New hope for kids with rare kidney diseases: drug trial aims to cut protein leak

NCT ID NCT05003986

Disease control Sponsor: Travere Therapeutics, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests an oral drug called sparsentan in 67 children with certain kidney diseases that cause protein to leak into urine. The goal is to see if the drug safely reduces protein levels over 108 weeks. Participants must have a certain level of kidney function to join.

Why investors are watching

Travere Therapeutics is testing sparsentan in children with proteinuric glomerular diseases, a group of kidney conditions marked by protein in the urine. This phase 2 study tracks safety and whether the drug reduces proteinuria over 108 weeks. For a small company, this readout matters because it could expand the drug's use beyond adults and support a pediatric label.

If it works: A positive result could show sparsentan works in children, opening a new patient group for the company. That could lead to regulatory submissions and broader commercial use of the drug.

If it fails: The trial could fail to show meaningful proteinuria reduction or raise safety concerns in children. Pediatric trials often struggle with enrollment and dosing, so delays or negative data would hurt the company's plans for this drug.

AI-written from the trial record. Speculative, and not investment advice.

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Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 67 people

The number the study aims to enrol. It can still change while the study runs.

Started

Aug 2021

Expected to finish

Apr 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

1 year to 17 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria for All Subjects (All Three Populations): A subject must meet all of the following criteria to be eligible for participation in this study: * 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. Inclusion Criteria for Population 1: * 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. Note: The kidney biopsy may have been performed at any time in the past but must include light microscopy and electron microscopy characteristics and/or immunofluorescence findings consistent with FSGS or MCD. Inclusion Criteria for Population 2: * 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 \[ie, simultaneous mutations in 2 of the COL4A3, COL4A4, and COL4A5 genes\]) Inclusion Criteria for Population 3: * 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 Criteria for All Subjects (All Three Populations): A subject who meets any of the following will be excluded from this study: * 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. Note: Before menarche, pregnancy testing and contraceptive use are not required. However, subjects and their parents/legal guardians must be advised that, immediately upon menarche, subjects will be required to begin pregnancy testing and initiate contraceptive use. This requirement cannot be waived. * 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.

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Conditions

The condition(s) this trial relates to.

Alport syndrome focal segmental glomerulosclerosis hereditary nephritis IgA glomerulonephritis immunoglobulin A vasculitis lipoid nephrosis macular corneal dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Alport syndrome Focal segmental glomerulosclerosis IgA vasculitis Immunoglobulin A nephropathy Minimal change disease

How to take part

Only the study team decides who joins. These are the ways to reach them.

The study's own enquiry address

This study publishes an address for enquiries. See it below .
The places running it

38 sites in 8 countries. The list below names each one and where it is.
The official record

ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

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A doctor treating you

A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Show contact details

Study contacts

Contact

Email: •••••@•••••

Locations

Alder Hey Children's NHS Foundation Trust
RECRUITING

Liverpool, L12 2AP, United Kingdom
Atrium Health Levine Children's Hospital
COMPLETED

Charlotte, North Carolina, 28203, United States
Azienda Ospedale Università di Padova
RECRUITING

Padova, 35128, Italy
C.S. Mott Children's Hospital
RECRUITING

Ann Arbor, Michigan, 48109-5008, United States
Cedars-Sinai Medical Center
RECRUITING

Los Angeles, California, 90048, United States
Children's Hospital of Philadelphia
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
Children's Mercy Hospitals and Clinics
RECRUITING

Kansas City, Missouri, 64108, United States
Cohen Children's Medical Center
RECRUITING

New Hyde Park, New York, 11042, United States
Drottning Silvias Barn- och Ungdomssjukhus
RECRUITING

Gothenburg, 416 85, Sweden
Duke Molecular Physiology Institute
RECRUITING

Durham, North Carolina, 22710, United States
Emma Kinderziekenhuis
RECRUITING

Amsterdam, 1105 AZ, Netherlands
Fink Children's Ambulatory Care Center
WITHDRAWN

New York, New York, 10016, United States
Floating Hospital for Children at Tufts Medical Center
WITHDRAWN

Boston, Massachusetts, 02111, United States
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
RECRUITING

Milan, 20122, Italy
Great Ormond Street Hospital for Children NHS Foundation Trust
RECRUITING

London, WC1N3JH, United Kingdom
Hackensack University Medical Center
RECRUITING

Hackensack, New Jersey, 07601, United States
Hospital Universitari Vall d'Hebrón
RECRUITING

Barcelona, 08035, Spain
Hospital Universitario 12 de Octubre
RECRUITING

Madrid, 28041, Spain
Hospital Universitario La Paz
RECRUITING

Madrid, 28046, Spain
Hospital Universitario Virgen del Rocío
RECRUITING

Seville, 41013, Spain
IRCCS Istituto Giannina Gaslini
RECRUITING

Genova, 16147, Italy
Instytut Centrum Zdrowia Matki Polki
WITHDRAWN

Lodz, 93-338, Poland
Instytut Pomnik - Centrum Zdrowia Dziecka
WITHDRAWN

Warsaw, 04-730, Poland
Jersey Shore University Medical Center
RECRUITING

Neptune City, New Jersey, 07753, United States
Karolinska Universitetssjukhuset Huddinge
RECRUITING

Stockholm, 141 86, Sweden
Manchester University NHS Foundation Trust
RECRUITING

Manchester, M13 9WL, United Kingdom
NHS Greater Glasgow and Clyde, Royal Hospital for Children
RECRUITING

Glasgow, G51 4TF, United Kingdom
Nationwide Children's Hospital
RECRUITING

Columbus, Ohio, 43205, United States
Nemours Children's Hospital
RECRUITING

Wilmington, Delaware, 19803, United States
Nicklaus Children's Hospital
RECRUITING

Miami, Florida, 33155, United States
Ospedale Pediatrico Bambino Gesù
RECRUITING

Roma, 00165, Italy
Policlinico Bari Ospedale Pediatrico Giovanni XXIII
RECRUITING

Bari, 70126, Italy
Radboud Universitair Medisch Centrum
WITHDRAWN

Nijmegen, 6525 GA, Netherlands
Seattle Children's Hospital
RECRUITING

Seattle, Washington, 98105, United States
St. Christopher's Hospital for Children
WITHDRAWN

Philadelphia, Pennsylvania, 19134, United States
Texas Children's Hospital
RECRUITING

Houston, Texas, 77030, United States
UT Health - John P. and Kathrine G. McGovern Medical School
WITHDRAWN

Houston, Texas, 77030, United States
Uniklinik Köln, Klinik und Poliklinik für Kinder- und Jugendmedizin
RECRUITING

Cologne, 50937, Germany
University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital for Children
RECRUITING

Bristol, BS2 8BJ, United Kingdom
University of Iowa, Stead Family Children's Hospital
WITHDRAWN

Iowa City, Iowa, 52242, United States
University of Miami, Leonard M. Miller School of Medicine
RECRUITING

Miami, Florida, 33136, United States
University of Minnesota, Masonic Children's Hospital
RECRUITING

Minneapolis, Minnesota, 55454, United States
University of North Carolina at Chapel Hill
RECRUITING

Chapel Hill, North Carolina, 27599, United States
University of Oklahoma Health Sciences Center (OUHSC)
RECRUITING

Oklahoma City, Oklahoma, 73104, United States
Universitätsklinikum Hamburg-Eppendorf
RECRUITING

Hamburg, 20246, Germany
Universitätsklinikum Heidelberg - Angelika Lautenschläger - Kinderklinik
RECRUITING

Heidelberg, 69120, Germany
Uniwersytecki Szpital Dziecięcy w Krakowie
RECRUITING

Krakow, 30-663, Poland

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