Study of Sparsentan for Pediatric Glomerular Diseases
This study is testing an oral medication called sparsentan in children and teenagers aged 1 to 17 years old who have certain kidney diseases that cause protein in their urine (proteinuric glomerular diseases). These conditions include Focal Segmental Glomerulosclerosis (FSGS), Minimal Change Disease (MCD), Immunoglobulin A Nephropathy (IgAN), IgA Vasculitis, and Alport Syndrome. The main goals are to see how safe sparsentan is, how well it's tolerated, and if it reduces the amount of protein in the urine over 108 weeks. The study is currently recruiting about 67 participants.
- Study design
- This is an open-label study, meaning you and your doctors will know which treatment you are receiving. It will include about 67 children and teenagers divided into three groups based on their specific kidney disease.
- What's involved
- You would take sparsentan once daily for 108 weeks. There will be multiple visits for evaluations, including blood tests for drug levels (pharmacokinetic evaluations) at different time points.
- Compensation
- Not stated in the trial record.
- Follow-up
- The study will evaluate safety and urine protein levels after the last patient completes their Week 108 visit.
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NCT05003986
Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases
Recruiting
PHASE2Ages 1–17InterventionalTreatmentTravere Therapeutics, Inc.
~67 participants
Updated 2026-05-12 on ClinicalTrials.gov
What's tested:Sparsentan
At a glance
Recruiting sites
38 of 47 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), AEs leading to treatment discontinuation, and adverse events of interest (AEOIs)
Measured over After the last patient has undergone the week 108 visit (Visit 15).
+1 more outcome measured
Conditions
Where it's being run
47 sites across 23 statesItaly5
United Kingdom5
Spain4
North Carolina3
Germany3
Poland3
Florida2
New Jersey2
Study leadership
- Radko Komers, MD, PhD · STUDY_DIRECTOR · Travere Therapeutics, Inc.
Who to contact
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Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
The subject or parent/legal guardian (as appropriate) is willing and able to provide signed informed consent/assent, and where required, the subject is willing to provide assent before any screening procedures per local requirements.
The subject has an estimated glomerular filtration rate (eGFR) ≥30 mL/min/1.73 m2 at screening.
The subject has a mean seated blood pressure between the 5th and 95th percentile for sex and height.
The subject is male or female ≥1 year at screening and \<18 years of age at Day 1 (Baseline).
The subject has a UP/C ≥1.5 g/g (170 mg/mmol) at screening AND one of the following:
Kidney biopsy-proven FSGS or MCD histological patterns and clinical presentation consistent with primary FSGS or MCD and qualifying proteinuria at screening despite history or ongoing treatment with corticosteroids and/or other immunosuppressive disease-modifying agents.
Documentation of a genetic mutation in a podocyte protein associated with FSGS or MCD. Subjects with a documented podocytic mutation do not require kidney biopsy.
Kidney biopsy-proven FSGS histological pattern with medical history and clinical presentation consistent with maladaptive cause of the lesion.
The subject is male or female ≥2 years at screening and \<18 years of age at Day 1 (Baseline).
The subject has UP/C ≥0.6 g/g (68 mg/mmol) at screening AND one of the following diagnoses:
Kidney biopsy-confirmed IgAN, IgAV, or AS
Diagnosis of AS by genetic testing (pathogenic X-linked Collagen, Type IV, Alpha-5 (COL4A5) mutation OR autosomal-recessive mutations in both alleles of Collagen, Type IV, Alpha-3 (COL4A3) and/or Collagen, Type IV, Alpha-4 (COL4A4) OR autosomal-dominant COL4A3 and/or COL4A4 and digenic mutations \[ie, simultaneous mutations in 2 of the COL4A3, COL4A4, and COL4A5 genes\])
The subject is male or female ≥8 years at screening and \<18 years of age at Day 1 (Baseline).
The subject has UP/C ≥1.0 g/g (113 mg/mmol) at screening AND has kidney biopsy-confirmed IgAN
Subject weighs ≥40 kg
The subject has been on ACEI and/or ARB therapy for at least 12 weeks prior to screening
Exclusion
The subject weighs \<7.3 kg at screening.
The subject has FSGS or MCD histological pattern secondary to viral infections, drug toxicities, or malignancies.
The subject has immunoglobulin A (IgA) glomerular deposits not in the context of primary IgAN or IgAV (ie, secondary to another condition; eg, systemic lupus erythematosus and liver cirrhosis).
The subject has had an acute onset or presentation of glomerular disease or a diagnostic biopsy or a relapse of glomerular disease requiring new or different class of immunosuppressive treatment (including, but not limited to, systemic corticosteroids, calcineurin inhibitors and mycophenolate mofetil, abatacept, cyclophosphamide, rituximab, ofatumumab, and ocrelizumab) within 6 months before screening.
Subjects taking chronic immunosuppressive medications (including systemic steroids) not on a stable dose for ≥1 month before screening.
The subject requires any of the prohibited concomitant medications as defined in the study protocol.
The subject has undergone any organ transplantation, with the exception of corneal transplants.
The subject has a documented history of congenital or acquired heart failure (modified Ross heart failure classification for children Class II to Class IV) and/or previous hospitalization for heart failure or unexplained dyspnea, orthopnea, paroxysmal nocturnal dyspnea, ascites, and/or peripheral edema.
The subject has hemodynamically significant cardiac valvular disease.
The subject has clinically significant congenital vascular disease.
The subject has jaundice, hepatitis, or known hepatobiliary disease, or alanine aminotransferase and/or aspartate aminotransferase \>2 times the upper limit of the normal range at screening.
The subject has a history of malignancy within the past 2 years.
The subject has a screening hematocrit \<27% (0.27 L/L) or a hemoglobin value \<9 g/dL (90 g/L).
The subject has a screening potassium value \>5.5 milliequivalent (mEq)/L (5.5 mmol/L).
The subject has any abnormal clinical laboratory screening values that are considered by the Investigator to be clinically significant.
The subject has a history of allergic response to any angiotensin II antagonist or endothelin receptor antagonist, including sparsentan, or has a hypersensitivity to any of the excipients in the study medication.
The female subject is pregnant, plans to become pregnant during the course of the study, or is breastfeeding.
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 study medication until 28 days after the last dose of study medication. 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. One additional barrier method must also be used during vaginal 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 28 days after the last dose of study medication. 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 oophorectomy. All female subjects of childbearing potential must have a negative serum pregnancy test result at screening (Visit 1) and a negative urine pregnancy test result, with positive results confirmed by serum, at every study visit from Day 1 (Visit 3) and after.
The subject has participated in a study of another study medication within 28 days before screening or plans to participate in such a study during the course of this study.
The subject has had prior exposure to sparsentan.
The subject or parent/legal guardian (as appropriate), in the opinion of the Investigator, are unable to adhere to the requirements of the study including but not limited to, a history of noncompliance and/or any other reason that causes the Investigator to believe the subject would not be a good candidate for the study.
For Population 3 - the subject is unable to swallow the study medication tablets whole.
What this trial measures
- 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 108After the last patient has undergone the Week 108 visit (Visit 15)
Change from baseline in UP/C over 108 weeks