ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 (PH1)
The ILLUMINATE-C study is investigating a new medicine called Oxlumo for adults with Primary Hyperoxaluria Type 1 (PH1). PH1 is a rare genetic condition where the body produces too much of a substance called oxalate, which can build up and cause serious problems, especially in the kidneys. This study is in its final stages (Phase 3), meaning it’s looking to confirm that Oxlumo is both effective at lowering oxalate levels in the blood and safe for patients. Researchers will measure how much oxalate levels change over six months and also check on overall health, kidney function, and quality of life. The goal is to see if this medicine can make a real difference for people living with advanced PH1.
At a glance
What is this study about?
This study, called ILLUMINATE-C, is an important research project for people living with Primary Hyperoxaluria Type 1 (PH1). PH1 is a very rare and serious genetic condition. In people with PH1, the liver doesn't work correctly to break down a natural substance called oxalate. This means too much oxalate builds up in the body. This build-up can cause damage to many organs, especially the kidneys, often leading to kidney stones, kidney failure, and other issues.
The ILLUMINATE-C study is trying to understand if a new medicine, called Oxlumo, can help manage PH1. Oxlumo is designed to reduce the amount of oxalate your body produces. Researchers are looking closely at how much oxalate levels in the blood change after taking the medicine over six months. They are also checking on how the medicine affects your overall health, including your kidney function and how you feel day-to-day. This is a "Phase 3" study, which means it’s one of the final steps before a new medicine might become widely available.
The main goal of this study is to confirm if Oxlumo is safe and effective in lowering oxalate levels. Lowering oxalate is crucial in PH1 to prevent further damage to organs and improve quality of life. The study design involves carefully monitoring patients to gather detailed information about the medicine's effects. This includes regular blood tests, checks on overall health, and asking about how the condition impacts your daily life.
Key takeaways
- This study evaluates a new medicine, Oxlumo, for Primary Hyperoxaluria Type 1 (PH1).
- It aims to reduce harmful oxalate levels and improve patient health.
- Participation involves regular clinic visits and receiving injections of the study medicine.
- The study duration for active treatment and follow-up is approximately six months.
- It's for adults 18 years and older with PH1.
Who may be eligible?
This study is looking for adult patients who have Primary Hyperoxaluria Type 1 (PH1).
To join, you must be 18 years old or older. The study is open to both men and women. The researchers will check your medical history and current health to make sure the study is a good fit for you.
It's important that your doctor agrees that taking part in this study is appropriate for your specific health situation. They will help determine if you meet all the necessary requirements to participate safely.
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 18 years old or older?
- Have you been diagnosed with Primary Hyperoxaluria Type 1 (PH1)?
- Are you able to attend regular clinic appointments for about six months?
- Are you comfortable receiving injections under the skin?
What does participation involve?
If you decide to take part in the ILLUMINATE-C study, you would receive the study medicine, Oxlumo, as an injection. This medicine is typically given under the skin. You would have regular visits to the clinic over a period of about six months. During these visits, the research team would perform various assessments. This includes blood tests to measure oxalate levels, urine tests, and checks of your overall health. They might also do ultrasounds to check your kidneys and ask you questions about how you are feeling and how the condition affects your daily life (this is called quality of life assessments). The total duration of your active participation in the study, including all treatments and follow-up checks, would be around six months.
Potential risks and benefits
Locations (4)
- —UnverifiedFrance
- —UnverifiedNetherlands
- —UnverifiedBelgium
- —UnverifiedItaly
Common questions
What is Primary Hyperoxaluria Type 1 (PH1)?
PH1 is a rare genetic condition where your body produces too much of a harmful substance called oxalate, which can damage your kidneys and other organs.
What is the purpose of the medicine Oxlumo in this study?
Oxlumo is being studied to see if it can help reduce the amount of oxalate your body makes, which could help manage PH1.
How will I receive the medicine if I join?
The medicine, Oxlumo, is given as an injection, typically under your skin.
How long will I be in the study?
Your active participation, including receiving the medicine and having check-ups, would last about six months.
Can women participate in this study?
Yes, this study is open to both men and women who meet the age and health requirements.
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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