Pulse Dexamethasone in Focal Segmental Glomerulosclerosis
Dexamethasone
Urogenital Diseases+19
+ Bone Diseases
+ Bone Diseases, Metabolic
Treatment Study
Summary
Study start date: March 1, 2000
Actual date on which the first participant was enrolled.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 mg/kg/day 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 mg/d x 4 days), for 8 months. The primary endpoint will be induction of complete remission, defined as urine protein less than 300 mg/d. 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.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.20 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
Criteria
INCLUSION CRITERIA: Adults and children. Biopsy proven FSGS, including idiopathic FSGS and collapsing FSGS, but excluding HIV-associated FSGS and secondary FSGS associated with morbid obesity, sickle cell anemia, reflux nephropathy, chronic tubular injury, congenital renal anomalies, and reduced nephron mass. Glomerular filtration rate will be estimated using 4 variable MDRD GFR equation, which incorporates age, race, gender, and serum creatinine. If disease has been present less than or equal to 1 year, estimated GFR must be greater than or equal to 40 ml/min. If disease has been present greater than or equal to 1 year, estimated GFR must be greater than or equal to 60 ml/min. Nephrotic range proteinuria, defined as urine protein greater than or equal to 3.5 g/1.73 m(2)/d. Patients who received steroids for FSGS and who entered complete remission but have relapsed with nephrotic range proteinuria will be eligible. If hypertensive, adequate blood pressure control (target BP less than or equal to 130/80 mm Hg at greater than 75% of measurement in adults). Patients must have been taking an angiotensin converting enzyme inhibitor or angiotensin receptor antagonist for at least 6 weeks, unless intolerant of these medications. Women with child-bearing potential must maintain an effective birth control regimen (oral contraceptive, intrauterine device, barrier plus spermicide). EXCLUSION CRITERIA: Inability to give informed consent or cooperate with study. Poorly controlled diabetes (as defined by hemoglobin A1C of greater than or equal to 8.5 on entry screening, or daily insulin requirement of greater than or equal to 100 units) or hypertension (defined as systolic BP consistently greater than 160 and/or diastolic BP consistently greater than 100 on three or more drugs). Evidence of chronic or occult infection. Specifically, must not have evidence of active hepatitis B, hepatitis C, HIV or untreated mycobacterial infection. Current or prior use of cytotoxic agents or cyclosporin for FSGS. If previously treated with these agents for a non-renal indication, therapy must have concluded at least 12 months prior to study. Existence of any other condition which would complicate the implementation or interpretation of the study. History of steroid-induced psychiatric disorder, known avascular necrosis of hip or symptomatic osteoporosis (e.g., known compression fractures), brittle diabetes mellitus, or glaucoma. Patients with other psychiatric disorders will be evaluated on a case by case basis. Patients who have been treated with steroids equivalent to 1 mg/kg/day for greater than or equal to 8 weeks without remission will be excluded.
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 1 location
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Bethesda, United StatesOpen National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) in Google Maps