Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases

  • STATUS
    Recruiting
  • End date
    Jun 1, 2025
  • participants needed
    57
  • sponsor
    Travere Therapeutics, Inc.
Updated on 6 October 2021
corticosteroids
glomerular filtration rate
total protein
proteinuria
mammogram
kidney biopsy

Summary

To evaluate the safety, efficacy and tolerability of sparsentan oral suspension and assess changes in proteinuria after once-daily dosing over the 108-week treatment period.

Description

This is a multicenter, open-label, 108-week study of sparsentan in approximately 57 pediatric subjects aged 1 year to <18 years with selected proteinuric glomerular diseases, divided into 2 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 biopsy-confirmed immunoglobulin A nephropathy (IgAN), immunoglobulin A vasculitis (IgAV), or subjects with Alport syndrome (AS)

The study will evaluate long-term safety, tolerability, and efficacy with pharmacokinetic evaluations at Day 1 (Baseline), Day 2 (Visit 3), and Week 12 (Visit 9). For each population, subjects will be enrolled in 3 cohorts based on age ranges.

Details
Condition Focal glomerulosclerosis, IgA nephropathy, Glomerulonephritis, Autoimmune disease, Collagen disease, Alport's Syndrome, Lipoid nephrosis, Nephritis, Focal Segmental Glomerulosclerosis, minimal change disease, Immunoglobulin A-Associated Vasculitis, Immunoglobulin A-Associated Vasculitis, Immunoglobulin A-Associated Vasculitis, Immunoglobulin A-Associated Vasculitis, Immunoglobulin A-Associated Vasculitis, Immunoglobulin A-Associated Vasculitis, Immunoglobulin A-Associated Vasculitis
Treatment sparsentan
Clinical Study IdentifierNCT05003986
SponsorTravere Therapeutics, Inc.
Last Modified on6 October 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

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, 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 age, 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
The subject has a UP/C 1.5 g/g at screening AND one of the following
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
Biopsy-proven FSGS histological pattern with medical history and clinical presentation consistent with maladaptive cause of the lesion
Note: The 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 to <18 years of age at screening
The subject has UP/C 1.0 g/g at screening AND one of the following diagnoses
Biopsy-confirmed IgAN or IgAV
AS (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])

Exclusion Criteria

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% or a hemoglobin value <9 g/dL
The subject has a screening potassium value >5.5 milliequivalent (mEq)/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 investigational product
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 investigational product until 90 days after the last dose of investigational product. 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 in place for at least 3 months. One additional barrier method must also be used during 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 90 days after the last dose of investigational product. 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 investigational product 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
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