Clinical Trial: Steroid Treatment for Kidney Disease

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Pulse Dexamethasone Over 48 Weeks for Podocyte Disease

Brief Summary:

Focal segmental glomerulosclerosis (FSGS) and minimal change disease are kidney diseases that are associated with increased excretion of protein in the urine. Approximately half of FSGS patients will lose kidney function within 8 years of diagnosis and will require dialysis. The purpose of this study is to determine whether intermittent oral steroid therapy can cause sustained remission of FSGS and MCD.

Approximately 70 participants, including adults and children older than age 2, will be enrolled in this study. They will receive 48 doses of oral dexamethasone over a period of 48 weeks. One group will take two daily doses every 2 weeks; the other group will take four daily doses every 4 weeks. Doctors will monitor participants before, during, and after the steroid treatment with extensive exams and testing. At the completion of the study, researchers will evaluate the safety and efficacy of the drug treatment.


Detailed Summary:

The major causes of primary nephritic syndrome in adults and children are idiopathic podocyte diseases, minimal change (MCD) and focal segmental glomerulosclerosis (FSGS). Our objective is to determine whether intermittent oral dexamethasone administered over 48 weeks can induce complete remission in these patients. This is an open-label multi-center pilot study designed to obtain preliminary evidence of efficacy and to establish safety. This is part of a long-term effort to define the most effective mode of administering pulse dexamethasone and is expected to lead to a trial comparing daily prednisone to pulse dexamethasone.

We will enroll up to 70 patients with nephritic-level proteinuria due to biopsy-proven MCD (up to 30 patients) or FSGS (up to 40 patients). We will include adults and children greater than 2.0 years of age. Children with MCD must have received a minimum of 4 weeks and a maximum of 10 weeks of high-dose daily steroids, since many children are responsive to short courses of daily steroids; these requirements will define a steroid-resistant population. For children with FSGS and adults with MCD or FSGS, there is no minimum duration of prior steroids and there is a maximum of 8 weeks of prior high-dose daily steroids; these requirements will define a population that has received a short steroid course without response. If steroids have been used, inclusion criteria require persistent nephrotic syndrome (thus excluding steroid-sensitive nephrotic syndrome, whether steroid-dependent or frequently relapsing).

Patients may enroll at NIH or at collaborating centers. Those patients who enroll at NIH will visit the NIH Clinical Center at least 4 times. Patients enrolled at collaborating centers have the option to come to the NIH Clinical Center to complete research tests; under these circumstances they will be e
Sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Current Primary Outcome: Remission Status of Patients After Intermittent Oral Dexamethasone Administered Over 48 Weeks [ Time Frame: 48 weeks from baseline ]

Complete remission is defined as proteinuria <0.3 g/d. Partial remission is defined as a 50% fall in proteinuria compared to baseline, proteinuria <3.5 g/d, and a preserved estimated glomerular filtration rate (eGFR), specified as >60% of baseline. Limited response is defined as a 50% fall in proteinuria compared to baseline. All other outcomes are described as non-response.


Original Primary Outcome:

Current Secondary Outcome:

  • Urine Protein [ Time Frame: 48 weeks from baseline ]
  • CKD-EPI eGFR [ Time Frame: 48 weeks from baseline ]
    Estimate glomerular filtration rate (eGFR) using the CKD-EPI (Chronic Kidney Disease Epidemiology Collaboration) formula.


Original Secondary Outcome:

Information By: National Institutes of Health Clinical Center (CC)

Dates:
Date Received: July 28, 2003
Date Started: July 2003
Date Completion:
Last Updated: September 20, 2011
Last Verified: September 2011