Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases

NCT05003986 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: RTRX-RE021-201
• Study Type: interventional
• Target: 67
• Locations: 8 countries
• Sites: 47

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.

Conditions

Focal Segmental Glomerulosclerosis; IgA Vasculitis; Alport Syndrome; Minimal Change Disease; Immunoglobulin A Nephropathy

Keywords

Alport, AS, FSGS, IgAN, IgAV, MCD, pediatrics, peds

Outcome Measures

Primary Outcomes (2)

• Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), AEs leading to treatment discontinuation, and adverse events of interest (AEOIs)

  The incidence of TEAEs, SAEs, AEs leading to treatment discontinuation, and AEOIs

  After the last patient has undergone the week 108 visit (Visit 15).

• Urine protein/creatinine ratio (UP/C) at week 108

  Change from baseline in UP/C over 108 weeks

  After the last patient has undergone the Week 108 visit (Visit 15)

Secondary Outcomes (9)

• 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.

• Steady-state PK parameters area under the plasma concentration-time curve during a dosing interval ([AUCτ])

  Week 108

• Steady-state PK parameters [Cmax_ss]

  Week 108

• Steady-state PK parameters [Cmin_ss]

  Week 108

• Urine albumin/creatinine ratio (UA/C) over 108 weeks

  Week 108

Study Arms (3)

Population 1: FSGS and/or MCD

EXPERIMENTAL

Subjects with selected proteinuric glomerular diseases associated with FSGS and MCD histological patterns

Drug: Sparsentan

Population 2: IgAN, IgAV, or AS

EXPERIMENTAL

Subjects with kidney biopsy-confirmed immunoglobulin A nephropathy (IgAN), immunoglobulin A vasculitis (IgAV), or Alport syndrome (AS)

Drug: Sparsentan

Population 3: IgAN

EXPERIMENTAL

Subjects with kidney biopsy-confirmed IgAN

Drug: Sparsentan

Interventions

Sparsentan DRUG

Population 1: 800 mg Sparsentan (oral suspension)

Also known as: RE-021

Population 1: FSGS and/or MCD

Eligibility Criteria

• Age: 1 Year - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.
• 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).

You may not qualify if:

• 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).

Sponsors & Collaborators

• Travere Therapeutics, Inc.: lead

Study Sites (47)

Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States

RECRUITING

Nemours Children's Hospital

Wilmington, Delaware, 19803, United States

RECRUITING

University of Miami, Leonard M. Miller School of Medicine

Miami, Florida, 33136, United States

RECRUITING

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

RECRUITING

University of Iowa, Stead Family Children's Hospital

Iowa City, Iowa, 52242, United States

WITHDRAWN

Floating Hospital for Children at Tufts Medical Center

Boston, Massachusetts, 02111, United States

WITHDRAWN

C.S. Mott Children's Hospital

Ann Arbor, Michigan, 48109-5008, United States

RECRUITING

University of Minnesota, Masonic Children's Hospital

Minneapolis, Minnesota, 55454, United States

RECRUITING

Children's Mercy Hospitals and Clinics

Kansas City, Missouri, 64108, United States

RECRUITING

Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

RECRUITING

Jersey Shore University Medical Center

Neptune City, New Jersey, 07753, United States

RECRUITING

Cohen Children's Medical Center

New Hyde Park, New York, 11042, United States

RECRUITING

Fink Children's Ambulatory Care Center

New York, New York, 10016, United States

WITHDRAWN

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

RECRUITING

Atrium Health Levine Children's Hospital

Charlotte, North Carolina, 28203, United States

COMPLETED

Duke Molecular Physiology Institute

Durham, North Carolina, 22710, United States

RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

RECRUITING

University of Oklahoma Health Sciences Center (OUHSC)

Oklahoma City, Oklahoma, 73104, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

St. Christopher's Hospital for Children

Philadelphia, Pennsylvania, 19134, United States

WITHDRAWN

Texas Children's Hospital

Houston, Texas, 77030, United States

RECRUITING

UT Health - John P. and Kathrine G. McGovern Medical School

Houston, Texas, 77030, United States

WITHDRAWN

Seattle Children's Hospital

Seattle, Washington, 98105, United States

RECRUITING

Uniklinik Köln, Klinik und Poliklinik für Kinder- und Jugendmedizin

Cologne, 50937, Germany

RECRUITING

Universitätsklinikum Hamburg-Eppendorf

Hamburg, 20246, Germany

RECRUITING

Universitätsklinikum Heidelberg - Angelika Lautenschläger - Kinderklinik

Heidelberg, 69120, Germany

RECRUITING

Policlinico Bari Ospedale Pediatrico Giovanni XXIII

Bari, 70126, Italy

RECRUITING

IRCCS Istituto Giannina Gaslini

Genova, 16147, Italy

RECRUITING

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy

RECRUITING

Azienda Ospedale Università di Padova

Padua, 35128, Italy

RECRUITING

Ospedale Pediatrico Bambino Gesù

Roma, 00165, Italy

RECRUITING

Emma Kinderziekenhuis

Amsterdam, 1105 AZ, Netherlands

RECRUITING

Radboud Universitair Medisch Centrum

Nijmegen, 6525 GA, Netherlands

RECRUITING

Uniwersytecki Szpital Dziecięcy w Krakowie

Krakow, 30-663, Poland

RECRUITING

Instytut Centrum Zdrowia Matki Polki

Lodz, 93-338, Poland

WITHDRAWN

Instytut Pomnik - Centrum Zdrowia Dziecka

Warsaw, 04-730, Poland

WITHDRAWN

Hospital Universitari Vall d'Hebrón

Barcelona, 08035, Spain

RECRUITING

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

RECRUITING

Hospital Universitario La Paz

Madrid, 28046, Spain

RECRUITING

Hospital Universitario Virgen del Rocío

Seville, 41013, Spain

RECRUITING

Drottning Silvias Barn- och Ungdomssjukhus

Gothenburg, 416 85, Sweden

RECRUITING

Karolinska Universitetssjukhuset Huddinge

Stockholm, 141 86, Sweden

RECRUITING

University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital for Children

Bristol, BS2 8BJ, United Kingdom

RECRUITING

NHS Greater Glasgow and Clyde, Royal Hospital for Children

Glasgow, G51 4TF, United Kingdom

RECRUITING

Alder Hey Children's NHS Foundation Trust

Liverpool, L12 2AP, United Kingdom

RECRUITING

Great Ormond Street Hospital for Children NHS Foundation Trust

London, WC1N3JH, United Kingdom

RECRUITING

Manchester University NHS Foundation Trust

Manchester, M13 9WL, United Kingdom

RECRUITING

Related Publications (1)

• Rheault MN. Treatment Approaches for Alport Syndrome. J Am Soc Nephrol. 2026 Jan 1;37(1):172-179. doi: 10.1681/ASN.0000000897. Epub 2025 Sep 12.

  PMID: 40938675 DERIVED

Related Links

• Sponsor Website
• Study Website

MeSH Terms

Conditions

Glomerulosclerosis, Focal Segmental; Nephrosis, Lipoid; Glomerulonephritis, IGA; IgA Vasculitis; Nephritis, Hereditary; Macular dystrophy, corneal type 1

Interventions

sparsentan

Condition Hierarchy (Ancestors)

Glomerulonephritis; Nephritis; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Nephrosis; Autoimmune Diseases; Immune System Diseases; Vasculitis; Vascular Diseases; Cardiovascular Diseases; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemostatic Disorders; Hemorrhagic Disorders; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Immune Complex Diseases; Hypersensitivity; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms; Urogenital Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Collagen Diseases; Connective Tissue Diseases

Study Officials

• Radko Komers, MD, PhD

  Travere Therapeutics, Inc.

  STUDY DIRECTOR

Central Study Contacts

Travere Call Center

CONTACT

1-877-659-5518; medinfo@travere.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 29, 2021
• First Posted: August 13, 2021
• Study Start: August 12, 2021
• Primary Completion (Estimated): March 12, 2027
• Study Completion (Estimated): April 12, 2027
• Last Updated: December 23, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share
• Time Frame: Requests can be made following completion of the study and full publication of the study data in a peer reviewed journal for up to 36 months following its publication.
• Access Criteria: Requires submission and approval of intended use and a data sharing agreement.

Locations

US (23); IT (5); PL (3); ES (4); DE (3); NL (2); GB (5); SE (2)