Proteinuric glomerular diseases including: •Focal segmental glomerulosclerosis (FSGS) •Minimal change disease (MCD) •Immunoglobulin A nephropathy (IgAN) •Immunoglobulin A vasculitis (IgAV) •Alport syndrome (AS) MedDRA version: 21.1 Level: PT Classification code 10067757 Term: Focal segmental glomerulosclerosis System Organ Class: 10038359 - Renal and urinary disorders MedDRA version: 20.0 Level: PT Classification code 10021263 Term: IgA nephropathy System Organ Class: 10038359 - Renal and
Conditions
Sponsors
Travere Therapeutics, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: For All Subjects (Both Populations) A subject must meet all of the following criteria to be eligible for participation in this study: 1. The subject or parent/legal guardian (as appropriate) is willing and able to provide signed informed consent, and where required, the subject is willing to provide assent before any screening procedures per local requirements. 2. The subject has an eGFR =30 mL/min/1.73 m2 at screening. 3. The subject has a mean seated blood pressure between the 5th and 95th percentile for age, sex, and height. For Population 1 1. The subject is male or female =1 year at screening to =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: For All Subjects (Both Populations) A subject who meets any of the following criteria will be excluded from this study: 1. The subject weighs 2 times the upper limit of the normal range at screening. 12. The subject has a history of malignancy within the past 2 years. 13. The subject has a screening hematocrit 5.5 mEq/L. 15. The subject has any abnormal clinical laboratory screening values that are considered by the Investigator to be clinically significant. 16. The subject has a history of allergic response to any Ang II antagonist or ERA, including sparsentan, or has a hypersensitivity to any of the excipients in the investigational product. 17. Female subjects of childbearing potential, beginning at menarche, who do not agree to use 1 highly reliable (ie, can achieve a failure rate of <1% per year) method of contraception from 7 days before the first dose of the investigational product until 90 days after the last dose of investigational product. Examples of highly reliable contraception methods include stable oral, implanted, transdermal, or injected contraceptive hormones associated with the inhibition of ovulation or an intrauterine device (IUD) in place for at least 3 months. One additional barrier method must also be used during sexual activity, such as a diaphragm, diaphragm with spermicide (preferred), or male partner’s use of male condom or male condom with spermicide (preferred), from Day 1/Randomization until 90 days after the last dose of investigational product. Female subjects of childbearing potential are defined as those who are fertile after menarche, unless permanently sterile; permanent sterilization methods include hysterectomy, bilateral salpingectomy, and bilateral oophorect
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objects of the study are as follows: • Evaluate the safety and tolerability of sparsentan oral suspension • Assess changes in proteinuria after once-daily dosing of sparsentan oral suspension over the 108-week treatment period;Secondary Objective: The secondary objectives are as follows: • Assess the PK of sparsentan oral suspension in a pediatric population • Assess changes in estimated eGFR after once-daily dosing of sparsentan oral suspension over the 108-week treatment period;Primary end point(s): 1. Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), adverse events (AEs) leading to treatment discontinuation, and adverse events of interest (AEOIs) 2. Change from baseline in urine protein/creatinine ratio (UP/C) over the 108-week treatment period;Timepoint(s) of evaluation of this end point: 1. During the whole study period 2. Baseline to all study visits to 108 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Observed plasma PK concentrations at scheduled timepoints and visits 2. Steady-state PK parameters (area under the plasma concentration-time curve during a dosing interval [AUCt], maximum steady-state plasma drug concentration during a dosage interval [Cmax_ss], and minimum steady-state plasma drug concentration [Cmin_ss]) derived from population PK analysis 3. Change from baseline in urine albumin/creatinine ratio (UA/C) over the 108-week treatment period 4. Change from baseline in eGFR over the 108-week treatment period 5. The proportion of subjects with focal segmental glomerulosclerosis (FSGS) and/or minimal change disease (MCD) histological patterns achieving partial remission, defined as UP/C =1.5 g/g and >40% reduction in UP/C over the 108-week treatment period;Timepoint(s) of evaluation of this end point: 1. Day 1, day 2, week 12 2. Day 1, day 2, week 12 3. Baseline to all study visits to 108 weeks 4. Baseline to all study visits to 108 weeks 5. Baseline to all study visits to 108 weeks | — |
Countries
France, Germany, Italy, Netherlands, Poland, Spain, Sweden, United Kingdom, United States
Contacts
Public ContactTravere Therapeutics Call Center
Travere Therapeutics, Inc.
Outcome results
None listed