A single-arm, multicenter, phase III study to assess efficacy, pharmacokinetics, safety and tolerability of atrasentan in pediatric patients of 2 to <18 years of age with primary immunoglobulin A nephropathy (IgAN)
IgA nephropathy is an illness where the body's immune system accidentally damages tiny filters in the kidneys. This damage causes protein to spill into the urine and can lead to long-term kidney problems. This study is testing an investigational medicine called atrasentan in children and young people aged 2 to 17. The main goal is to see if atrasentan can lower protein levels in the urine over 36 weeks. Because this is a 'single-arm' study, everyone who takes part will receive the study medicine—no one receives a dummy pill (placebo). Doctors will also study how the body handles the medicine and check for any side effects. By taking part, young people help doctors discover whether atrasentan could become a helpful new treatment option for children living with this kidney condition.
At a glance
What is this study about?
IgA nephropathy (sometimes called Berger's disease) is a condition that affects the kidneys. The immune system makes proteins called antibodies to fight off germs. In IgA nephropathy, one of these antibodies builds up inside the kidneys. Over time, this causes irritation and swelling in the tiny filters that clean your blood. When these filters are inflamed, they begin leaking protein into your urine, which can lead to kidney damage over time.
Atrasentan is a new medicine designed to block certain signals in the kidney that cause blood vessels to tighten and become inflamed. By blocking these signals, doctors hope atrasentan can ease the strain on the kidneys and significantly reduce the amount of protein leaking into the urine.
While new treatments are often tested in adults first, it is essential to know if they are safe and effective for younger patients. This study aims to find out the best dose of atrasentan for children and teenagers, how their bodies process it, and whether it helps protect their kidneys over time.
Key takeaways
- Testing a new medicine called atrasentan for children and teenagers with IgA nephropathy.
- Open to young people aged 2 to 17 years old with protein in their urine.
- All participants receive the study medicine; there is no dummy pill (placebo).
- Main goal is to reduce kidney inflammation and urine protein leakage over 36 weeks.
- Participation includes regular clinic visits, blood tests, and morning urine collections.
Who may be eligible?
This study is designed for children and young people aged between 2 and 17 years old who have been diagnosed with primary IgA nephropathy. Participants must have persistent protein in their urine, as this shows that their kidneys need extra protection and treatment.
Young people may not be able to join if their kidney condition is caused by another illness (such as lupus), if their kidney function is already very low, or if they have other serious health problems. The study doctors will also check any current medicines to make sure they can be safely combined with atrasentan.
Before taking part, the study team will carry out standard health checks, including blood and urine tests, to make sure the trial is safe and suitable for your child.
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.
- The patient is aged between 2 and 17 years old.
- The patient has a confirmed diagnosis of primary IgA nephropathy.
- There is still protein detected in the patient's urine.
- The family is willing to attend regular specialist hospital visits.
- The patient is able to take daily medication (tablet or liquid).
What does participation involve?
Taking part in this study involves taking the study medicine, atrasentan, by mouth once a day. Because this is an open, single-arm study, every child enrolled receives the active medicine.
Families will need to attend regular clinic visits at a specialist hospital. The key part of the study lasts for 36 weeks (around 9 months), with regular check-ups during and after this period. At clinic visits, the medical team will measure your child's height, weight, and blood pressure, and take blood samples to check how the body is handling the medicine.
You will also be asked to collect first-morning urine samples at home before certain visits. These samples allow doctors to accurately measure the levels of protein in the urine over time. The study team will provide all the necessary containers and clear instructions.
Potential risks and benefits
Locations (2)
- —UnverifiedFrance
- —UnverifiedSpain
Common questions
Will my child definitely receive the study medicine?
Yes. This is a single-arm study, meaning all participants receive atrasentan. There is no placebo (dummy pill).
How is the medicine taken?
Atrasentan is taken once daily by mouth, either as a tablet or in a liquid formulation suitable for younger children.
How long will the trial last?
The main part of the study looks at results over 36 weeks (around 9 months), followed by safety check-ups.
Do we have to pay for the treatment?
No. The study medicine and all trial-related tests are provided free of charge, and travel expenses are often reimbursed.
Can we leave the study if we change our minds?
Yes. You and your child can withdraw from the study at any point without giving a reason, and regular NHS care will continue as normal.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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