Randomized, Double-Blind, Safety and Efficacy Study of RE-021 (Sparsentan) in Focal Segmental Glomerulosclerosis

Completed · Phase 2

Conditions studied: Focal Segmental Glomerulosclerosis

In brief

This study will investigate whether RE-021 (Sparsentan), a selective dual-acting receptor antagonist with affinity for endothelin (A type) and angiotensin II receptors (Type 1), is safe and effective in treating patients with focal segmental glomerulosclerosis (FSGS).

Key facts

Study ID: NCT01613118
Run by: Travere Therapeutics, Inc.
People needed: 109
Starts: 2014-03-01
Expected to finish: 2024-03-25
Last updated by the study team: 2025-05-15

Who can join

Age: 8 and older, up to 75. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Biopsy-proven FSGS OR documentation of a genetic mutation in a podocyte protein associated with the disease.
Urine protein/creatinine ratio (Up/C) at or above 1.0 g/g.
Estimated glomerular filtration rate (eGFR) >30.
Mean seated blood pressure (BP) >100/60 mmHg and <145/95 in patients >/= 18 years of age. Mean seated BP for patients <18 years of age should be >90/60 mmHg and <95th percentile for age, gender, and height.
If a patient is taking immunosuppressive medications (except for Rituximab or cyclophosphamide), the dose and/or levels must be stable for 1 month prior to randomization and the Investigator should not have plans to alter the regimen during the first 8 weeks of treatment, except to stabilize levels. Patients on Rituximab or cyclophosphamide will be eligible provided they have not been taking these medications for 3 months prior to randomization.
US Sites: Males or females 8 to 75 years of age willing and able to provide written informed consent and/or assent, with informed consent signed by patient or parent/legal guardian.
EU Sites: Males or females 18 to 75 years of age willing and able to provide written informed consent, with informed consent, signed by patient or legal guardian.

You may not qualify if…

Patients with FSGS secondary to another condition.
Patients with history of type 1 diabetes mellitus, uncontrolled type 2 diabetes mellitus (HBA1c>8%), or non-fasting blood glucose >180 mg/dL at screening.
Patients who have had any organ transplant.
Patients with a requirement for any of the medications indicated on the list of Excluded Medications, with the exception of ACE and ARBs.
Patients with a documented history of heart failure (NYHA Class II-IV), and / or previous hospitalization for heart failure or unexplained dyspnea, orthopnea, paroxysmal nocturnal dyspnea, ascites and peripheral edema. Patients with clinically significant cerebrovascular disease (transient ischemic attack or stroke) and/or coronary artery disease (hospitalization for myocardial infarction or unstable angina, new onset of angina with positive functional tests or coronary angiogram revealing stenosis, coronary revascularization procedure) within 6 months before screening.
Patients with clinically significant cardiac conduction defects, including second or third degree atrioventricular block, left bundle branch block, sick sinus syndrome, atrial fibrillation, atrial flutter, an accessory bypass tract, or any arrhythmia requiring medication.
Patients with jaundice, hepatitis, or known hepatobiliary disease (includes asymptomatic cholelithiasis); alanine aminotransferase and/or aspartate aminotransferase >2 times the upper limit of normal at Screening.
Patients positive for human immunodeficiency virus (HIV), and markers indicating acute (positivity of at least one of the following: Hepatitis B surface antigen [HBsAg], Hepatitis B "e" antigen [HBeAg], Hepatitis B virus [HBV] DNA in blood or liver, Immunoglobulin M Hepatitis B core antibody) or chronic (HBsAg and/or HBeAg and/or Hepatitis B virus [HBV] DNA positivity) HBV infection, or hepatitis C virus (HCV) infection (reactive anti-HCV antibody and/or HCV RNA). Testing at screening is only required for patients >/= 18 years of age.
History of malignancy other than adequately treated basal cell or squamous cell skin cancer within the past 5 years.
Patients with hemodynamically significant valvular disease.
Hematocrit (HCT) <27 or hemoglobin (Hgb) <9.
Potassium >5.5 mEq/L.
Patients >18 years of age with Estimated Glomerular Filtration Rate (eGFR) ≥60 ml mL/min who have N-terminal prohormone of brain natriuretic peptide (NT-proBNP) ≥200 pg/mL (57.8 pmol/L). For patients >18 years of age with eGFR <60 mL/min, the following parameters requiring echocardiography (ECHO) at screening should be used for exclusion:
NT-proBNP ≥300 pg/mL in patients >18 years of age with eGFR 45 59.9 mL/min
NT-proBNP = 200-299 pg/mL in patients >18 years of age with eGFR 45 59.9 mL/min, and abnormal ejection fraction (EF <55) and/or diastolic dysfunction on ECHO
NT-proBNP ≥400 pg/mL in patients >18 years of age with eGFR 30.0 44.9 mL/min
NT-proBNP = 200-399 pg/mL in patients >18 years of age with eGFR 30.0 44.9 mL/min, and abnormal ejection fraction (EF <55) and/or diastolic dysfunction on ECHO.
Patients >/= 18 years of age with body mass index (BMI) >40. Patients <18 years of age with a BMI in the 99% percentile plus 5 units.
Patients who have abnormal clinical laboratory values at Screening, which are designated by the Principal Investigator as clinically significant.
Patients with a history of drug or alcohol abuse within the past two years.
Patients with a history of an allergic response to any angiotensin II antagonist or endothelin receptor antagonist.
Women who are pregnant or breastfeeding.
Women of child-bearing potential (WOCBP) who are unwilling or unable to use two reliable methods of contraception, with at least one being highly reliable (e.g. oral, implanted or injected contraceptive hormones or an intrauterine device) and one being a barrier method, in order to avoid pregnancy for the entire study period and for 90 days post study participation. WOCBP, defined as all women physiologically capable of becoming pregnant, includes any female who has experienced menarche and who has not undergone successful surgical sterilization (hysterectomy, bilateral tubal ligation or bilateral ovariectomy) or is not postmenopausal (defined as amenorrhea >12 consecutive months and for women on hormone replacement therapy, only with documented plasma follicle stimulating hormone level greater than 35 mIU/mL). Women using oral, implanted or injected contraceptive hormones, an intrauterine device, barrier methods (diaphragm, condoms, spermicidal) to prevent pregnancy, practicing abstinence or where the partner is sterile (e.g. vasectomy) As well as postmenopausal women who have fertilized eggs implanted are also considered WOCBP.
Patients who have participated in another investigational drug study within 28 days prior to screening, or who will participate in another drug study during the course of this study.
Prior exposure to Sparsentan, dual acting receptor antagonist (DARA), or PS433540.

Where it is running

Balboa Nephrology Medical Group — San Diego, California, United States
Los Angeles Biomedical Research Institute — Torrance, California, United States
Colorado Kidney Care — Denver, Colorado, United States
University of Miami Miller School of Medicine — Miami, Florida, United States
Miami Children's Hospital — Miami, Florida, United States
University of Iowa Children's Hospital — Iowa City, Iowa, United States
Renal and Transplant Associates of New England, PC — Springfield, Massachusetts, United States
University of Minnesota — Minneapolis, Minnesota, United States
The Children's Mercy Hospital — Kansas City, Missouri, United States
NYU Langone Medical Center — New York, New York, United States
Icahn School of Medicine at Mount Sinai — New York, New York, United States
Columbia University Medical Center — New York, New York, United States
SUNY Stony Brook Hospital — Stony Brook, New York, United States
UNC Kidney Center, Pediatrics — Chapel Hill, North Carolina, United States
University North Carolina (UNC) Kidney Center — Chapel Hill, North Carolina, United States
Akron Nephrology Associates — Akron, Ohio, United States
The Cleveland Clinic Foundation — Cleveland, Ohio, United States
Unversity of Oklahoma Health Sciences Center — Oklahoma City, Oklahoma, United States
Northeast Clinical Research Center — Bethlehem, Pennsylvania, United States
The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
Temple University School of Medicine — Philadelphia, Pennsylvania, United States
University of Pennsylvania, Perelman School of Medicine — Philadelphia, Pennsylvania, United States
Vanderbilt University Medical Center — Nashville, Tennessee, United States
Clinical Advancement Center — San Antonio, Texas, United States
Southern Utah Kidney and Hypertension Center — St. George, Utah, United States

Full record on ClinicalTrials.gov

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