Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases
NCT ID: NCT05003986
Last Updated: 2025-12-23
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
67 participants
INTERVENTIONAL
2021-08-12
2027-04-12
Brief Summary
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Detailed Description
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* 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
Conditions
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Keywords
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Population 1: FSGS and/or MCD
Subjects with selected proteinuric glomerular diseases associated with FSGS and MCD histological patterns
Sparsentan
Population 1: 800 mg Sparsentan (oral suspension)
Population 2: IgAN, IgAV, or AS
Subjects with kidney biopsy-confirmed immunoglobulin A nephropathy (IgAN), immunoglobulin A vasculitis (IgAV), or Alport syndrome (AS)
Sparsentan
Population 2: 400 mg Sparsentan (oral suspension)
Population 3: IgAN
Subjects with kidney biopsy-confirmed IgAN
Sparsentan
Population 3: 400 mg Sparsentan (tablets)
Interventions
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Sparsentan
Population 1: 800 mg Sparsentan (oral suspension)
Sparsentan
Population 2: 400 mg Sparsentan (oral suspension)
Sparsentan
Population 3: 400 mg Sparsentan (tablets)
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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.
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.
* 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 Criteria
* 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.
1 Year
17 Years
ALL
No
Sponsors
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Travere Therapeutics, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Radko Komers, MD, PhD
Role: STUDY_DIRECTOR
Travere Therapeutics, Inc.
Locations
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Cedars-Sinai Medical Center
Los Angeles, California, United States
Nemours Children's Hospital
Wilmington, Delaware, United States
University of Miami, Leonard M. Miller School of Medicine
Miami, Florida, United States
Nicklaus Children's Hospital
Miami, Florida, United States
University of Iowa, Stead Family Children's Hospital
Iowa City, Iowa, United States
Floating Hospital for Children at Tufts Medical Center
Boston, Massachusetts, United States
C.S. Mott Children's Hospital
Ann Arbor, Michigan, United States
University of Minnesota, Masonic Children's Hospital
Minneapolis, Minnesota, United States
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, United States
Hackensack University Medical Center
Hackensack, New Jersey, United States
Jersey Shore University Medical Center
Neptune City, New Jersey, United States
Cohen Children's Medical Center
New Hyde Park, New York, United States
Fink Children's Ambulatory Care Center
New York, New York, United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States
Atrium Health Levine Children's Hospital
Charlotte, North Carolina, United States
Duke Molecular Physiology Institute
Durham, North Carolina, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
University of Oklahoma Health Sciences Center (OUHSC)
Oklahoma City, Oklahoma, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
St. Christopher's Hospital for Children
Philadelphia, Pennsylvania, United States
Texas Children's Hospital
Houston, Texas, United States
UT Health - John P. and Kathrine G. McGovern Medical School
Houston, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States
Uniklinik Köln, Klinik und Poliklinik für Kinder- und Jugendmedizin
Cologne, , Germany
Universitätsklinikum Hamburg-Eppendorf
Hamburg, , Germany
Universitätsklinikum Heidelberg - Angelika Lautenschläger - Kinderklinik
Heidelberg, , Germany
Policlinico Bari Ospedale Pediatrico Giovanni XXIII
Bari, , Italy
IRCCS Istituto Giannina Gaslini
Genova, , Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, , Italy
Azienda Ospedale Università di Padova
Padua, , Italy
Ospedale Pediatrico Bambino Gesù
Roma, , Italy
Emma Kinderziekenhuis
Amsterdam, , Netherlands
Radboud Universitair Medisch Centrum
Nijmegen, , Netherlands
Uniwersytecki Szpital Dziecięcy w Krakowie
Krakow, , Poland
Instytut Centrum Zdrowia Matki Polki
Lodz, , Poland
Instytut Pomnik - Centrum Zdrowia Dziecka
Warsaw, , Poland
Hospital Universitari Vall d'Hebrón
Barcelona, , Spain
Hospital Universitario 12 de Octubre
Madrid, , Spain
Hospital Universitario La Paz
Madrid, , Spain
Hospital Universitario Virgen del Rocío
Seville, , Spain
Drottning Silvias Barn- och Ungdomssjukhus
Gothenburg, , Sweden
Karolinska Universitetssjukhuset Huddinge
Stockholm, , Sweden
University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital for Children
Bristol, , United Kingdom
NHS Greater Glasgow and Clyde, Royal Hospital for Children
Glasgow, , United Kingdom
Alder Hey Children's NHS Foundation Trust
Liverpool, , United Kingdom
Great Ormond Street Hospital for Children NHS Foundation Trust
London, , United Kingdom
Manchester University NHS Foundation Trust
Manchester, , United Kingdom
Countries
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Central Contacts
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References
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Rheault MN. Treatment Approaches for Alport Syndrome. J Am Soc Nephrol. 2025 Sep 12. doi: 10.1681/ASN.0000000897. Online ahead of print.
Related Links
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Sponsor Website
Study Website
Other Identifiers
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RTRX-RE021-201
Identifier Type: -
Identifier Source: org_study_id