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

Purpose

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

Condition	Intervention	Phase
Primary Focal Segmental Glomerulosclerosis	Drug: fresolimumab Drug: Placebo	Phase 2

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Investigator) Primary Purpose: Treatment
Official Title:	A Phase 2, Multicenter, Double-Blind, Parallel Dosing, Randomized Study of Fresolimumab or Placebo in Patients With Steroid-Resistant Primary Focal Segmental Glomerulosclerosis

Further study details as provided by Genzyme:

Primary Outcome Measures:

Percentage of patients achieving partial remission (PR) or complete remission (CR) in urinary protein: creatinine ratio (Up/c ratio) [ Time Frame: Up to Day 112 ] [ Designated as safety issue: No ]
Number of patients reporting adverse events (AEs), serious adverse events (SAEs), and medical events of interest (MEOIs) [ Time Frame: Up to Day 112 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Percentage of patients achieving CR in Up/c ratio [ Time Frame: Up to Day 112 ] [ Designated as safety issue: No ]
Percentage of patients achieving PR in Up/c ratio [ Time Frame: Up to Day 112 ] [ Designated as safety issue: No ]
Change from baseline in Up/c ratio and urinary protein excretion rate [ Time Frame: Up to Day 112 ] [ Designated as safety issue: No ]
Time to first PR or CR [ Time Frame: Up to Day 112 ] [ Designated as safety issue: No ]
Change from baseline in eGFR (estimated glomerular filtration rate) [ Time Frame: Up to Day 112 ] [ Designated as safety issue: No ]
Percentage of patients achieving PR or CR with stable eGFR (estimated glomerular filtration rate) [ Time Frame: Up to Day 112 ] [ Designated as safety issue: No ]
Pharmacokinetics as measured by Cmax, tmax, AUC 0-last, C trough, t 1/2z [ Time Frame: Up to Day 252 ] [ Designated as safety issue: No ]

Estimated Enrollment:	88
Study Start Date:	August 2012
Estimated Study Completion Date:	December 2014
Estimated Primary Completion Date:	July 2014 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: fresolimumab 1 mg/kg total body weight	Drug: fresolimumab 1 mg/kg total body weight administered intravenous (IV)
Experimental: fresolimumab 4 mg/kg total body weight	Drug: fresolimumab 4 mg/kg total body weight administered intravenous (IV)
Placebo Comparator: Placebo	Drug: Placebo Placebo administered to match active treatment group

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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 and/or ARB 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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01665391

Contacts

Contact: Medical Information	800-745-4447	medinfo@genzyme.com
Contact: Medical Information	617-252-7832	medinfo@genzyme.com

Locations

United States, Alabama
The University of Alabama at Birmingham	Recruiting
Birmingham, Alabama, United States
Principal Investigator: Denyse Thornley-Brown, MD
United States, California
University of California, San Fransico	Recruiting
San Francisco, California, United States
Principal Investigator: Flavio Vincenti, MD
United States, Georgia
Emory University	Recruiting
Atlanta, Georgia, United States
Principal Investigator: Antonio Guasch, MD
United States, Maryland
Johns Hopkins School of Medicine	Recruiting
Baltimore, Maryland, United States
Principal Investigator: Mohamed Atta, MD, MPH
United States, Minnesota
Mayo Clinic	Recruiting
Rochester, Minnesota, United States
Principal Investigator: Fernando Fervenza, MD, PhD
United States, New York
Columbia University Medical Center	Recruiting
New York, New York, United States
Principal Investigator: Jai Radhakrishnan, MD, MS, MRCP, FASN
Clinical Research Development Associates	Recruiting
Rosedale, New York, United States
Principal Investigator: David Scott, MD
United States, North Carolina
Duke University Medical Center	Recruiting
Durham, North Carolina, United States
Principal Investigator: Michelle Winn, MD
United States, Ohio
MetroHealth Systems	Recruiting
Cleveland, Ohio, United States
Principal Investigator: John Sedor, MD
United States, Pennsylvania
University of Pennsylvania	Recruiting
Philadelphia, Pennsylvania, United States
Principal Investigator: Lawrence Holzman, MD
United States, Tennessee
Southeast Renal Research Institute	Recruiting
Chattanooga, Tennessee, United States
Principal Investigator: James Tumlin, MD
Vanderbuilt University Medical Center	Recruiting
Nashville, Tennessee, United States
Principal Investigator: Dwyer Jamie, MD
United States, Washington
University of Washington	Recruiting
Seattle, Washington, United States
Principal Investigator: Peter Nelson, MD

Sponsors and Collaborators

Genzyme

Investigators

Study Director:

Medical Monitor

Genzyme

More Information

No publications provided

Responsible Party:	Genzyme
ClinicalTrials.gov Identifier:	NCT01665391 History of Changes
Other Study ID Numbers:	GC1008FSGS03110, 2010-019545-25, U1111-1139-9082
Study First Received:	August 13, 2012
Last Updated:	March 27, 2013
Health Authority:	United States: Food and Drug Administration

Additional relevant MeSH terms:

Glomerulosclerosis, Focal Segmental
Glomerulonephritis
Nephritis
Kidney Diseases
Urologic Diseases

Antibodies, Monoclonal
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 19, 2013