Purpose

To determine the long-term nephroprotective potential of treatment with sparsentan as compared to an angiotensin receptor blocker in patients with primary and genetic focal segmental glomerulosclerosis (FSGS).

Condition

Eligibility

Eligible Ages
Between 8 Years and 75 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion Criteria

for the Double-blind Period: - Sites within the US: The patient is male or female aged 8 to 75 years, inclusive, weighing ≥20 kg at screening - Sites outside the US: The patient is male or female aged 18 to 75 years, inclusive, weighing ≥20 kg at screening - Biopsy-proven focal segmental glomerulosclerosis (FSGS) lesion(s) or documentation of a genetic mutation in a podocyte protein associated with FSGS. - Urine protein/creatinine (UP/C) ≥1.5 g/g (170 mg/mmol) at screening - eGFR ≥30 mL/min/1.73 m2 at screening. - Women of childbearing potential must agree to the use of one highly reliable method of contraception from 7 days prior to the first dose of study medication until 90 days after the last dose of study medication, plus one additional barrier method during sexual activity

Exclusion Criteria

for the Double-blind Period: - FSGS secondary to another condition - Positive serological tests of another primary or secondary glomerular disease not consistent with a diagnosis of primary or genetic FSGS - History of type 1 diabetes mellitus, uncontrolled type 2 diabetes mellitus, or nonfasting blood glucose >180 mg/dL (10.0 mmol/L) - Treated with rituximab, cyclophosphamide, or abatacept within ≤3 months prior to screening; if taking other chronic immunosuppressive medications, the dosage must be stable prior to screening - Documented history of heart failure, coronary artery disease, or cerebrovascular disease - Significant liver disease - Positive at screening for the human immunodeficiency virus or markers indicating acute or chronic hepatitis B virus infection or hepatitis C infection - History of malignancy other than adequately treated basal cell or squamous cell skin cancer or cervical carcinoma within the past 2 years - Screening hematocrit value <27% (0.27 L/L) or hemoglobin value <9 g/dL (90 g/L) - Screening potassium value of >5.5 mEq/L (5.5 mmol/L) - Extreme obesity (ie, ≥18 years of age with a body mass index (BMI) >40, or is <18 years of age with a BMI in the 99th percentile plus 5 units at screening, in whom there is a causal relationship between obesity and the development of FSGS - History of alcohol or illicit drug use disorder - History of serious side effect or allergic response to any angiotensin II antagonist or endothelin receptor antagonist - Female patient is pregnant, plans to become pregnant during the course of the study, or is breastfeeding. Key Inclusion Criteria for the Open-label Extension Based on assessments at the Week 108 visit: - Complete participation in the double-blind period, including the Week 112 visit. - Patient received blinded study medication through the duration of the double-blind period (ie, did not permanently discontinue study medication) Key Exclusion Criteria for the Open-label Extension Based on Assessments at Week 108 and 112 visits: - Progression to end-stage renal disease requiring replacement therapy - The patient developed criteria for discontinuation between Week 108 and Week 112 - The patient was unable to initiate, or developed contraindications to, treatment with RAAS inhibitors between Week 108 and Week 112 - eGFR ≤20 mL/min/1.73 m2 at Week 108

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Double (Participant, Investigator)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
sparsentan for double-blind and open-label extension
Sparsentan will be administered as a single oral morning dose; an initial dose of 400 mg daily titrating up to a target dose of 800 mg, daily
  • Drug: sparsentan
    Double-blind period: target dose of 800 mg daily; Open-label extension: target dose based on dosage from week 114 daily
    Other names:
    • RE-021
Active Comparator
Irbesartan
Irbesartan will be administered as a single oral morning dose; an initial dose of 150 mg daily titrating up to a target dose of 300 mg, daily
  • Drug: Irbesartan
    target dose of 300 mg daily
    Other names:
    • Irbesartan Tablets USP

Recruiting Locations

Retrophin Investigational Site
Florence, Alabama 35630

Retrophin Investigational Site
Homewood, Alabama 35209

Retrophin Investigational Site
Mesa, Arizona 85210

Retrophin Investigational Site
Phoenix, Arizona 85027

Retrophin Investigational Site
Los Angeles, California 90027

Retrophin Investigational Site
Northridge, California 91324

Retrophin Investigational Site
Palo Alto, California 94304

Retrophin Investigational Site
San Diego, California 92103

Retrophin Investigational Site
San Dimas, California 91773

Retrophin Investigational Site
San Francisco, California 94118

Retrophin Investigational Site
Torrance, California 90502

Retrophin Investigational Site
Victorville, California 92395

Retrophin Investigational Site
Aurora, Colorado 80045

Retrophin Investigational Site
Denver, Colorado 80205

Retrophin Investigational Site
Denver, Colorado 80230

Retrophin Investigational Site
Norwich, Connecticut 06360

Retrophin Investigational Site
Wilmington, Delaware 19803

Retrophin Investigational Site
Washington, District of Columbia 20037

Retrophin Investigational Site
Coral Springs, Florida 33071

Retrophin Investigational Site
Fort Lauderdale, Florida 33308

Retrophin Investigational Site
Miami, Florida 33126

Retrophin Investigational Site
Miami, Florida 33136

Retrophin Investigational Site
Miami, Florida 33155

Retrophin Investigational Site
Ocala, Florida 34471

Retrophin Investigational Site
Orlando, Florida 32827

Retrophin Investigational Site
Port Charlotte, Florida 33952

Retrophin Investigational Site
Tampa, Florida 33612

Retrophin Investigational Site
Temple Terrace, Florida 33637

Retrophin Investigational Site
Winter Park, Florida 32789

Retrophin Investigational Site
Atlanta, Georgia 30322

Retrophin Investigational Site
Lawrenceville, Georgia 30046

Meridian ID
Meridian, Idaho 83642

Retrophin Investigational Site
Chicago, Illinois 60611

Retrophin Investigational Site
Hinsdale, Illinois 60521

Retrophin Investigational Site
Merrillville, Indiana 46410

Retrophin Investigational Site
Iowa City, Iowa 52242

Retrophin Investigational Site
Kansas City, Kansas 66160

Retrophin Investigational Site
Wichita, Kansas 67214

Retrophin Investigational Site
Louisville, Kentucky 40202

Retrophin Investigational Site
Monroe, Louisiana 71201

Retrophin Investigational Site
New Orleans, Louisiana 70121

Retrophin Investigational Site
Shreveport, Louisiana 71101

Retrophin Investigational Site
Baltimore, Maryland 21287

Retrophin Investigational Site
Boston, Massachusetts 02111

Retrophin Investigational Site
Boston, Massachusetts 02215

Retrophin Investigational Site
Springfield, Massachusetts 01107

Retrophin Investigational Site
Ann Arbor, Michigan 48109

Retrophin Investigational Site
Minneapolis, Minnesota 55404

Retrophin Investigational Site
Minneapolis, Minnesota 55455

Retrophin Investigational Site
Kansas City, Missouri 64108

Retrophin Investigational Site
Kansas City, Missouri 64111

Retrophin Investigational Site
Saint Louis, Missouri 63110

Retrophin Investigational Site
Las Vegas, Nevada 89129

Retrophin Investigational Site
Reno, Nevada 89511

Retrophin Investigational Site
Hackensack, New Jersey 07601

Retrophin Investigational Site
Bronx, New York 10467

Retrophin Investigational Site
Flushing, New York 11355

Retrophin Investigational Site
New Hyde Park, New York 11040

Retrophin Investigational Site
New York, New York 10016

Retrophin Investigational Site
New York, New York 10029

Retrophin Investigational Site
New York, New York 10032

Retrophin Investigational Site
Stony Brook, New York 11794

Retrophin Investigational Site
Chapel Hill, North Carolina 27514

Retrophin Investigational Site
Charlotte, North Carolina 28204

Retrophin Investigational Site
Greenville, North Carolina 27834

Retrophin Investigational Site
Raleigh, North Carolina 27609

Retrophin Investigational Site
Winston-Salem, North Carolina 27103

Retrophin Investigational Site
Akron, Ohio 44302

Retrophin Investigational Site
Cleveland, Ohio 44195

Retrophin Investigational Site
Columbus, Ohio 43210

Retrophin Investigational Site
Oklahoma City, Oklahoma 73104

Retrophin Investigational Site
Bend, Oregon 97701

Retrophin Investigational Site
Roseburg, Oregon 97471

Retrophin Investigational Site
Bethlehem, Pennsylvania 18017

Retrophin Investigational Site
Camp Hill, Pennsylvania 17011

Retrophin Investigational Site
Philadelphia, Pennsylvania 19104

Retrophin Investigational Site
Upland, Pennsylvania 19013

Retrophin Investigational Site
Columbia, South Carolina 29203

Retrophin Investigational Site
Memphis, Tennessee 38103

Retrophin Investigational Site
Amarillo, Texas 79106

Retrophin Investigational Site
Corpus Christi, Texas 78412

Retrophin Investigational Site
Dallas, Texas 75231

Retrophin Investigational Site
Dallas, Texas 75235

Retrophin Investigational Site
Dallas, Texas 75246

Retrophin Investigational Site
El Paso, Texas 79935

Retrophin Investigational Site
Fort Worth, Texas 76104

Retrophin Investigational Site
Houston, Texas 77030

Retrophin Investigational Site
Houston, Texas 77054

Retrophin Investigational Site
Lewisville, Texas 75057

Retrophin Investigational Site
Sherman, Texas 75090

Retrophin Investigational Site
Webster, Texas 77598

Retrophin Investigational Site
Saint George, Utah 84790

Retrophin Investigational Site
Salt Lake City, Utah 84112

Retrophin Investigational Site
Salt Lake City, Utah 84115

Retrophin Investigational Site
Alexandria, Virginia 22304

Retrophin Investigational Site
Hampton, Virginia 23666

Retrophin Investigational SIte
Lansdowne Town Center, Virginia 20176

Retrophin Investigational Site
Spokane, Washington 99204

Retrophin Investigational Site
Tacoma, Washington 98405

Retrophin Investigational Site
Morgantown, West Virginia 26506

Retrophin Investigational Site
Marshfield, Wisconsin 54449

Retrophin Investigational Site
Milwaukee, Wisconsin 53226

Retrophin Investigational Site
Wauwatosa, Wisconsin 53226

More Details

NCT ID
NCT03493685
Status
Recruiting
Sponsor
Retrophin, Inc.

Study Contact

Retrophin Call Center
1-877-659-5518
medinfo@retrophin.com

Detailed Description

This is a randomized, multicenter, double-blind, parallel, active-control study. Approximately 300 patients aged 8 to 75 years (inclusive) will be enrolled in the study. The study will be conducted in approximately 300 study centers, globally. The investigational drug (sparsentan) is a dual-acting angiotensin receptor blocker and endothelin receptor antagonist. The active control is irbesartan. Patients who meet eligibility criteria will require washout from renin-angiotensin-aldosterone system (RAAS) blockers, if applicable prior to their first dose of study drug. Patients will be randomly assigned in a 1:1 ratio to receive either sparsentan or active control (irbesartan). After completing the double-blind portion of the study, patients may participate in the open-label extension for treatment with sparsentan if they meet eligibility criteria.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.