Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases
- Conditions
- IgA VasculitisFocal Segmental GlomerulosclerosisMinimal Change DiseaseImmunoglobulin A NephropathyAlport Syndrome
- Interventions
- Registration Number
- NCT05003986
- Lead Sponsor
- Travere Therapeutics, Inc.
- Brief Summary
To evaluate the safety, efficacy and tolerability of sparsentan oral suspension and tablets, and assess changes in proteinuria after once-daily dosing over 108 weeks.
- Detailed Description
This is a multicenter, open-label, 112-week study of sparsentan in approximately 67 pediatric subjects aged ≥1 year to \<18 years with selected proteinuric glomerular diseases, divided into 3 populations, defined as follows:
* Population 1: Subjects with selected proteinuric glomerular diseases associated with Focal Segmental Glomerulosclerosis (FSGS) and Minimal Change Disease (MCD) histological patterns
* Population 2: Subjects with kidney biopsy-confirmed immunoglobulin A nephropathy (IgAN), immunoglobulin A vasculitis (IgAV), or Alport syndrome (AS)
* Population 3: Subjects with kidney biopsy-confirmed IgAN
The study will evaluate long-term safety, tolerability, and efficacy with pharmacokinetic (PK) evaluations at Day 1 (Baseline), Day 2 (Visit 4), and Week 12 (Visit 9) in Population 1 and Population 2. In Population 3, PK values will be evaluated at Day 1 (Baseline) and at Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, and 96. For Population 1 and Population 2, subjects will be enrolled in 3 cohorts based on age ranges. For Population 3, subjects will be enrolled in one cohort.
Study Enrollment:
* Population 1: FSGS and/or MCD (30 subjects total)
1. Cohort 1 (6 subjects): ≥8 years to \<18 years
2. Cohort 2 (18 subjects): ≥3 years to \<8 years
3. Cohort 3 (6 subjects): ≥1 year to \<3 years
* Population 2: IgAN, IgAV, or AS (27 subjects total)
1. Cohort 1 (9 subjects): ≥8 years to \<18 years
2. Cohort 2 (12 subjects): ≥5 years to \<8 years
3. Cohort 3 (6 subjects): ≥2 years to \<5 years
* Population 3: IgAN (10 subjects total) 1. 10 subjects: ≥8 years to \<18 years
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 67
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Population 1: FSGS and/or MCD Sparsentan Subjects with selected proteinuric glomerular diseases associated with FSGS and MCD histological patterns Population 3: IgAN Sparsentan Subjects with kidney biopsy-confirmed IgAN Population 2: IgAN, IgAV, or AS Sparsentan Subjects with kidney biopsy-confirmed immunoglobulin A nephropathy (IgAN), immunoglobulin A vasculitis (IgAV), or Alport syndrome (AS)
- Primary Outcome Measures
Name Time Method Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), AEs leading to treatment discontinuation, and adverse events of interest (AEOIs) After the last patient has undergone the week 108 visit (Visit 15). The incidence of TEAEs, SAEs, AEs leading to treatment discontinuation, and AEOIs
Urine protein/creatinine ratio (UP/C) at week 108 After the last patient has undergone the Week 108 visit (Visit 15) Change from baseline in UP/C over 108 weeks
- Secondary Outcome Measures
Name Time Method Urine albumin/creatinine ratio (UA/C) over 108 weeks Week 108 Change from baseline in UA/C over 108 weeks
Estimated glomerular filtration rate (eGFR) over 108 weeks Week 108 Change from baseline in eGFR over 108 weeks
Steady-state PK parameters area under the plasma concentration-time curve during a dosing interval ([AUCτ]) Week 108 Steady-state PK parameters \[AUCτ\]
Steady-state PK parameters [Cmax_ss] Week 108 Maximum steady-state plasma drug concentration \[Cmax_ss\]
Steady-state PK parameters [Cmin_ss] Week 108 Minimum steady-state plasma drug concentration \[Cmin_ss\]
Observed plasma Pharmacokinetic (PK) concentrations At scheduled Day 1, Day 2 and Week 12 visits for Population 1 and 2. At scheduled Day 1 and Week 4, 8, 12, 24, 36, 48, 60, 72, 84, and 96 visits for Population 3. Observed plasma PK concentrations at scheduled timepoints and visits
Proportion of subjects achieving complete remission of proteinuria Week 108 The proportion of subjects achieving complete remission of proteinuria, defined as UP/C \<0.3 g/g over 108 weeks
Proportion of subjects with FSGS and/or MCD histological patterns achieving partial remission Week 108 The proportion of subjects with FSGS and/or MCD histological patterns achieving partial remission defined as UP/C ≤1.5 g/g and \>40% reduction in UP/C over 108 weeks
Proportion of subjects who discontinue study medication due to inability to tolerate the oral suspension Week 108 The proportion of subjects who discontinue study medication due to inability to tolerate the smell, taste, aftertaste, volume of administration, or method of administration of oral suspension in Population 1 and Population 2
Trial Locations
- Locations (1)
Travere Investigational Site
🇬🇧Manchester, United Kingdom