An open-label, single-arm, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients who have completed a clinical trial (parent study) in the iptacopan pediatric program.
This clinical trial is a follow-on study for young people who have finished an earlier trial testing a medicine called iptacopan. The medicine is being developed to treat two different rare conditions: paroxysmal nocturnal haemoglobinuria (PNH), which causes red blood cells to break down too quickly, and IgA nephropathy (IgAN), an immune condition that can damage the kidneys. Because patients have already taken part in an initial study, this trial focuses on long-term safety and tolerability. Doctors want to see how well the medicine continues to work over an extended period. All participants in this study receive iptacopan, meaning there is no dummy pill (placebo). Throughout the trial, doctors will regularly monitor participants through physical check-ups, blood tests, and urine tests to make sure the treatment remains safe and effective as young people grow.
At a glance
What is this study about?
Paroxysmal nocturnal haemoglobinuria (PNH) and IgA nephropathy (IgAN) are two long-term conditions linked to the immune system. In PNH, part of the body's natural defence system mistakenly attacks and breaks down healthy red blood cells, which can cause severe tiredness, anaemia, and blood clots. In IgAN, a protein made by the immune system builds up inside the kidneys, causing inflammation that can leak protein into the urine and slowly damage kidney function over time.
Researchers have created iptacopan, an oral medicine designed to calm the specific part of the immune system causing this damage. Younger patients have already received this medicine in earlier paediatric studies. This extension trial allows those who completed the earlier study to carry on receiving iptacopan so doctors can gather important information about its safety over several years.
Long-term studies are vital, especially for children and teenagers, because their bodies are constantly growing and developing. By following participants closely over time, doctors can see whether the medicine continues to keep blood counts stable or protect kidney function without causing unwanted side effects. The results will help determine whether iptacopan could become a standard long-term treatment for young people living with these rare conditions.
Key takeaways
- This study is an extension trial for young people who completed an earlier iptacopan study.
- It covers two conditions: paroxysmal nocturnal haemoglobinuria (PNH) and IgA nephropathy (IgAN).
- The main goal is to test the long-term safety and tolerability of iptacopan.
- All participants receive the study medicine; there is no placebo.
- Taking part involves regular hospital visits for blood tests, urine tests, and general check-ups.
Who may be eligible?
This study is only open to young people who have already taken part in and successfully finished an earlier iptacopan clinical trial (known as a 'parent study') for either PNH or IgA nephropathy. People who have not been in one of these specific previous trials cannot join.
Male and female participants of any age who finished the previous trial may be considered. To take part, participants must be able to keep taking the study medicine as prescribed and be willing to attend regular hospital check-ups.
The study doctor will also review each person's general health to ensure that continuing the treatment remains safe. Anyone who developed a new, serious health issue or a severe reaction during the first study may not be able to continue.
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.
- Have you or your child completed a previous paediatric iptacopan clinical trial?
- Do you have a diagnosis of either PNH or IgA nephropathy?
- Are you able to take the study medicine as instructed by the doctors?
- Can you attend regular hospital appointments for tests and safety check-ups?
- Has the study doctor confirmed that continuing iptacopan is medically safe for you?
What does participation involve?
Taking part means continuing to take iptacopan as directed by the study team. Participants will need to visit their hospital clinic for regular monitoring appointments. During these visits, doctors will check vital signs like blood pressure and temperature, perform gentle physical examinations, and ask about any symptoms or side effects. Participants will give regular blood samples to check their red blood cell counts and kidney health, and those with IgA nephropathy will provide urine samples to measure protein levels. The study is planned to last over a long period to monitor long-term safety, and the study team will explain the exact schedule of clinic visits before you decide to continue.
Potential risks and benefits
Locations (1)
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Common questions
What is an extension study?
An extension study allows people who finished an earlier clinical trial to keep receiving the treatment so doctors can learn about its long-term safety and effects.
Will anyone receive a placebo (dummy pill)?
No. This is an open-label study, which means every participant receives the real medicine, and both you and your doctor know you are taking it.
How is iptacopan taken?
Iptacopan is taken by mouth, usually as a capsule or tablet, following the exact dosing routine prescribed by the study doctor.
Can I leave the study if I change my mind?
Yes. You are completely free to leave the trial at any point, for any reason, and your regular medical care will continue as normal.
Are there extra costs to take part?
No. The study medicine and all related tests are provided free of charge, and travel costs to hospital visits are typically reimbursed.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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