Safety, efficacy & pharmacokinetics of Sparsentan in pediatric subjects with selected kidney disease
Research type
Research Study
Full 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 (EPPIK)
IRAS ID
1003784
Contact name
Lynley K. Thinnes
Contact email
Sponsor organisation
Travere Therapeutics, Inc.
Eudract number
2021-000621-27
Clinicaltrials.gov Identifier
Research summary
Travere Therapeutics, Inc. (the Sponsor) is running a Phase 2, open-label research study to see if a test drug, named sparsentan, will help in the treatment of selected protieneuric kidney diseases and if it is safe to use in paediatric participants. Participants will have one of the selected proteinuric glomerular diseases listed below:
• Focal segmental glomerulosclerosis (FSGS)
• Minimal change disease (MCD)
• Immunoglobulin A nephropathy (IgAN)
• Immunoglobulin A vasculitis (IgAV)
• Alport syndrome (AS)Currently, there are no products approved for the prevention or treatment of any of the conditions listed above in paediatrics.
The study is planned to involve approximately 57 children and young people who have proteinuric glomerular diseases. Participants will be divided into 2 populations based on the type of kidney disease they have. The study includes three phases: a screening phase (to check eligibility for the study), a “washout” phase (where certain medications the participant may be currently taking will be stopped), and a treatment phase of up to 108 weeks where participants will receive the study drug. It will involve up to 15 visits to the study site. Overall, the trial is expected to run for approximately 2 years and 3 months.
Study assessments include: blood tests, urinalysis, electrocardiogram, questionnaires, physical examination, and peripheral oedema assessment. Participants may also have a few study visits conducted at their home through a home care vendor contracted by the Sponsor.
REC name
North East - Newcastle & North Tyneside 2 Research Ethics Committee
REC reference
21/NE/0191
Date of REC Opinion
11 Jan 2022
REC opinion
Favourable Opinion