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Looking for participantsPhase2

Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases

Sponsor: Travere Therapeutics, Inc.

NCT ID: NCT05003986

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
Sparsentan (drug), Sparsentan (drug), Sparsentan (drug)
How long the study runs
Study runs about 68 months (dates as stated)
About the drug or intervention
Sparsentan — drug: Population 1: 800 mg Sparsentan (oral suspension) · Sparsentan — drug: Population 2: 400 mg Sparsentan (oral suspension) · Sparsentan — drug: Population 3: 400 mg Sparsentan (tablets)
Patient visit burden
Not specified by the sponsor

In plain English

This study looks at whether a medicine called sparsentan can help children and teenagers with kidney conditions that cause protein to leak into their urine (proteinuria). It covers several kidney diseases: focal segmental glomerulosclerosis (FSGS), minimal change disease (MCD), immunoglobulin A nephropathy (IgAN), IgA vasculitis and Alport syndrome. The study is funded by Travere Therapeutics, Inc.

Who can take part

  • Children under 18, with different minimum ages for each group: at least 1 year for FSGS or MCD, at least 2 years for IgAN, IgA vasculitis or Alport syndrome, and at least 8 years (and weighing at least 40 kg) for one IgAN group
  • Kidneys still working reasonably well, based on a blood test called eGFR (estimated glomerular filtration rate) of 30 or above
  • Blood pressure within a normal range for the child's sex and height
  • A certain level of protein in the urine, which differs by group: highest for FSGS/MCD, lower for IgAN/IgA vasculitis/Alport syndrome, and in between for the older IgAN group
  • For most groups, the diagnosis confirmed by a kidney biopsy (a small sample of kidney tissue) or, in some cases, by genetic testing
  • For the older IgAN group: already taking certain blood-pressure-type medicines (ACE inhibitors or ARBs) for at least 12 weeks before screening

Who may not be able to

  • Weighing less than 7.3 kg
  • Kidney disease caused by infections, medicines or cancer, or IgA deposits caused by another condition
  • A recent flare-up or new treatment (such as steroids or other immunosuppressive medicines) within 6 months before screening, or immunosuppressive medicines not on a steady dose for at least 1 month
  • Certain heart problems, including heart failure or significant heart valve disease
  • Liver problems such as jaundice or hepatitis, or certain high liver blood test results
  • Cancer within the past 2 years
  • Certain blood test results that are too low (haemoglobin or haematocrit) or too high (potassium)
  • Allergy to sparsentan or similar medicines
  • Pregnancy, breastfeeding, or (for girls who can become pregnant) not agreeing to use reliable contraception during the study
  • An organ transplant (except corneal transplants), or having taken sparsentan before
  • Taking part in another medicine study within 28 days before screening, or planning to during this study
  • For the older IgAN group: being unable to swallow tablets whole

What taking part involves

  • • Taking the study medicine sparsentan — how it is taken and the dose is not stated — ask the trial team
  • • For the older IgAN group, continuing existing ACE inhibitor (ACEI) or ARB treatment

Time commitment: Not stated — ask the trial team about how long the study lasts, how many visits are needed, and what tests or procedures are involved.

Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.

Type of study
Testing a treatment
Ages
1 Year to 17 Years
Who
All
Number of participants
67
Started
2021-08-12
Last checked
2026-05

Plain English Summary

What is this study?

  • • Testing a new treatment for focal segmental glomerulosclerosis
  • • Phase2 - 67 participants
  • • 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

Who can take part?

  • • Ages 1 Year to 17 Years
  • • Diagnosed with focal segmental glomerulosclerosis

Where?

  • • Bristol - University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital for Children
  • • Glasgow - NHS Greater Glasgow and Clyde, Royal Hospital for Children
  • • Liverpool - Alder Hey Children's NHS Foundation Trust
  • • London - Great Ormond Street Hospital for Children NHS Foundation Trust
  • • +1 more UK sites

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

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.

More detail

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

Focal Segmental GlomerulosclerosisMinimal Change DiseaseImmunoglobulin A NephropathyIgA VasculitisAlport Syndrome

How this trial compares with your answers

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What we know so far

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Still need:

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Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 1 Year - 17 Years
  • Who can join: All genders

Biomarkers mentioned

eGFRhave a negativeand a negativewith positive

Treatment history

Treatments you must have had:

  • ✓ kidney biopsy
  • ✓ any of the prohibited concomitant medications as defined in the study protocol
  • ✓ to begin pregnancy testing and initiate contraceptive use

What the study is looking for

  • ✓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 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:
See the full criteria
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.

Where Is This Study? (5 UK sites)

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

Bristol BS2 8BJ, United Kingdom

Recruiting
Hospital R&D contact (matched)

Diana Benton

research@uhbw.nhs.uk0117 342 0233

NHS Greater Glasgow and Clyde, Royal Hospital for Children

Glasgow G51 4TF, United Kingdom

Recruiting
Hospital R&D contact (matched)

Radek Penar

radoslaw.penar@nhs.scotn/a

Alder Hey Children's NHS Foundation Trust

Liverpool L12 2AP, United Kingdom

Recruiting
Hospital R&D contact (matched)

Kelly Davies

research@alderhey.nhs.uk0151 2525570

Great Ormond Street Hospital for Children NHS Foundation Trust

London WC1N3JH, United Kingdom

Recruiting
Hospital R&D contact (matched)

Main Email: Research.Governance@gosh.nhs.uk

Research.Governance@gosh.nhs.uk0207 905 2700

Manchester University NHS Foundation Trust

Manchester M13 9WL, United Kingdom

Recruiting
Hospital R&D contact (matched)

Elizabeth Mainwaring

R&D.applications@mft.nhs.uk0161 276 3340

How to Get in Touch

Travere Call Center

Sponsor contact

CONTACT

1-877-659-5518 medinfo@travere.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-05