Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases
This study is for children and teenagers aged 1 to 17 who have specific kidney diseases. These conditions include Focal Segmental Glomerulosclerosis (FSGS), Minimal Change Disease (MCD), IgA Nephropathy (IgAN), IgA Vasculitis (IgAV), and Alport Syndrome. The main goal is to test a new medicine called sparsentan. Researchers want to find out if sparsentan is safe for children, how well it works to reduce protein in their urine, and if their bodies handle it well. They will study this over a long period, about two years, using either a liquid or tablet form of the medicine. This research is important for finding better ways to help young people manage these kidney conditions.
At a glance
What is this study about?
This study is exploring a new medication called sparsentan for children and teenagers with certain kidney conditions. These conditions are sometimes grouped under the term "proteinuric glomerular diseases," which just means problems with the tiny filters in the kidneys (glomeruli) that lead to too much protein leaking into the urine. Examples of these conditions include Focal Segmental Glomerulosclerosis (FSGS), Minimal Change Disease (MCD), IgA Nephropathy (IgAN), IgA Vasculitis (IgAV), and Alport Syndrome. Over time, these conditions can seriously affect kidney health, so finding effective treatments is very important.
The main aim of this research is to see if sparsentan can safely and effectively reduce the amount of protein in the urine of young patients. Reducing protein in the urine is often a key goal in managing these kidney diseases, as it can help protect the kidneys from further damage. The study will also look at how children's bodies react to sparsentan and how well they tolerate the medicine over a long period, about two years. It's a phase 2 study, which means it's still in the earlier stages of human testing, focusing on safety and whether the medicine shows promise.
Researchers will observe different groups of children and teenagers with these conditions. Some groups will have FSGS or MCD, others will have IgAN, IgAV, or Alport Syndrome, and one group will specifically have IgAN. By studying these different groups, the researchers hope to gather detailed information on how sparsentan works for each specific condition and across different age ranges, from young children to teenagers. This long-term study aims to provide crucial information that could lead to new treatment options for these challenging kidney diseases.
Key takeaways
- This study is testing a new medicine called sparsentan for children and teenagers with specific kidney diseases.
- It aims to see if sparsentan is safe and can reduce protein in the urine over approximately two years.
- The study includes young people aged 1 to 17 years old.
- Participation involves taking medication daily and attending regular clinic visits for monitoring.
- All studies have potential benefits and risks, which will be fully explained before joining.
- You can always withdraw from the study at any time.
Who may be eligible?
This study is for children and teenagers aged 1 to 17 years old. To join, they need to have a parent or guardian who understands and agrees to the study. If the child is old enough, they’ll also need to agree to take part themselves.
Children need to have a certain level of kidney function, measured by a test called eGFR, which should be 30 or higher. Their blood pressure also needs to be within a healthy range for their age and height. Crucially, they must have one of the specific kidney conditions being studied, such as FSGS, MCD, IgAN, IgAV, or Alport Syndrome. For FSGS or MCD, they will need to show a significant amount of protein in their urine. For IgAN, IgAV, or Alport Syndrome, they also need to show protein in their urine, though the amount required is slightly lower. For some of these conditions, a past kidney biopsy (a small tissue sample) or genetic test might be needed to confirm the diagnosis.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Is your child between 1 and 17 years old?
- Do you (as parent/guardian) understand and agree to the study, and does your child (if old enough) also agree?
- Does your child have one of these kidney conditions: FSGS, MCD, IgAN, IgAV, or Alport Syndrome?
- Does your child have a certain amount of protein in their urine?
- Does your child have healthy enough kidney function (eGFR 30 or higher)?
- Is your child's blood pressure within a healthy range for their age and height?
What does participation involve?
If your child takes part in this study, they will receive sparsentan either as a liquid or a tablet once a day for about two years (108 weeks). The study itself will last a little longer, around 112 weeks, which is about 2 years and 3 months. During this time, your child will have several visits to the clinic.
These visits will involve regular check-ups to see how your child is doing, how the medicine is affecting them, and how much protein is in their urine. They will also have blood tests at certain times to check how their body is handling the medicine (this is called PK evaluation). The number and timing of these blood tests will vary slightly depending on their specific kidney condition group. The study aims to monitor their health closely while they are receiving the treatment.
Potential risks and benefits
Locations (47)
- Cedars-Sinai Medical CenterVerified postcodeLos Angeles, United States· Recruiting
- Nemours Children's HospitalVerified postcodeWilmington, United States· Recruiting
- University of Miami, Leonard M. Miller School of MedicineVerified postcodeMiami, United States· Recruiting
- Nicklaus Children's HospitalVerified postcodeMiami, United States· Recruiting
- University of Iowa, Stead Family Children's HospitalVerified postcodeIowa City, United States· Withdrawn
- Floating Hospital for Children at Tufts Medical CenterVerified postcodeBoston, United States· Withdrawn
- C.S. Mott Children's HospitalVerified postcodeAnn Arbor, United States· Recruiting
- University of Minnesota, Masonic Children's HospitalVerified postcodeMinneapolis, United States· Recruiting
- Children's Mercy Hospitals and ClinicsVerified postcodeKansas City, United States· Recruiting
- Hackensack University Medical CenterVerified postcodeHackensack, United States· Recruiting
- Jersey Shore University Medical CenterVerified postcodeNeptune City, United States· Recruiting
- Cohen Children's Medical CenterVerified postcodeNew Hyde Park, United States· Recruiting
Common questions
What is sparsentan?
Sparsentan is the study medicine being tested. It's designed to help with certain kidney conditions that cause too much protein to leak into the urine.
What are the kidney conditions included in this study?
The study is for children with Focal Segmental Glomerulosclerosis (FSGS), Minimal Change Disease (MCD), IgA Nephropathy (IgAN), IgA Vasculitis (IgAV), and Alport Syndrome.
How long will my child be in the study?
Your child will receive the study medicine for about two years (108 weeks), and the entire study period will last roughly 2 years and 3 months (112 weeks).
Why do they need to take blood samples?
Blood samples help the doctors understand how your child's body is absorbing and using the sparsentan medicine, and to monitor their overall health.
Can my child stop participating if they want to?
Yes, absolutely. You can withdraw your child from the study at any time, for any reason, and it won't affect their regular medical care.
How to find out more
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Always speak to your GP or specialist before deciding to take part in a study.
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