A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Efficacy and Safety of Dose-Titrated PLS240 in the Treatment of Secondary Hyperparathyroidism in Individuals with End Stage Kidney Disease on Hemodialysis (PATH-2) With an Open-Label Extension
This study, called PATH-2, is investigating a new medication known as PLS240. It's for adults with severe kidney disease (ESKD) who are undergoing regular kidney dialysis and also have a condition called secondary hyperparathyroidism (SHPT). SHPT means your body makes too much parathyroid hormone (PTH), which can cause health problems. The study wants to find out if PLS240 is effective at lowering PTH levels and if it's safe to use. Participants will be randomly given either PLS240 or a placebo (a dummy pill with no active medicine) for a period, with some then continuing into an open-label phase where everyone receives PLS240. This helps researchers understand the full effects of the medicine.
At a glance
What is this study about?
This research study, known as PATH-2, is looking at a new medication called PLS240. It's designed for people who have very serious kidney disease, called End-Stage Kidney Disease (ESKD), and are receiving regular kidney dialysis. Many people with ESKD also develop a condition called secondary hyperparathyroidism (SHPT). This means their parathyroid glands, which are small glands in the neck, produce too much of a hormone called PTH. High levels of PTH can cause various health issues, including problems with bones and blood calcium.
The main goal of this study is to see if PLS240 can effectively lower PTH levels in these patients and keep them within a healthy range. Researchers also want to understand if the medication is safe and what side effects it might have. Understanding both the benefits and potential risks is really important before any new medicine can be made widely available.
During the first part of the study, some participants will receive PLS240 and others will receive a placebo – a pill that looks exactly like PLS240 but contains no active medicine. This 'blinded' approach helps researchers get a clear picture of how well PLS240 works compared to receiving no specific treatment. After this initial phase, all participants who wish to continue will then receive PLS240, allowing researchers to gather more safety information for a longer period.
Key takeaways
- This study is testing a new medicine, PLS240, for a common problem in people on dialysis.
- It aims to see if PLS240 can lower high hormone levels (PTH) and if it's safe.
- Participants will receive either PLS240 or a dummy pill at first, then all receive PLS240.
- Close monitoring of your health will happen throughout the study.
- Study participation is voluntary, and you can withdraw at any time.
Who may be eligible?
To be able to join this study, people must be adults, aged 18 years or older, with no upper age limit.
You must have been diagnosed with End-Stage Kidney Disease (ESKD), which means your kidneys have stopped working well, and you need to have regular haemodialysis treatment. You also need to have a history of secondary hyperparathyroidism (SHPT), which means your body is producing too much parathyroid hormone.
There might be other specific health requirements or conditions that mean someone cannot take part in the study, even if they meet these main points. These will be discussed with you by the study team.
- Are you 18 years old or older?
- Do you have severe kidney disease (ESKD)?
- Are you currently receiving regular haemodialysis?
- Have you been told you have secondary hyperparathyroidism (SHPT)?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you decide to take part in this study, it will involve several visits to the study clinic over a number of months. You will be given either the study medication (PLS240) or a placebo (a dummy pill) for the first part of the study. Neither you nor your doctor will know which you are receiving – this is to compare the effects fairly. After this, if you continue, everyone will receive PLS240.
During your visits, the study team will regularly check your health. This will include blood tests to measure your PTH levels, calcium and phosphorus levels, and other important markers. They will also check your vital signs (like blood pressure and heart rate), perform physical examinations, and take heart tracings (ECGs) to monitor your heart health. You will be asked about any side effects or changes you notice in your health.
Taking part in this study will last for a significant period – many weeks or months – to allow researchers to gather enough information about the long-term effects of the medication. The exact schedule of visits and assessments will be explained in detail by the study team.
Potential risks and benefits
Locations (4)
- —Portugal
- —Bulgaria
- —Spain
- —Poland
Common questions
What is secondary hyperparathyroidism (SHPT)?
It's a condition common in people with kidney disease where your body makes too much of a hormone called PTH, which can cause health problems.
What is haemodialysis?
Haemodialysis is a treatment that uses a machine to filter waste and excess fluid from your blood when your kidneys can no longer do this.
What does 'double-blind' mean?
It means that neither you nor your doctor will know if you are receiving the active medicine (PLS240) or a dummy pill (placebo) during the first part of the study.
Will I have to pay to be in the study?
No, you will not have to pay to participate in the study, and all study-related treatments and tests are provided free of charge.
What if I change my mind about taking part?
You can withdraw from the study at any time, for any reason, without it affecting your usual medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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