Study of Efficacy and Safety of Iptacopan in Participants With IC-MPGN
This study is looking into a new medicine called iptacopan for a serious kidney condition known as IC-MPGN (Immune Complex Mediated Membranoproliferative Glomerulonephritis). We want to find out if iptacopan is effective and safe for people aged 12 to 60. Participants will either receive iptacopan or a dummy medicine (placebo), alongside their usual care. The main goals are to see if iptacopan can reduce the amount of protein in the urine and improve how well the kidneys are working. We'll also check if it helps with tiredness. This is a "Phase III" study, which means it's one of the final steps before a medicine might be made available to everyone. Your participation could help us understand more about treating IC-MPGN.
At a glance
What is this study about?
This study is a crucial step in understanding if a new medicine called iptacopan can help people living with a specific type of kidney disease called Immune Complex Mediated Membranoproliferative Glomerulonephritis, often shortened to IC-MPGN. This condition affects the tiny filters in your kidneys, which can cause them to not work as well as they should, leading to problems like protein leaking into your urine. Current treatments aim to manage the condition, but new options are always needed.
The research aims to compare iptacopan with a 'dummy' medicine (known as a placebo). This comparison is important to be sure that any positive changes seen are due to the new medicine and not other factors. All participants, regardless of whether they receive iptacopan or the placebo, will also continue to receive their standard kidney disease care. The study is particularly interested in whether iptacopan can reduce the amount of protein found in urine and improve kidney function, which are key indicators of kidney health.
We also want to understand if the medicine can help with symptoms like fatigue, which can be very common and impact daily life for people with kidney disease. The study is designed to be very thorough and carefully monitored to ensure the safety of everyone taking part. After the main study, participants may have the option to continue receiving iptacopan in a separate extension study, which allows for longer-term monitoring of the medicine's effects.
Key takeaways
- This study tests a new medicine (iptacopan) for a kidney disease called IC-MPGN.
- It aims to see if iptacopan can improve kidney function and reduce protein in urine.
- Participants will get either iptacopan or a dummy medicine (placebo), plus their usual care.
- The study includes adults (18-60) and adolescents (12-17).
- Close medical monitoring will be provided for all participants.
- You can withdraw from the study at any time without affecting your care.
Who may be eligible?
To join this study, you must be male or female, and between 12 and 60 years old. If you're an adult (18-60), you need to have been diagnosed with IC-MPGN through a kidney biopsy within the last year. If you're an adolescent (12-17), the biopsy must have been within the last three years. If an adult hasn't had a recent biopsy, one might be needed at the start of the study.
Before you can join, you must have been taking certain blood pressure medicines like ACE inhibitors or ARBs for at least 90 days at the highest dose you can manage. Other kidney-protecting medicines you're taking should also have been at a steady dose for at least 90 days. Your urine protein levels and kidney function also need to meet specific levels for the study.
You also need to have received important vaccinations against certain infections (meningitis and pneumonia) before starting the study medicine. If you haven't, or need a booster, this will need to be done at least two weeks before treatment begins, or you might need to take antibiotics as a precaution.
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.
- Are you between 12 and 60 years old?
- Have you been diagnosed with IC-MPGN through a kidney biopsy?
- Have you been on stable doses of your kidney medicines (like blood pressure drugs) for at least 3 months?
- Do your urine protein levels and kidney function fall within specific ranges?
- Have you been properly vaccinated against meningitis and pneumonia, or are you willing to do so?
What does participation involve?
If you decide to take part in this study, you will be randomly assigned to either receive the new medicine, iptacopan, or a dummy medicine (placebo). You won't know which one you are receiving, and neither will your study doctor, which helps ensure fair results. Both iptacopan and the placebo will be taken in addition to your current standard care for IC-MPGN.
Throughout the study, you will have regular visits to the clinic for assessments. These will include blood tests, urine tests to measure protein levels and kidney function, and possibly other checks to monitor your health and how you're responding to the treatment. We will also ask you questions about how you are feeling, particularly about tiredness.
The total duration of the main study treatment period will involve taking the medicine for a set amount of time. After this, you will have two choices: either stop the study medicine and undergo a 30-day safety check-up, or, if eligible and interested, you might have the option to continue taking iptacopan in a separate long-term extension study.
Potential risks and benefits
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Common questions
What is IC-MPGN?
It's a rare kidney disease where your immune system attacks the filtering parts of your kidneys, causing damage and issues like protein in your urine.
What is a placebo?
A placebo is a 'dummy' medicine that looks like the real treatment but contains no active ingredients. It helps us see if the new medicine is truly effective.
Will I know if I'm getting the real medicine or the placebo?
No, neither you nor your study doctor will know. This is called 'double-blind' and ensures the study results are unbiased.
What does 'standard of care' mean?
This refers to the usual and best available treatments that doctors currently use for your condition, which you will continue to receive alongside the study medicine.
How long will the study last?
The main treatment part of the study has a set duration. After that, you'll either have a short follow-up or the option to continue into an extended study.
How to find out more
Novartis Pharmaceuticals
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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