Status:
COMPLETED
Pilot Studies of Novel Therapies to Treat Resistant Focal Segmental Glomerulosclerosis (FSGS)
Lead Sponsor:
Northwell Health
Collaborating Sponsors:
University of North Carolina
The Cleveland Clinic
Conditions:
Focal Glomerulosclerosis
Eligibility:
All Genders
2-40 years
Phase:
PHASE1
Brief Summary
The current management of primary FSGS is predicated on the assumption that the disease is caused by an immune-mediated disturbance in glomerular barrier function. Therefore, most treatment protocols ...
Detailed Description
Description of study visits Screening Visit: Eligibility Studies 1. History and physical examination 2. Urine protein and creatinine excretion. Proteinuria (Up/c) will be expressed as the protein:cr...
Eligibility Criteria
Inclusion
- Inclusion Criteria:
- Aged 2-42 years at onset of proteinuria
- Aged ≤ 42 years at time of randomization (randomization date before 43rd birthday)
- Estimated glomerular filtration rate (GFR) ≥ 40 ml/min/1.73 m2 at most recent measurement prior to randomization
- For patients \< age 18 years: Schwartz formula
- For patients ≥ age 18 years: Cockroft-Gault formula
- Up/c \> 1.0 g/g creatinine on first morning void at time of randomization
- Biopsy confirmed as primary FSGS (including all subtypes) by study pathologist.
- Steroid resistance: During the last treatment course with high dose steroids prior to randomization, the patient must have demonstrated steroid resistance defined below and not have had a complete remission of proteinuria (Up/c \< 0.2 or dipstick urine protein negative/trace) subsequently. The course of steroid treatment that defines resistance must be the same or equivalent to at least 4 weeks of every day dosing with a minimum cumulative dose of 56 mg/kg or 1680 mg of prednisone or its equivalent.
- May be taking angiotensin-converting enzyme inhibitor (ACEI), angiotensin receptor blocking agent (ARB), vitamin E, or lipid lowering therapy
- Willingness to comply with clinical trial protocol, medications, and follow-up visits, etc.
- Screen failure in FSGS-CT based on prior treatment with excluded medication
- Treatment failure in FSGS-CT based on failure to achieve remission after 26 weeks or 52 weeks of test therapy, i.e., cyclosporine or mycophenolate mofetil (MMF) + oral dexamethasone pulses
- Exclusion Criteria
- Secondary FSGS
- Treated with cyclophosphamide, chlorambucil, levamisole, methotrexate, nitrogen mustard, or other immunosuppressive medications in the 30 days prior to randomization
- Lactation, pregnancy, or refusal of birth control in women of child bearing potential
- Participation in another therapeutic trial concurrently or for 30 days prior to randomization
- Active/serious infection (including, but not limited to hepatitis B or C, HIV)
- Malignancy
- Systemic lupus erythematosus (SLE) or multiple sclerosis
- Hepatic disease defined as serum AST/ALT \> 2.5X the upper limit of normal
- Patients with blood pressure \> 140/95 or \> 95th percentile for age/height while receiving maximal doses of 3 or more antihypertensive agents.
- Diabetes mellitus (DM) type I or II.
- Hematocrit \< 30%
- Organ transplantation
- Obesity (based on estimated dry weight at disease onset prior to steroid therapy) defined as:
- Body mass index (BMI) \> 97th percentile for age if aged 2-20 years
- BMI \> 40 kg/m2 if aged ≥ 21 years
- Allergy to study medications
- Inability to consent/assent
Exclusion
Key Trial Info
Start Date :
July 1 2005
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 1 2007
Estimated Enrollment :
21 Patients enrolled
Trial Details
Trial ID
NCT00193648
Start Date
July 1 2005
End Date
October 1 2007
Last Update
October 22 2007
Active Locations (2)
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1
Howard Trachtman
New Hyde Park, New York, United States, 11040
2
Debbie Gipson
Chapel Hill, North Carolina, United States, 27599-7155