Primary Focal Segmental Glomerulosclerosis
Conditions
Brief summary
The primary objectives of this trial are as follows: * to compare the achievement of a partial remission (PR) or complete remission (CR) in urinary protein: creatinine ratio (Up/c ratio) in patients treated with fresolimumab versus placebo * to compare the safety profile of patients treated with fresolimumab versus placebo The secondary objectives are as follows: * To compare the reduction in proteinuria in patients treated with fresolimumab versus placebo * To evaluate fresolimumab dose-dependent reduction in proteinuria * To compare the change in renal function (estimated glomerular filtration rate \[eGFR\]) in patients treated with fresolimumab versus placebo * To evaluate the multiple-dose pharmacokinetics of fresolimumab
Interventions
DRUGfresolimumab
1 mg/kg total body weight administered intravenous (IV)
DRUGPlacebo
Placebo administered to match active treatment group
Sponsors
Genzyme, a Sanofi Company
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* The patient's renal biopsy is consistent with the diagnosis of primary Focal Segmental Glomerulosclerosis (FSGS) including all histological subtypes. * The patient has an eGFR ≥ 30 mL/min/1.73 m2 * The patient has a urinary total protein:creatinine ratio ≥ 3 mg protein/mg creatinine * In the opinion of the Investigator, the patient has steroid-resistant FSGS. The patient must have been treated for FSGS with a course of high-dose steroid therapy for a minimum of 4 weeks * The patient has been treated with an ACEi (angiotensin converting enzyme inhibitor) and/or ARB (angiotensin receptor blocker) at a stable dose for a minimum of 4 weeks prior to Visit 2 (treatment start)
Exclusion criteria
* The patient has FSGS which in the Investigator's opinion is secondary to another condition * The patient has been taking prednisone at a dose \> 10 mg/day (or equivalent dose of an alternative glucocorticoid) within 4 weeks prior to Visit 1 (Screening Visit). * The patient has received any other systemically administered immunosuppressive drugs (other than glucocorticoids) within 8 weeks prior to Visit 1. * The patient has received rituximab within 6 months prior to Visit 1. * The patient has a history of organ transplantation.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Percentage of patients achieving partial remission (PR) or complete remission (CR) in urinary protein: creatinine ratio (Up/c ratio) | Up to Day 112 |
| Number of patients reporting adverse events (AEs), serious adverse events (SAEs), and medical events of interest (MEOIs) | Up to Day 112 |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline in eGFR (estimated glomerular filtration rate) | Up to Day 112 |
| Percentage of patients achieving CR in Up/c ratio | Up to Day 112 |
| Percentage of patients achieving PR or CR with stable eGFR (estimated glomerular filtration rate) | Up to Day 112 |
| Change from baseline in Up/c ratio and urinary protein excretion rate | Up to Day 112 |
| Time to first PR or CR | Up to Day 112 |
| Percentage of patients achieving PR in Up/c ratio | Up to Day 112 |
| Mean Fresolimumab serum concentration at each sample collection time point | Up to Day 252 |
Countries
Brazil, Germany, Italy, Spain, United States
Outcome results
None listed