A Study of Fresolimumab in Patients With Steroid-Resistant Primary Focal Segmental Glomerulosclerosis (FSGS) (FSGS)
Primary Purpose
Primary Focal Segmental Glomerulosclerosis
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
fresolimumab
fresolimumab
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Primary Focal Segmental Glomerulosclerosis
Eligibility 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 (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.
Sites / Locations
- Investigational Site Number 4008
- Investigational Site Number 4029
- Investigational Site Number 4009
- Investigational Site Number 4023
- Investigational Site Number 4005
- Investigational Site Number 4019
- Investigational Site Number 4004
- Investigational Site Number 4002
- Investigational Site Number 4022
- Investigational Site Number 4012
- Investigational Site Number 4025
- Investigational Site Number 4006
- Investigational Site Number 4024
- Investigational Site Number 4016
- Investigational Site Number 4003
- Investigational Site Number 4014
- Investigational Site Number 4018
- Investigational Site Number 4001
- Investigational Site Number 4020
- Investigational Site Number 4027
- Investigational Site Number 4013
- Investigational Site Number 4104
- Investigational Site Number 4302
- Investigational Site Number 4301
- Investigational Site Number 4304
- Investigational Site Number 4401
- Investigational Site Number 4404
- Investigational Site Number 4403
- Investigational Site Number 4405
- Investigational Site Number 4503
- Investigational Site Number 4504
- Investigational Site Number 4502
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Placebo Comparator
Arm Label
fresolimumab 1 mg/kg total body weight
fresolimumab 4 mg/kg total body weight
Placebo
Arm Description
Outcomes
Primary Outcome Measures
Percentage of patients achieving partial remission (PR) or complete remission (CR) in urinary protein: creatinine ratio (Up/c ratio)
Number of patients reporting adverse events (AEs), serious adverse events (SAEs), and medical events of interest (MEOIs)
Secondary Outcome Measures
Percentage of patients achieving CR in Up/c ratio
Percentage of patients achieving PR in Up/c ratio
Change from baseline in Up/c ratio and urinary protein excretion rate
Time to first PR or CR
Change from baseline in eGFR (estimated glomerular filtration rate)
Percentage of patients achieving PR or CR with stable eGFR (estimated glomerular filtration rate)
Mean Fresolimumab serum concentration at each sample collection time point
Full Information
NCT ID
NCT01665391
First Posted
August 13, 2012
Last Updated
August 12, 2015
Sponsor
Genzyme, a Sanofi Company
1. Study Identification
Unique Protocol Identification Number
NCT01665391
Brief Title
A Study of Fresolimumab in Patients With Steroid-Resistant Primary Focal Segmental Glomerulosclerosis (FSGS)
Acronym
FSGS
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
Study Type
Interventional
2. Study Status
Record Verification Date
August 2015
Overall Recruitment Status
Completed
Study Start Date
August 2012 (undefined)
Primary Completion Date
June 2014 (Actual)
Study Completion Date
November 2014 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Genzyme, a Sanofi Company
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Focal Segmental Glomerulosclerosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
36 (Actual)
8. Arms, Groups, and Interventions
Arm Title
fresolimumab 1 mg/kg total body weight
Arm Type
Experimental
Arm Title
fresolimumab 4 mg/kg total body weight
Arm Type
Experimental
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
fresolimumab
Intervention Description
1 mg/kg total body weight administered intravenous (IV)
Intervention Type
Drug
Intervention Name(s)
fresolimumab
Intervention Description
4 mg/kg total body weight administered intravenous (IV)
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo administered to match active treatment group
Primary Outcome Measure Information:
Title
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
Title
Number of patients reporting adverse events (AEs), serious adverse events (SAEs), and medical events of interest (MEOIs)
Time Frame
Up to Day 112
Secondary Outcome Measure Information:
Title
Percentage of patients achieving CR in Up/c ratio
Time Frame
Up to Day 112
Title
Percentage of patients achieving PR in Up/c ratio
Time Frame
Up to Day 112
Title
Change from baseline in Up/c ratio and urinary protein excretion rate
Time Frame
Up to Day 112
Title
Time to first PR or CR
Time Frame
Up to Day 112
Title
Change from baseline in eGFR (estimated glomerular filtration rate)
Time Frame
Up to Day 112
Title
Percentage of patients achieving PR or CR with stable eGFR (estimated glomerular filtration rate)
Time Frame
Up to Day 112
Title
Mean Fresolimumab serum concentration at each sample collection time point
Time Frame
Up to Day 252
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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 (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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Monitor
Organizational Affiliation
Genzyme, a Sanofi Company
Official's Role
Study Director
Facility Information:
Facility Name
Investigational Site Number 4008
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35205
Country
United States
Facility Name
Investigational Site Number 4029
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85032
Country
United States
Facility Name
Investigational Site Number 4009
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
Investigational Site Number 4023
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
Investigational Site Number 4005
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Investigational Site Number 4019
City
Springfield
State/Province
Illinois
ZIP/Postal Code
01107
Country
United States
Facility Name
Investigational Site Number 4004
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Investigational Site Number 4002
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892
Country
United States
Facility Name
Investigational Site Number 4022
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Investigational Site Number 4012
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
Investigational Site Number 4025
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48202
Country
United States
Facility Name
Investigational Site Number 4006
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Investigational Site Number 4024
City
St Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Investigational Site Number 4016
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Investigational Site Number 4003
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Investigational Site Number 4014
City
Rosedale
State/Province
New York
ZIP/Postal Code
11422
Country
United States
Facility Name
Investigational Site Number 4018
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Investigational Site Number 4001
City
Chattanooga
State/Province
Tennessee
ZIP/Postal Code
37404
Country
United States
Facility Name
Investigational Site Number 4020
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
Investigational Site Number 4027
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Investigational Site Number 4013
City
Seattle
State/Province
Washington
ZIP/Postal Code
98195
Country
United States
Facility Name
Investigational Site Number 4104
City
Porto Alegre
ZIP/Postal Code
90035 003
Country
Brazil
Facility Name
Investigational Site Number 4302
City
Aachen
ZIP/Postal Code
52074
Country
Germany
Facility Name
Investigational Site Number 4301
City
Düsseldorf
ZIP/Postal Code
40225
Country
Germany
Facility Name
Investigational Site Number 4304
City
Hamburg
ZIP/Postal Code
20246
Country
Germany
Facility Name
Investigational Site Number 4401
City
Bari
ZIP/Postal Code
70124
Country
Italy
Facility Name
Investigational Site Number 4404
City
Bergamo
ZIP/Postal Code
24127
Country
Italy
Facility Name
Investigational Site Number 4403
City
Montichiari
ZIP/Postal Code
25018
Country
Italy
Facility Name
Investigational Site Number 4405
City
Reggio Calabria
ZIP/Postal Code
89124
Country
Italy
Facility Name
Investigational Site Number 4503
City
Barcelona
ZIP/Postal Code
08025
Country
Spain
Facility Name
Investigational Site Number 4504
City
Madrid
ZIP/Postal Code
28040
Country
Spain
Facility Name
Investigational Site Number 4502
City
Madrid
ZIP/Postal Code
28041
Country
Spain
12. IPD Sharing Statement
Citations:
PubMed Identifier
35224732
Citation
Hodson EM, Sinha A, Cooper TE. Interventions for focal segmental glomerulosclerosis in adults. Cochrane Database Syst Rev. 2022 Feb 28;2(2):CD003233. doi: 10.1002/14651858.CD003233.pub3.
Results Reference
derived
Learn more about this trial
A Study of Fresolimumab in Patients With Steroid-Resistant Primary Focal Segmental Glomerulosclerosis (FSGS)
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