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 (EPPIK)

Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Conditions:
    Alport Syndrome
    Nephrotic Syndrome
  • Age: Between 1 years - 17 years
  • Gender: Male or Female
  • Other Inclusion Criteria:
    For all Subjects:
    1. Subjects or parent/legal guardians are 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
    2. The subject has an eGFR =30 mL/min/1.73 m2 at screening
    3. The subject has a mean seated blood pressure between the 5th and 95th percentile for age, sex, and height
    Population 1: Subjects with FSGS and MCD
    1. Subject is male or female =1 year at screening and <18 years of age at Day 1.
    2. Subject has a UP/C =1.5 g/g at screening AND one of the following:
      • Biopsy-proven focal segmental glomerulosclerosis(FSGS) or minimal change disease (MCD)
      • Documentation of a genetic mutation in a podocyte protein associated with FSGS or MCD
      • Biopsy-proven FSGS histological pattern with medical history and clinical presentation consistent with maladaptive cause of the lesion
    Population 2: Subjects with IgAN, IgAV, or AS
    1. The subject is male or female =2 years to <18 years of age at screening
    2. The subject has UP/C =1.0 g/g at screening AND one of the following diagnoses:
      • Biopsy-confirmed immunoglobulin A nephropathy(IgAN) or immunoglobulin A vasculitis (IgAV)
      • Alport syndrome

You may not be eligible for this study if the following are true:

  • For all Subjects:
    1. Subject weighs <7.3 kg at screening
    2. Subject has FSGS or MCD histological pattern secondary to viral infections, drug toxicities, or malignancies
    3. Subject has immunoglobulin A (IgA) glomerular deposits not in the context of primary IgAN or IgAV
    4. 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 within 6 months before screening
    5. Subjects taking chronic immunosuppressive medications
    6. Requires any of the prohibited concomitant medications
    7. Has undergone any organ transplantation, with the exception of corneal transplants
    8. Documented history of congenital or acquired heart failure
    9. Has hemodynamically significant cardiac valvular disease
    10. Clinically significant congenital vascular disease.
    11. 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
    12. History of malignancy within the past 2 years.
    13. Has any abnormal clinical laboratory screening values that are considered by the Investigator to be clinically significant
    14. Has a history of allergic response to any Ang II antagonist or ERA, including sparsentan, or has a hypersensitivity to any of the excipients in the investigational product
    15. Female subjects of childbearing potential, beginning at menarche, who do not agree to use 1 highly reliable metjhod of contraception
    16. subject has had prior exposure to sparsentan
    17. participated in a study of another investigational product within 28 days before screening
    18. The subject or parent/legal guardian (as appropriate), in the opinion of the Investigator, is 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

If you are registered as a volunteer, please log in to contact the study team/express interest in this study.