Everything You Need to Know About Travere’s New Clinical Trial for Children, EPPIK

Travere Therapeutics has recently launched their Phase 2 pediatric clinical trial, EPPIK (Evaluating Problematic Proteinuria in Kids) which is aimed at helping children with FSGS, MCD, IgAN and other rare kidney diseases. We know that with a clinical study specifically designed for kids, parents and caregivers may have some questions. We asked Travere to answer some of these questions and have complied an FAQ list for our community around EPPIK.

1. What is the EPPIK study?
   • EPPIK (Evaluating Problematic Proteinuria in Kids) is a Phase 2 study of children 1-17 years old with focal segmental glomerulosclerosis (FSGS), minimal change disease (MCD), IgA nephropathy (IgAN), also known as Berger’s disease, IgA vasculitis (IgAV), also known as Henoch-Schönlein purpura, or Alport syndrome (AS). These rare kidney diseases are associated with progression to end-stage kidney disease and currently have limited or no treatment options. This 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.
2. What is sparsentan?
   • Sparsentan is a novel Dual Endothelin Angiotensin Receptor Antagonist (DEARA) – this means it is a dual acting, highly selective antagonist of both the endothelin A receptor (ETAR) and the angiotensin II subtype 1 receptor (AT1R).
   • In the EPPIK Study, sparsentan is taken once a day orally.
3. What is a Phase 2 study?
   • A Phase 2 study is a clinical trial that helps researchers determine how safe, tolerable and effective an investigational drug may be in specific patient populations, such as those noted above. While the EPPIK study is a Phase 2 study, sparsentan has been studied in another Phase 2 study as well as two Phase 3 studies involving approximately 880 patients (some of whom were children). The purpose of the EPPIK study is to gain further experience on the use of sparsentan in children.
4. How long does this study take?
   • Patients who meet all eligibility criteria and are enrolled in the study may expect to participate in the study for about 2 years and 3 months.
5. Will you be enrolling children with congenital Nephrotic Syndrome or infantile Nephrotic Syndrome?
   • Yes, children ages 1-17 with one of those syndromes who meet the study criteria will be able to participate in the EPPIK Study. The specific disorders we are looking for are noted above in the answer to question 1.
6. How many children and young people are you looking to enroll in this study?
   • We will be looking to enroll approximately 57 children in the EPPIK study.
7. How many study sites are there available? Where are these sites located?
   • Sites will be in the United States, UK, and various countries in the EU (France, Germany, Italy, Spain, Netherlands, Poland, Sweden). The final list of sites has not been fully determined yet.
8. Because this study is for children, how is it different than a study for adults?
   • The main difference is how much blood is drawn from the children to do all the tests necessary to evaluate sparsentan in the study. Patient safety is always our number one priority, and, with children, we won’t sample as much blood as we would from an adult. We narrowed the number of tests and worked with the central lab to be able to get results from a smaller sample size. We also must ensure that we adequately inform both the child who might participate in the study and the child’s parents/guardians about all the requirements the study has as well as any risks that may be associated with participating in the study. We do this by developing different study assents or ways to communicate what the study entails so that participating families understand the purpose of the study and what is involved.
9. Are you asking kids about the taste, smell, etc. of the study drug?
   • Yes, we will be asking the children to answer simple questions on how the drug tastes, smells, feels, etc.
10. How do you determine which participants will be required to have pregnancy testing or take birth control?
    • We realize this is a delicate subject for some children and their parents, but it is very important that any female child who has had her first menstruation period take birth control according to the protocol. This class of drugs is known to potentially cause harm to a developing fetus, and therefore we do not want any female participant who is able to have a child to become pregnant.
11. Is there flexibility where lab samples can be collected for my child? Is there an option for home care visits to collect these samples?
    • Yes, there is some flexibility in where labs are taken. If the child can’t make it to the clinical site to have the labs drawn, which is always the preferred way, we have contracted with a company that will come to the patient’s home or other convenient location, to draw the labs and send them in for testing.
12. Does my child have to stop the medications they’re on? What drugs can they remain on?
    • That depends, if they are enrolled in this study, they will need to stop any drug that functions similarly to the investigational drug. That is, if they are on an ACE inhibitor or ARB, they will need to stop those drugs at least 2 weeks before they begin taking the experimental drug and will not be able to take them while they are participating in the study. They will be able to continue with certain blood pressure medications if not in the same drug class, as well as many other medications they may be on. The Investigator will have a complete list of drugs that are allowed and not allowed.
13. What will happen to the results of this clinical study?
    • After all the patients have been enrolled and have completed the study, the data will be analyzed to see if it was successful in reducing the amount of protein in the urine and if it slowed the decline in kidney function as measured by estimated glomerular filtration rate, or eGFR. How well the drug was tolerated, that is, the safety profile, will also be analyzed. Those results will, in all likelihood, be put into a document known as a manuscript and published in a scientific journal. Data will also be added to the study listing on the clinicaltrials.gov website at the end of the study.
14. If my child is in another trial studying sparsentan, do they have to enroll in this new study? If so, will their study site change? 
    • No, if your child is participating in another trial with sparsentan they would not be eligible to join the EPPIK study. They would stay in the study they are currently in.

For more information about EPPIK, contact medinfo@travere.com