Primary Focal Segmental GlomerulosclerosisPhase 2FSGSNCT01665391
A Study of Fresolimumab in Patients With Steroid-Resistant Primary Focal Segmental Glomerulosclerosis (FSGS)
A Phase 2 study testing fresolimumab in people with Primary Focal Segmental Glomerulosclerosis, run by Genzyme, a Sanofi Company at 32 sites in 5 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Focal Segmental Glomerulosclerosis
- What is being tested?
- fresolimumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 36 participants
- Where
- 32 sitesin 5 countries
- Study dates
- Aug 2012to Nov 2014
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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.
Trial locations and contacts
32 sites in 5 countries.
32 sites in 5 countries
United States21 sites
Investigational Site Number 4012
Ann Arbor, Michigan, 48109
Investigational Site Number 4005
Atlanta, Georgia, 30322
Investigational Site Number 4004
Baltimore, Maryland, 21287
Investigational Site Number 4002
Bethesda, Maryland, 20892
Investigational Site Number 4008
Birmingham, Alabama, 35205
Investigational Site Number 4022
Boston, Massachusetts, 02115
Investigational Site Number 4001
Chattanooga, Tennessee, 37404
Investigational Site Number 4020
Dallas, Texas, 75390
Investigational Site Number 4025
Detroit, Michigan, 48202
Investigational Site Number 4027
Houston, Texas, 77030
Investigational Site Number 4016
New York, New York, 10029
Investigational Site Number 4003
New York, New York, 10032
Investigational Site Number 4018
Philadelphia, Pennsylvania, 19104
Investigational Site Number 4029
Phoenix, Arizona, 85032
Investigational Site Number 4006
Rochester, Minnesota, 55905
Investigational Site Number 4014
Rosedale, New York, 11422
Investigational Site Number 4009
San Francisco, California, 94143
Investigational Site Number 4013
Seattle, Washington, 98195
Investigational Site Number 4019
Springfield, Illinois, 01107
Investigational Site Number 4024
St Louis, Missouri, 63110
Investigational Site Number 4023
Stanford, California, 94305
Italy4 sites
Investigational Site Number 4401
Bari, 70124
Investigational Site Number 4404
Bergamo, 24127
Investigational Site Number 4403
Montichiari, 25018
Investigational Site Number 4405
Reggio Calabria, 89124
Germany3 sites
Investigational Site Number 4302
Aachen, 52074
Investigational Site Number 4301
Düsseldorf, 40225
Investigational Site Number 4304
Hamburg, 20246
Spain3 sites
Investigational Site Number 4503
Barcelona, 08025
Investigational Site Number 4504
Madrid, 28040
Investigational Site Number 4502
Madrid, 28041
About this study
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
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
Read more
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
- Being tested
- fresolimumab, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
fresolimumabDrug
1 mg/kg total body weight administered intravenous (IV)
fresolimumabDrug
4 mg/kg total body weight administered intravenous (IV)
PlaceboDrug
Placebo administered to match active treatment group
| Study group | Role | What they receive |
|---|---|---|
| fresolimumab 1 mg/kg total body weight | Experimental | Not described Receives: Drug: fresolimumab |
| fresolimumab 4 mg/kg total body weight | Experimental | Not described Receives: Drug: fresolimumab |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of patients achieving partial remission (PR) or complete remission (CR) in urinary protein: creatinine ratio (Up/c ratio)
Measured: Up to Day 112
Number of patients reporting adverse events (AEs), serious adverse events (SAEs), and medical events of interest (MEOIs)
Measured: Up to Day 112
Secondary outcomes (7)
Percentage of patients achieving CR in Up/c ratio
Measured: Up to Day 112
Percentage of patients achieving PR in Up/c ratio
Measured: Up to Day 112
Change from baseline in Up/c ratio and urinary protein excretion rate
Measured: Up to Day 112
Time to first PR or CR
Measured: Up to Day 112
Change from baseline in eGFR (estimated glomerular filtration rate)
Measured: Up to Day 112
Percentage of patients achieving PR or CR with stable eGFR (estimated glomerular filtration rate)
Measured: Up to Day 112
Mean Fresolimumab serum concentration at each sample collection time point
Measured: Up to Day 252
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Aug 2012 (estimated)
- Study startAug 2012
- Primary completionJun 2014
- Study completionNov 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Primary Focal Segmental GlomerulosclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Genzyme, a Sanofi Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01665391
- Sponsor's protocol ID
- GC1008FSGS03110
- Other IDs
- 2010-019545-25, U1111-1139-9082, DRI12792
- Acronym
- FSGS
- Status last verified
- Aug 2015
- First submitted
- 13 Aug 2012
- Last updated
- 26 Aug 2015
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Hong Q, Kim H, Cai GY, Chen XM, He JC, Lee K. Modulation of TGF-beta signaling new approaches toward kidney disease and fibrosis therapy. Int J Biol Sci. 2025 Feb 3;21(4):1649-1665. doi: 10.7150/ijbs.101548. eCollection 2025.
PubMed 39990662Hodson 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.
PubMed 35224732
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01665391
- Acronym / sponsor ID
- FSGS / GC1008FSGS03110
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- Aug 2012
- Primary completion
- Jun 2014 (actual)
- Study completion
- Nov 2014 (actual)
- First posted
- 15 Aug 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| fresolimumab 1 mg/kg total body weight | Experimental | Drug: fresolimumab |
| fresolimumab 4 mg/kg total body weight | Experimental | Drug: fresolimumab |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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)
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| fresolimumab1 mg/kg total body weight administered intravenous (IV) | Drug | fresolimumab 1 mg/kg total body weight |
| fresolimumab4 mg/kg total body weight administered intravenous (IV) | Drug | fresolimumab 4 mg/kg total body weight |
| PlaceboPlacebo administered to match active treatment group | Drug | Placebo |
Primary outcome
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
Number of patients reporting adverse events (AEs), serious adverse events (SAEs), and medical events of interest (MEOIs)
Time frame: Up to Day 112
7 secondary outcomes
Percentage of patients achieving CR in Up/c ratio
Time frame: Up to Day 112
Percentage of patients achieving PR in Up/c ratio
Time frame: Up to Day 112
Change from baseline in Up/c ratio and urinary protein excretion rate
Time frame: Up to Day 112
Time to first PR or CR
Time frame: Up to Day 112
Change from baseline in eGFR (estimated glomerular filtration rate)
Time frame: Up to Day 112
Percentage of patients achieving PR or CR with stable eGFR (estimated glomerular filtration rate)
Time frame: Up to Day 112
Mean Fresolimumab serum concentration at each sample collection time point
Time frame: Up to Day 252
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Aug 2015.
- Derived from this study: PubMed 39990662Hong Q, Kim H, Cai GY, Chen XM, He JC, Lee K. Modulation of TGF-beta signaling new approaches toward kidney disease and fibrosis therapy. Int J Biol Sci. 2025 Feb 3;21(4):1649-1665. doi: 10.7150/ijbs.101548. eCollection 2025.
- Derived from this study: PubMed 35224732Hodson 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Monitor study director
Genzyme, a Sanofi Company
32 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing fresolimumab in people with Primary Focal Segmental Glomerulosclerosis, run by Genzyme, a Sanofi Company at 32 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Aug 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 32 sites in United States, Italy, Germany, Spain, Brazil. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01665391, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Aug 2015; AzTrials last refreshed its copy on 8 Nov 2026.