What are the aims/goals/objectives of EPPIK?
a. The EPPIK Study is a 108-week pediatric study that is evaluating sparsentan in FSGS, MCD, IgAN, IgAV and Alport syndrome. This study is an EU regulatory requirement to support approval submissions for IgAN and FSGS. It is not designed to ultimately support an approval in MCD, IgAV or Alport syndrome.
b. This study aims to evaluate the safety and effectiveness of an investigational therapy, sparsentan, in children with selected rare kidney diseases. In other words, to evaluate whether sparsentan helps in the treatment of kidney diseases and if it is safe to use in children. The kidney diseases selected are those in which Travere Therapeutics thinks sparsentan will be helpful and well-tolerated.
c. In more detail, the EPPIK study aims to further understand if an investigational therapy, sparsentan, can help the kidneys filter protein and slow the decline of kidney function. The amount of protein found in the urine (called proteinuria) is seen as a marker of kidney function. Lowering proteinuria levels is associated with better kidney health outcomes.
About the drug or intervention:
Participants receive sparsentan, the investigational drug, taken as a liquid by mouth.
How many patients will you be enrolled in this study?
Approximately 57 children will participate in the EPPIK Study.
Where are the clinical sites located?
Clinical study sites will be located in the US, UK and various countries in the EU.
Who can participate in the EPPIK Study?
a. Children ages 1 to 17 years with certain rare kidney diseases, including Alport syndrome (ages 2 – 17 at screening).
b. Children who are 18 during screening are not eligible for the EPPIK Study as they are considered adults on Day 1.
c. Additional requirements will need to be met in order for your child to participate. The study doctor will review these criteria during the screening visit to determine eligibility.
What is the eGFR that is eligible for this study?
The child should have an eGFR >30 mL/min/1.73m2 at screening for any of the 5 diseases.
What are the permitted UP/C (urine protein to creatinine ratio – a measure of protein loss in the urine or proteinuria) ranges?
UP/C is dependent on the type of glomerular disease of the patient.
For Alport syndrome patients the UP/C must be >/= 1.0 g/g.
Are children who are on dialysis or have had a transplant eligible for EPPIK?
No, children who are on dialysis or have had a transplant are not eligible for EPPIK.
What is involved for the patient?
Patients will participate for about 2 years and 3 months and will receive sparsentan.
Will telehealth or homecare be available to reduce the amount of office visits?
Depending on the reason for homecare or telehealth, both would be available to the patient. The study center will provide this information.
Is reimbursement for transportation and lodging available?
Yes, reimbursement for your expenses related to the clinical study such as transportation, lodging and meals is available. Please contact the study center staff for more information.