Focal Segmental Glomerulosclerosis Clinical Trial (FSGS-CT)
Glomerulosclerosis, Focal
About this trial
This is an interventional treatment trial for Glomerulosclerosis, Focal focused on measuring Focal Segmental Glomerulosclerosis
Eligibility Criteria
Inclusion Criteria: Age 2-40 years at onset of signs or symptoms of FSGS Age ≤ 40 years at time of randomization (randomization date before 41st birthday) Estimated GFR ≥ 40 ml/min/1.73 m2 at most recent measure prior to randomization For participants < age 18 years: Schwartz formula For participants ≥ age 18 years: Cockroft-Gault formula Up/c > 1.0 g protein/g creatinine on first am void at time of randomization Biopsy confirmed as primary FSGS (including all subtypes) by study pathologist. A minimum of 1 glomerulus demonstrating segmental sclerosis on light microscopy will be required to confirm the diagnosis. Steroid resistance: The participant must have demonstrated steroid resistance (defined as a failure to achieve a sustained Up/c ≤ 1.0) based on at least one treatment course with high dose steroids prior to randomization which satisfies both of the following conditions: minimal treatment duration of 4 weeks minimum cumulative dose of 56 mg/kg or 1680 mg of prednisone or its equivalent. In addition, the participant must not have had a complete remission of proteinuria (Up/c < 0.2 or dipstick urine protein 0/trace) subsequent to the latest qualifying 4-week course demonstrating steroid resistance. Willingness to follow the clinical trial protocol, including medications, and baseline and follow-up visits and procedures. Participants may be taking ACEI, ARB, Vitamin E, or lipid lowering therapy. Exclusion Criteria: Secondary FSGS Prior therapy with sirolimus, CSA, tacrolimus, MMF, or azathioprin (Imuran) Treated with cytoxan, chlorambucil, levamisole, methotrexate, or nitrogen mustard in the last 30 days Lactation, pregnancy, or refusal of birth control in women of child bearing potential Participation in another therapeutic trial concurrently or 30 days prior to randomization Active/serious infection (including, but not limited to Hepatitis B, C, or HIV) Malignancy Blood pressure > 140/95 or > 95th percentile for age/height. Participant is receiving 4 or more antihypertensive agents for the primary purpose of controlling blood pressure. Participants with previously diagnosed diabetes mellitus type I or II: the diagnosis of DM I or II will be based on local criteria for participants with an established diagnosis. If hyperglycemia is detected during the screening period, the WHO criteria for the diagnosis of DM I and II will be used. Clinical evidence of cirrhosis or chronic active liver disease Abnormal laboratory values at the time of study entry: Absolute neutrophil count (ANC) < 2000/mm3, or Hematocrit (HCT) < 28% History of significant gastrointestinal disorder, e.g, severe chronic diarrhea (> 5 watery stools per day) or active peptic ulcer disease. Organ transplantation Obesity (based on estimated dry weight at onset of disease prior to steroid therapy) defined as BMI > 97th percentile for age if aged 2-20 years BMI > 40 kg/m2 for age ≥21 years Allergy to study medications Inability to consent/assent Note: Participants with conditions meeting exclusion criteria at a particular evaluation for eligibility may be re-evaluated at a later time to determine if the conditions have changed so that all entry criteria are met. In particular, if blood pressure > 140/95 or > 95th percentile for age/height while the participant is on less than three antihypertensive agents, the participant may be re-evaluated for eligibility after adding other antihypertensive agents so long as the total number of agents does not exceed three.
Sites / Locations
- Data Coordinating Center; Cleveland Clinic Foundation; Quantitative Health Sciences; 9500 Euclid Avenue
Arms of the Study
Arm 1
Arm 2
Active Comparator
Active Comparator
1
2
Cyclosporin
MMF and Dexamethasone