A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of BIA 28-6156 in Subjects With Parkinson’s Disease With a Pathogenic Variant in the Glucocerebrosidase (GBA1) Gene
This research study is testing a new medicine, BIA 28-6156, for people who have Parkinson's disease because of a specific gene fault called GBA1. The study is run in two groups; some people will get the new medicine, and others will get a dummy medicine (placebo). This helps doctors see if the new medicine is really working. The main goal is to see if the medicine can slow down how Parkinson's disease affects daily activities like eating, dressing, and walking. Researchers will also look at how safe the medicine is, how well people can cope with it, and how it behaves in the body. If successful, this could be an important step forward for treating this specific type of Parkinson's.
At a glance
What is this study about?
This study is an important step in finding new treatments for Parkinson's disease. Specifically, it focuses on people who have a particular genetic link to their Parkinson's, involving a gene called GBA1. Not everyone with Parkinson's has this gene change, but for those who do, this study is designed to see if a new medicine, BIA 28-6156, can help.
The study is set up to compare the new medicine with a 'placebo', which looks exactly like the real medicine but contains no active ingredients. This is a common and important way to test new medicines, as it helps researchers truly understand if any changes seen are due to the medicine itself or other factors. Neither the patient nor the doctor will know who is getting which, to keep the results fair and unbiased.
The main thing researchers want to find out is whether the new medicine can slow down the speed at which Parkinson's symptoms affect everyday life, such as things like moving, eating, and getting dressed. They will also be carefully watching for any side effects and how the body handles the medicine to make sure it's safe.
Key takeaways
- This study is testing a new medicine (BIA 28-6156) for Parkinson's disease linked to the GBA1 gene.
- It aims to see if the medicine can slow down the impact of Parkinson's on daily activities and movement.
- Participants will receive either the new medicine or a placebo, without knowing which one.
- Regular clinic visits will monitor health, symptoms, and potential side effects.
- It's a Phase 2 study, meaning it's an early stage of testing for effectiveness and safety.
Who may be eligible?
To join this study, people must have Parkinson's disease that is linked to a specific genetic change in the GBA1 gene. This is a very particular type of Parkinson's, so not everyone with the condition will be able to take part.
Participants need to be adults, aged 18 or older. The study is open to both men and women.
There might be other specific health requirements or conditions that would mean someone cannot join the study, so it's always best to discuss your full medical history with the study team.
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.
- Do I have Parkinson's disease?
- Has my Parkinson's been linked to a change in the GBA1 gene?
- Am I 18 years old or older?
- Am I willing to have regular hospital visits and assessments?
- Am I able to take the study medicine as instructed?
What does participation involve?
If you join this study, you would be randomly assigned to either receive the new medicine, BIA 28-6156, or a placebo (a dummy medicine without active ingredients). Neither you nor your study doctor will know which one you are receiving. You would take the medication as instructed by the study team.
Throughout the study, you would have regular visits to the clinic. During these visits, doctors and nurses would perform various assessments to check your Parkinson's symptoms, including how well you can move and do daily activities. They will also monitor your overall health, carry out blood tests, and ask questions about any side effects you might be experiencing. The total length of your participation would be explained fully by the study team.
Potential risks and benefits
Locations (8)
- —UnverifiedGermany
- —UnverifiedNetherlands
- —UnverifiedSweden
- —UnverifiedPortugal
- —UnverifiedFrance
- —UnverifiedSpain
- —UnverifiedPoland
- —UnverifiedItaly
Common questions
What is Parkinson's disease linked to the GBA1 gene?
It's a specific type of Parkinson's disease where changes in the GBA1 gene are believed to play a role in its development.
What is a 'placebo'?
A placebo is a dummy medicine that looks like the real thing but doesn't contain any active drug. It helps compare the new medicine's effects accurately.
Will I know if I'm getting the real medicine or the placebo?
No, this is a 'blinded' study, meaning neither you nor your doctor will know who is receiving which, to ensure fair results.
What kind of tests will I have during the study?
You'll have regular check-ups, assessments of your daily activities and movement, and blood tests to monitor your health and the medicine's effects.
Can I leave the study if I change my mind?
Yes, you can choose to leave the study at any time, for any reason, without it affecting your standard medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.