A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel Study to Assess the Efficacy, Safety, Tolerability, PK, and Biomarker Effects of PTC857 in Adult Subjects With Amyotrophic Lateral Sclerosis (CARDINALS)
This research study is looking at a new medicine called PTC857 for people living with Amyotrophic Lateral Sclerosis (ALS), also known as Motor Neurone Disease. We want to find out if PTC857 can help slow down the disease’s effects on daily physical activities and if it improves how long people live. We are also checking how safe the medicine is and if it causes any side effects. Participants will either receive PTC857 or a dummy medicine (placebo), or another existing treatment called Utreloxastat. This will help us understand if PTC857 is effective and well-tolerated. The study lasts for 24 weeks.
At a glance
What is this study about?
This study is a ‘Phase 2’ clinical trial. This means we are in the middle stages of testing a new medication called PTC857 for people with Amyotrophic Lateral Sclerosis (ALS), which is also known as Motor Neurone Disease. We are trying to find out several important things: first, if the new medicine actually helps people with their daily activities and slows down the disease. Second, we want to understand how safe the medicine is and if it causes any unwanted side effects when compared to a dummy medicine (a placebo) or another existing treatment. We will also investigate how the body handles the medicine.
To figure this out, some people in the study will get the new medicine (PTC857), some will get a dummy medicine that looks exactly the same, and some might get another medicine called Utreloxastat. Neither you nor your study doctor will know which treatment you are receiving until the end of the study. This setup helps us be sure that any changes we see are truly due to the new medicine and not just other factors. Your health and progress will be carefully monitored over 24 weeks.
The main goal is to see if PTC857 can help preserve your ability to do everyday tasks like eating, dressing, and walking, and if it might improve your chances of survival. We also want to measure things like your breathing, how well your limbs are working, and your overall quality of life. By understanding these effects, we hope to learn if PTC857 could be a helpful new option for people living with ALS.
Key takeaways
- This study is testing a new medicine (PTC857) for people with ALS/Motor Neurone Disease.
- It aims to see if PTC857 can help with daily activities and improve survival over 24 weeks.
- Participants will receive PTC857, a dummy medicine, or Utreloxastat, but won't know which one.
- Your safety and health will be closely monitored throughout the study.
- It is a research study and may or may not personally benefit you, but helps future patients.
Who may be eligible?
To be considered for this study, you must be an adult, aged 18 years or older. There is no upper age limit, meaning older adults can also take part.
Both men and women are welcome to participate. The study team will need to check your medical history and current health to make sure this is the right study for you. There will be specific medical requirements that your doctor will discuss with you to ensure your safety and that the study results are clear.
- Are you 18 years old or older?
- Have you been diagnosed with Amyotrophic Lateral Sclerosis (ALS) / Motor Neurone Disease?
- Are you able to attend regular clinic visits for 24 weeks?
- Are you generally willing to take an oral liquid medicine?
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, you will be in the study for about 24 weeks (roughly 6 months). During this time, you will have regular visits to the study clinic. These visits will involve various health checks, such as physical examinations, monitoring your breathing with lung function tests, and checking your heart with an ECG (a heart tracing). You’ll also be asked questions about your daily activities and how you are feeling to assess your quality of life. Blood and possibly spinal fluid samples will be taken to see how the medicine is working in your body. You will be given either the study medicine (PTC857), a dummy medicine, or Utreloxastat, which you will take by mouth as a solution. The study team will carefully guide you on how to take your medicine throughout the 24 weeks. After the main treatment period, there will be follow-up checks to ensure your continued well-being.
Potential risks and benefits
Locations (11)
- —Germany
- —Italy
- —Norway
- —Ireland
- —Sweden
- —Spain
- —Poland
- —France
- —Belgium
- —Netherlands
- —Czechia
Common questions
What is ALS?
ALS stands for Amyotrophic Lateral Sclerosis, also known as Motor Neurone Disease. It's a condition that affects the brain and nerves, causing muscles to weaken over time.
What is a 'Phase 2' study?
A Phase 2 study is a stage of clinical research where a new medicine is tested in a small group of people to see if it works and to check its safety, after initial safety tests.
What is a 'placebo'?
A placebo is a dummy medicine that looks exactly like the real medicine but contains no active drug. It helps researchers understand if the new medicine's effects are genuine.
Will I know if I'm getting the real medicine?
No, this is a 'double-blind' study, meaning neither you nor your study doctor will know whether you are receiving the active medicine, the placebo, or Utreloxastat until the study finishes.
How long will I be in the study?
You will be in the study for 24 weeks, which is about 6 months, plus some follow-up checks.
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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