Once-A-Month Steroid Treatment for Patients With Focal Segmental Glomerulosclerosis
Status:
Completed
Trial end date:
2004-12-01
Target enrollment:
Participant gender:
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.
Phase:
Phase 2
Details
Lead Sponsor:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)