Effectiveness and Safety of Sparsentan as treatment for Primary Focal Segmental Glomerulosclerosis (FSGS)

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 300

Inclusion Criteria

1. Biopsy-proven FSGS or documentation of a genetic mutation in a podocyte protein associated with FSGS.
2. Male or female aged 18 to 75 years, inclusive weighing at least 20 kg at screening (Note: patients under 18 may be recruited only in the
United States).
3. UP/C =1.5 g/g at screening.
4. eGFR =30 mL/min/1.73 m2 at screening.
5. Mean seated blood pressure =100/60 mmHg and =160/100 mmHg.
6. WOCBP agree to the use of contraception and pregnancy testing as described in the protocol.
Are the trial subjects under 18? yes
Number of subjects for this age range: 60
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 180
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 60

Exclusion Criteria

1. FSGS secondary to another condition.
2. Positive findings on serological tests of another primary or secondary glomerular disease.
3. History of type 1 diabetes mellitus, uncontrolled type 2 diabetes mellitus (hemoglobin A1c [HbA1c] >8%), or nonfasting blood glucose >180 mg/dL at screening.
4. Any organ transplantation, with the exception of corneal transplants.
5. Treatment with any of the prohibited concomitant medications.
6. Treatment with rituximab, cyclophosphamide, or abatacept within =3 months prior to screening. If a patient is taking other chronic immunosuppressive medications, the dosage must be stable for =1 month prior to randomization.
7. Documented history of heart failure and/or previous hospitalization for heart failure or unexplained dyspnea, orthopnea, paroxysmal nocturnal dyspnea, ascites, and/or peripheral edema.
8. Clinically significant cerebrovascular disease and/or coronary artery disease.
9. Hemodynamically significant valvular disease.
10. Jaundice, hepatitis, or known hepatobiliary disease (excluding asymptomatic cholelithiasis), or transaminase levels >2 times the upper limit of the normal range at screening.
11. Positive at screening for the human immunodeficiency virus (HIV) or markers indicating acute or chronic hepatitis B (HBV) infection or hepatitis C virus (HCV) infection.
12. History of malignancy other than adequately treated basal cell or squamous cell skin cancer or cervical carcinoma within the past 2 years.
13. A screening hematocrit value <27% or hemoglobin value <9 g/dL.
14. A screening potassium value of >5.5 mEq/L.
15. Body mass index (BMI) >40 and there is a causal relationship to the FSGS lesion.
16. History of alcohol or illicit drug use, or excessive alcohol intake (>21 units per week within 2 years of screening)
17. History of serious side effect or allergic response to any angiotensin II antagonist or endothelin receptor antagonist or hypersensitivity to
any of the excipients
18. Female patient is pregnant, breastfeeding, or planning to conceive during the study.
19. Participation in a study of another investigational product within 28 days prior to screening.
20. Prior exposure to sparsentan.
21. Unable to adhere to the requirements of the study, including swallowing the study medication capsules whole.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The efficacy objective of the study is to determine the long-term nephroprotective potential of treatment with sparsentan as compared to an angiotensin receptor blocker in patients with primary and genetic FSGS.<br><br>The safety objective of the study is to assess the safety and tolerability of sparsentan by double-blind monitoring of safety endpoints.;Secondary Objective: Not applicable;Primary end point(s): Efficacy End points:<br>The primary efficacy endpoint is the slope of eGFR.<br><br>The surrogate efficacy endpoint is the proportion of patients achieving a target reduction in proteinuria<br><br>Safety End points:<br>• Descriptive statistics will be used to summarize the safety data.;Timepoint(s) of evaluation of this end point: Week 36 and Week 108<br>

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Secondary efficacy endpoints include:<br>-The percent change in eGFR.<br>-The percent change in eGFR from baseline to 4 weeks post-treatment.<br><br>;Timepoint(s) of evaluation of this end point: Weeks 108 and 112

Updated 10/10/2018

Effectiveness and Safety of Sparsentan as treatment for Primary Focal Segmental Glomerulosclerosis (FSGS)

Recruitment & Eligibility

Study & Design

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