Status:
COMPLETED
Focal Segmental Glomerulosclerosis Clinical Trial (FSGS-CT)
Lead Sponsor:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Collaborating Sponsors:
The Cleveland Clinic
Conditions:
Glomerulosclerosis, Focal
Eligibility:
All Genders
2-40 years
Phase:
PHASE3
Brief Summary
The FSGS Clinical Trial is a multi-center, prospective, controlled, open label randomized trial designed to determine if treatment with mycophenolate mofetil (MMF) in conjunction with pulse steroids i...
Detailed Description
Background/Rationale. Primary FSGS is a leading cause of end stage renal disease in both children and adults, with complete loss of kidney function in 50% of patients over 10 years. Evidence-based tre...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
November 1 2004
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
October 1 2009
Estimated Enrollment :
207 Patients enrolled
Trial Details
Trial ID
NCT00135811
Start Date
November 1 2004
End Date
October 1 2009
Last Update
May 22 2012
Active Locations (1)
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1
Data Coordinating Center; Cleveland Clinic Foundation; Quantitative Health Sciences; 9500 Euclid Avenue
Cleveland, Ohio, United States, 44195-5196