salt Wasting Congenital Adrenal Hyperplasia

This study has been completed.
Sponsor:
Information provided by:
Baylor College of Medicine
ClinicalTrials.gov Identifier:
NCT00529841
First received: September 12, 2007
Last updated: March 23, 2009
Last verified: March 2009
  Purpose

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 Intervention
Adrenal Hyperplasia, Congenital
Drug: Hydrocortisone sodium acetate

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Novel Therapeutic Modality for Congenital Adrenal Hyperplasia

Resource links provided by NLM:


Further study details as provided by Baylor College of Medicine:

Primary Outcome Measures:
  • Serum 17-OHP concentration in the morning [ Time Frame: 11 days ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • serum steroid hormone profiles [ Time Frame: 11 days ] [ Designated as safety issue: No ]
  • serum blood glucose [ Time Frame: study days 2,3 and 11 ] [ Designated as safety issue: Yes ]
  • serum sodium [ Time Frame: study days 2,3 and 11 ] [ Designated as safety issue: Yes ]

Enrollment: 7
Study Start Date: January 2007
Study Completion Date: September 2008
Primary Completion Date: September 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
Subcutaneous administration of medication via insulin pump
Drug: Hydrocortisone sodium acetate
Subcutaneous administration of medication via insulin pump
Other Name: Solu-Cortef injection

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.

  Eligibility

Ages Eligible for Study:   3 Years to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00529841

Locations
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
Investigators
Principal Investigator: Morey W Haymond, MD Baylor College of Medicine
  More Information

Publications:
Gordon B.Cutler et al. Congenital Adrenal Hyperplasia due to 21-Hydroxylase Deficiency NEJM 1990 Vol 323, No 26, 1806-1813
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

Responsible Party: Morey W.Haymond MD Professor of Pediatrics;Chief. Endocrine and Metabolism Section; Vice Chairman for Research, Baylor College of Medicine
ClinicalTrials.gov Identifier: NCT00529841     History of Changes
Other Study ID Numbers: H-19704, GCRC # 0962
Study First Received: September 12, 2007
Last Updated: March 23, 2009
Health Authority: United States: Institutional Review Board

Keywords provided by Baylor College of Medicine:
Salt Wasting Congenital Adrenal Hyperplasia
Subcutaneous Hydrocortisone

Additional relevant MeSH terms:
Adrenal Hyperplasia, Congenital
Adrenogenital Syndrome
Adrenocortical Hyperfunction
Hyperplasia
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
Pathologic Processes
Cortisol succinate
Hydrocortisone acetate
Hydrocortisone 17-butyrate 21-propionate
Hydrocortisone
Hydrocortisone-17-butyrate
Anti-Inflammatory Agents
Therapeutic Uses
Pharmacologic Actions
Dermatologic Agents

ClinicalTrials.gov processed this record on July 24, 2014