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A Study of Fresolimumab in Patients With Steroid-Resistant Primary Focal Segmental Glomerulosclerosis (FSGS)

Phase 2
Completed
Conditions
Primary Focal Segmental Glomerulosclerosis
Interventions
Drug: Placebo
Registration Number
NCT01665391
Lead Sponsor
Genzyme, a Sanofi Company
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

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
36
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.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboPlacebo-
fresolimumab 1 mg/kg total body weightfresolimumab-
fresolimumab 4 mg/kg total body weightfresolimumab-
Primary Outcome Measures
NameTimeMethod
Number of patients reporting adverse events (AEs), serious adverse events (SAEs), and medical events of interest (MEOIs)Up to Day 112
Percentage of patients achieving partial remission (PR) or complete remission (CR) in urinary protein: creatinine ratio (Up/c ratio)Up to Day 112
Secondary Outcome Measures
NameTimeMethod
Percentage of patients achieving CR in Up/c ratioUp to Day 112
Percentage of patients achieving PR in Up/c ratioUp to Day 112
Change from baseline in eGFR (estimated glomerular filtration rate)Up to Day 112
Change from baseline in Up/c ratio and urinary protein excretion rateUp to Day 112
Time to first PR or CRUp to Day 112
Percentage of patients achieving PR or CR with stable eGFR (estimated glomerular filtration rate)Up to Day 112
Mean Fresolimumab serum concentration at each sample collection time pointUp to Day 252

Trial Locations

Locations (32)

Investigational Site Number 4009

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San Francisco, California, United States

Investigational Site Number 4004

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Baltimore, Maryland, United States

Investigational Site Number 4005

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Atlanta, Georgia, United States

Investigational Site Number 4003

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New York, New York, United States

Investigational Site Number 4019

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Springfield, Illinois, United States

Investigational Site Number 4002

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Bethesda, Maryland, United States

Investigational Site Number 4018

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Philadelphia, Pennsylvania, United States

Investigational Site Number 4008

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Birmingham, Alabama, United States

Investigational Site Number 4016

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New York, New York, United States

Investigational Site Number 4022

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Boston, Massachusetts, United States

Investigational Site Number 4025

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Detroit, Michigan, United States

Investigational Site Number 4006

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Rochester, Minnesota, United States

Investigational Site Number 4027

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Houston, Texas, United States

Investigational Site Number 4404

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Bergamo, Italy

Investigational Site Number 4020

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Dallas, Texas, United States

Investigational Site Number 4503

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Barcelona, Spain

Investigational Site Number 4504

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Madrid, Spain

Investigational Site Number 4405

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Reggio Calabria, Italy

Investigational Site Number 4401

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Bari, Italy

Investigational Site Number 4304

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Hamburg, Germany

Investigational Site Number 4502

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Madrid, Spain

Investigational Site Number 4403

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Montichiari, Italy

Investigational Site Number 4024

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St Louis, Missouri, United States

Investigational Site Number 4012

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Ann Arbor, Michigan, United States

Investigational Site Number 4302

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Aachen, Germany

Investigational Site Number 4014

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Rosedale, New York, United States

Investigational Site Number 4013

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Seattle, Washington, United States

Investigational Site Number 4104

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Porto Alegre, Brazil

Investigational Site Number 4023

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Stanford, California, United States

Investigational Site Number 4301

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Düsseldorf, Germany

Investigational Site Number 4029

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Phoenix, Arizona, United States

Investigational Site Number 4001

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Chattanooga, Tennessee, United States

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