Ignite Creation Date:
2024-05-05 @ 11:19 AM
Last Modification Date:
2024-10-26 @ 9:04 AM
Study NCT ID:
NCT00004990
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
2000-03-18
Brief Title:
Once-A-Month Steroid Treatment for Patients With Focal Segmental Glomerulosclerosis
Sponsor:
National Institute of Diabetes and Digestive and Kidney Diseases NIDDK
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Pulse Dexamethasone in Focal Segmental Glomerulosclerosis
Status:
COMPLETED
Status Verified Date:
2004-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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
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
Detailed Description:
The objective of this study is to evaluate the effectiveness and toxicity of an alternative steroid dosing regimen for patients with focal segmental glomerulosclerosis FSGS using a pilot study design that will enroll 20 patients Although the literature reports variable steroid responsiveness remission rates of up to 30-40 percent have recently been reported in nephrotic adult patients treated with daily prednisone at 1 mgkgday for at least 4 months followed by a taper over 3-4 months Such a prolonged and aggressive steroid course is fraught with significant morbidity but this approach has been advocated by some authors because of the poor prognosis for renal survival in nephrotic patients with FSGS who do not achieve remission with steroid treatment We plan to test an eight month course of high dose steroid therapy administered in monthly pulses instead of daily doses for comparable efficacy in achieving remission and for the occurrence of adverse steroid side effects We plan to enroll patients with nephrotic syndrome due to biopsy-proven FSGS who have either not been treated or have responded to conventional steroid dosing regimens and relapsed We plan to treat them with monthly oral pulses of dexamethasone 40-60 mgd x 4 days for 8 months The primary endpoint will be induction of complete remission defined as urine protein less than 300 mgd Patients will also be evaluated for manifestations of steroid toxicity Patients will be seen in follow up at intervals up to 24 months following study entry If this study suggests that remission of nephrotic syndrome can be attained with this regimen and with an acceptable toxicity profile we will plan a randomized controlled trial of this regimen compared with daily or alternate day oral steroids
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 00-DK-0100 | None | None | None |