Protocol Number: 00-DK-0100

Title:

Pulse Dexamethasone in Focal Segmental Glomerulosclerosis

Number:

00-DK-0100

Summary:

This study will test the safety and effectiveness of a monthly dosing regimen of dexamethasone-a strong steroid medication-to treat patients with focal segmental glomerulosclerosis (FSGS). Patients with this disease have kidney fibrosis (scarring) and proteinuria (excessive excretion of protein in the urine) that, in about half of the patients eventually requires kidney dialysis or transplant. Currently, the most effective treatment for FSGS is high-dose steroids (prednisone) taken daily for 4 to 6 months. However, only about 30 percent of patients respond to this treatment, and it causes serious side effects in many patients. Other drugs, such as cyclosporin and cyclophosphamide, improve proteinuria in even fewer patients (about 10 percent) and also have serious side effects. This study will explore whether a monthly pulse dose of steroids will achieve disease remission with less toxicity.

Adults and children with FSGS who: 1) have not received steroid treatment, or 2) could not tolerate daily steroid treatment, or 3) relapsed after conventional steroid treatment may be eligible for this study. Those enrolled will take dexamethasone by mouth for 4 days every 4 weeks for a total of 8 months. Patients will undergo various tests before treatment starts (baseline), during the course of treatment, and in follow-up visits to evaluate the effects of treatment as follows:

1. Review of kidney biopsy, medical evaluation, measurement of total daily urine protein excretion and kidney function, psychiatric testing for depression or other mood disorder

2. Measurements of blood pressure, blood chemistries and urine protein excretion - monthly during treatment

3. Questionnaire about the effects of treatment, if any, on mood and feelings - monthly during treatment

4. Photographs of the face and body (in underwear or shorts and tank top) to evaluate body fat distribution- baseline and 8 months

5. Eye examinations for cataracts and glaucoma - baseline and 8 months

6. Bone density scan (DEXA scan) of the lower spine and hip - baseline, 4 and 12 months

7. Magnetic resonance imaging (MRI) of the hips

8. Psychological evaluation and quality of life evaluation - baseline, 1, 2 and 8 months

9. Blood tests for adrenal gland function - baseline, 4 and 8 months

10. Blood and urine tests - 10, 12, 15, and 18 months

Patients who achieve remission (whose urine protein levels decrease to normal) before completing the 8 months of dexamethasone will take one more dose and then stop therapy, but continue with follow-up. Patients who achieve remission but relapse may be offered a second course of treatment.

Sponsoring Institute:

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Recruitment Detail

Type: Follow-up Of Previously Enrolled Subjects Only

Gender: Male & Female

Referral Letter Required: No

Population Exclusion(s): None

Eligibility Criteria: This study is not currently recruiting new subjects. If you have questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.

Special Instructions: Currently Not Provided

Disease Category:

PROTICD

Keywords:

Nephrotic Syndrome

Steroids

Osteoporosis

Adrenal Suppression

Proteinuria

Recruitment Keywords:

Focal Segmental Glomerulosclerosis

Conditions:

Glomerulonephritis

Nephrotic Syndrome

Investigational Drug(s):

None

Investigational Device(s):

None

Contacts:

This study is not currently recruiting new subjects. If you have questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.

Citations:

High-dose oral dexamethasone therapy for chronic childhood idiopathic thrombocytopenic purpura

Primary dexamethasone treatment of multiple myeloma

Response of resistant idiopathic thrombocytopenic purpura to pulsed high-dose dexamethasone therapy

• Questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.
• Technical questions regarding the Clinical Center web site, please contact the Department of Networks and Applications, CC.

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Warren Grant Magnuson Clinical Center (CC)
National Institutes of Health (NIH)
Bethesda, Maryland 20892. Last update: 10/23/2004