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Focal Segmental GlomerulosclerosisPhase 2EPPIKNCT05003986

Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases

A Phase 2 study testing Sparsentan in people with Focal Segmental Glomerulosclerosis and Minimal Change Disease, run by Travere Therapeutics, Inc. at 47 sites in 8 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 1 year to 17 years
What is being tested?
Sparsentan
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
67 participants
Where
47 sitesin 8 countries
Study dates
12 Aug 2021to 12 Apr 2027
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 1 year to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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.
Show all 39Show fewer
  • 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 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.

Who cannot join

None listed.

Trial locations and contacts

47 sites in 8 countries. Contact the study team or a site near you to ask about taking part.

Travere Call Center contact

1-877-659-5518

medinfo@travere.com

47 sites in 8 countries

United States23 sites, 17 recruiting

  • C.S. Mott Children's Hospital

    Ann Arbor, Michigan, 48109-5008

    Recruiting

  • Floating Hospital for Children at Tufts Medical Center

    Boston, Massachusetts, 02111

    Withdrawn

  • University of North Carolina at Chapel Hill

    Chapel Hill, North Carolina, 27599

    Recruiting

  • Atrium Health Levine Children's Hospital

    Charlotte, North Carolina, 28203

    Completed

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205

    Recruiting

  • Duke Molecular Physiology Institute

    Durham, North Carolina, 22710

    Recruiting

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601

    Recruiting

  • Texas Children's Hospital

    Houston, Texas, 77030

    Recruiting

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

    Houston, Texas, 77030

    Withdrawn

  • University of Iowa, Stead Family Children's Hospital

    Iowa City, Iowa, 52242

    Withdrawn

  • Children's Mercy Hospitals and Clinics

    Kansas City, Missouri, 64108

    Recruiting

  • Cedars-Sinai Medical Center

    Los Angeles, California, 90048

    Recruiting

  • University of Miami, Leonard M. Miller School of Medicine

    Miami, Florida, 33136

    Recruiting

  • Nicklaus Children's Hospital

    Miami, Florida, 33155

    Recruiting

  • University of Minnesota, Masonic Children's Hospital

    Minneapolis, Minnesota, 55454

    Recruiting

  • Jersey Shore University Medical Center

    Neptune City, New Jersey, 07753

    Recruiting

  • Cohen Children's Medical Center

    New Hyde Park, New York, 11042

    Recruiting

  • Fink Children's Ambulatory Care Center

    New York, New York, 10016

    Withdrawn

  • University of Oklahoma Health Sciences Center (OUHSC)

    Oklahoma City, Oklahoma, 73104

    Recruiting

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

    Recruiting

  • St. Christopher's Hospital for Children

    Philadelphia, Pennsylvania, 19134

    Withdrawn

  • Seattle Children's Hospital

    Seattle, Washington, 98105

    Recruiting

  • Nemours Children's Hospital

    Wilmington, Delaware, 19803

    Recruiting

Other trials in United States

Italy5 sites, 5 recruiting

  • Policlinico Bari Ospedale Pediatrico Giovanni XXIII

    Bari, 70126

    Recruiting

  • IRCCS Istituto Giannina Gaslini

    Genova, 16147

    Recruiting

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milan, 20122

    Recruiting

  • Azienda Ospedale Università di Padova

    Padova, 35128

    Recruiting

  • Ospedale Pediatrico Bambino Gesù

    Roma, 00165

    Recruiting

Other trials in Italy

United Kingdom5 sites, 5 recruiting

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

    Bristol, BS2 8BJ

    Recruiting

  • NHS Greater Glasgow and Clyde, Royal Hospital for Children

    Glasgow, G51 4TF

    Recruiting

  • Alder Hey Children's NHS Foundation Trust

    Liverpool, L12 2AP

    Recruiting

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    London, WC1N3JH

    Recruiting

  • Manchester University NHS Foundation Trust

    Manchester, M13 9WL

    Recruiting

Other trials in United Kingdom

Spain4 sites, 4 recruiting

  • Hospital Universitari Vall d'Hebrón

    Barcelona, 08035

    Recruiting

  • Hospital Universitario 12 de Octubre

    Madrid, 28041

    Recruiting

  • Hospital Universitario La Paz

    Madrid, 28046

    Recruiting

  • Hospital Universitario Virgen del Rocío

    Seville, 41013

    Recruiting

Other trials in Spain

Germany3 sites, 3 recruiting

  • Uniklinik Köln, Klinik und Poliklinik für Kinder- und Jugendmedizin

    Cologne, 50937

    Recruiting

  • Universitätsklinikum Hamburg-Eppendorf

    Hamburg, 20246

    Recruiting

  • Universitätsklinikum Heidelberg - Angelika Lautenschläger - Kinderklinik

    Heidelberg, 69120

    Recruiting

Other trials in Germany

Poland3 sites, 1 recruiting

  • Uniwersytecki Szpital Dziecięcy w Krakowie

    Krakow, 30-663

    Recruiting

  • Instytut Centrum Zdrowia Matki Polki

    Lodz, 93-338

    Withdrawn

  • Instytut Pomnik - Centrum Zdrowia Dziecka

    Warsaw, 04-730

    Withdrawn

Other trials in Poland

Netherlands2 sites, 1 recruiting

  • Emma Kinderziekenhuis

    Amsterdam, 1105 AZ

    Recruiting

  • Radboud Universitair Medisch Centrum

    Nijmegen, 6525 GA

    Withdrawn

Other trials in Netherlands

Sweden2 sites, 2 recruiting

  • Drottning Silvias Barn- och Ungdomssjukhus

    Gothenburg, 416 85

    Recruiting

  • Karolinska Universitetssjukhuset Huddinge

    Stockholm, 141 86

    Recruiting

Other trials in Sweden

About this study

Official title: A Phase 2, Open-Label, Single-Arm, Cohort Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Sparsentan Treatment in Pediatric Subjects With Selected Proteinuric Glomerular Diseases

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.

Read the 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
Being tested
Sparsentan
Keywords
Alport, AS, FSGS, IgAN, IgAV, MCD, pediatrics, peds
Medical subject headings
Glomerulosclerosis, Focal Segmental, Nephrosis, Lipoid, Glomerulonephritis, IGA, IgA Vasculitis, Nephritis, Hereditary, Macular dystrophy, corneal type 1

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • SparsentanDrug

    Also called RE-021

    Population 1: 800 mg Sparsentan (oral suspension)

  • SparsentanDrug

    Also called RE-021

    Population 2: 400 mg Sparsentan (oral suspension)

  • SparsentanDrug

    Also called RE-021

    Population 3: 400 mg Sparsentan (tablets)

Study groups in NCT05003986
Study groupRoleWhat they receive
Population 1: FSGS and/or MCDExperimentalSubjects with selected proteinuric glomerular diseases associated with FSGS and MCD histological patterns
Receives: Drug: Sparsentan
Population 2: IgAN, IgAV, or ASExperimentalSubjects with kidney biopsy-confirmed immunoglobulin A nephropathy (IgAN), immunoglobulin A vasculitis (IgAV), or Alport syndrome (AS)
Receives: Drug: Sparsentan
Population 3: IgANExperimentalSubjects with kidney biopsy-confirmed IgAN
Receives: Drug: Sparsentan

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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

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

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

    Change from baseline in UP/C over 108 weeks

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

Secondary outcomes (9)

  1. Observed plasma Pharmacokinetic (PK) concentrations

    Observed plasma PK concentrations at scheduled timepoints and visits

    Measured: 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.

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

    Steady-state PK parameters [AUCτ]

    Measured: Week 108

  3. Steady-state PK parameters [Cmax_ss]

    Maximum steady-state plasma drug concentration [Cmax_ss]

    Measured: Week 108

  4. Steady-state PK parameters [Cmin_ss]

    Minimum steady-state plasma drug concentration [Cmin_ss]

    Measured: Week 108

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

    Change from baseline in UA/C over 108 weeks

    Measured: Week 108

  6. Estimated glomerular filtration rate (eGFR) over 108 weeks

    Change from baseline in eGFR over 108 weeks

    Measured: Week 108

  7. Proportion of subjects achieving complete remission of proteinuria

    The proportion of subjects achieving complete remission of proteinuria, defined as UP/C <0.3 g/g over 108 weeks

    Measured: Week 108

  8. Proportion of subjects with FSGS and/or MCD histological patterns achieving partial remission

    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

    Measured: Week 108

  9. Proportion of subjects who discontinue study medication due to inability to tolerate the oral suspension

    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

    Measured: Week 108

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted13 Aug 2021
  2. Study start12 Aug 2021
  3. Primary completion12 Mar 2027 (estimated)
  4. Study completion12 Apr 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
67 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05003986
Sponsor's protocol ID
RTRX-RE021-201
Acronym
EPPIK
Status last verified
May 2026
First submitted
29 Jul 2021
Last updated
12 May 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Requests for clinical trial data, including language stating its intended use, should be directed to datarequest@travere.com. If approved, the requested information will be provided to the requestor after signing a data access agreement. 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. Travere reserves the right to decline or recommend modifications to a request if it does not comply with the data sharing policy or if it is determined that the request is made by a biased source.

Publications

  • Cambier A, Lachize-Neanne L, Haas M. IgA Nephropathy across the Age Spectrum: Urgent Need to Accelerate Pediatric Access to Therapeutic Innovation. Clin J Am Soc Nephrol. 2026 Jul 1. doi: 10.2215/CJN.0000001172. Online ahead of print. No abstract available.

    PubMed 42384439

  • 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.

    PubMed 40938675

  • Meliambro K, He JC, Campbell KN. Podocyte-targeted therapies - progress and future directions. Nat Rev Nephrol. 2024 Oct;20(10):643-658. doi: 10.1038/s41581-024-00843-z. Epub 2024 May 9.

    PubMed 38724717

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Sparsentan in people with Focal Segmental Glomerulosclerosis and Minimal Change Disease, run by Travere Therapeutics, Inc. at 47 sites in 8 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 39 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 12 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 47 sites in United States, Italy, United Kingdom, Spain, Germany and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Travere Call Center (1-877-659-5518, medinfo@travere.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), AEs leading to treatment discontinuation, and adverse events of interest (AEOIs) (time frame: After the last patient has undergone the week 108 visit (Visit 15).). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05003986, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 May 2026.

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