Efficacy, Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of BIA 28-6156 in GBA-PD
This research study is investigating a new medication called BIA 28-6156 for individuals living with Parkinson's disease. Specifically, it's for those whose Parkinson's is connected to a change in a gene called GBA1. The main goal is to find out if this new drug can effectively slow down the progression of movement problems, which are a key symptom of Parkinson's. Researchers will compare two different doses of BIA 28-6156 against a placebo (a dummy drug with no active ingredients) over 78 weeks. Participants will continue their usual Parkinson's medications throughout the study. This is a Phase 2 trial, meaning it's still in the earlier stages of drug development, looking at safety, effectiveness, and identifying the right dosage.
At a glance
What is this study about?
This study is exploring a new potential treatment, BIA 28-6156, for people who have Parkinson's disease that is linked to a specific genetic change in the GBA1 gene. Not everyone with Parkinson's has this genetic link, so one of the first steps in this study is to check for this particular gene change. The researchers want to understand if this new drug can help slow down the worsening of motor symptoms – the movement difficulties often experienced by people with Parkinson's.
The study will involve giving participants either one of two doses of BIA 28-6156 or a placebo (a tablet that looks the same but contains no active drug). This will be done without the participants or their doctors knowing who is receiving which treatment, to ensure the results are as fair and unbiased as possible. Over a period of 78 weeks, the study will carefully monitor participants to see if the drug is effective in delaying the progression of their motor symptoms, as well as checking for any side effects and how the body handles the drug. Participants will also continue taking their usual Parkinson's medications.
This is a Phase 2 study, which means it's an important step in researching new medications. It aims to gather more information about how well the drug works, if it's safe, and what might be the best dose, before potentially moving on to larger studies. The overall hope is that BIA 28-6156 could offer a new way to manage Parkinson's disease for individuals with this specific genetic link.
Key takeaways
- This study investigates a new drug for Parkinson's disease linked to a specific gene change (GBA1).
- Its main goal is to see if the drug can slow down the worsening of movement problems.
- Participants will receive either the drug or a placebo for 78 weeks, while continuing regular medications.
- Initial screening includes a genetic test to confirm the GBA1 gene change.
- Close medical monitoring and regular clinic visits are part of participation.
- Participation is voluntary, and you can withdraw at any time.
Who may be eligible?
To be considered for this study, you would first need to be between 35 and 80 years old. You should have received a diagnosis of Parkinson's disease by a neurologist at least one year ago, but no longer than seven years ago. Also, your Parkinson's symptoms should be generally stable, meaning your movement difficulties are not too severe. You also need to be taking your usual Parkinson's medications and able to understand and agree to take part.
If you meet these initial criteria, the next step involves a genetic test to see if you have the specific GBA1 gene change that the study is focusing on. If you have this gene change, you would then be considered for the main part of the study. Further checks would ensure your thinking abilities are good, you don't have severe uncontrolled movements or major side effects from your current medications, and your weight is within an expected range.
There are also requirements for women who could become pregnant and men with female partners about using effective contraception throughout the study. Individuals who don't meet these specific criteria, or have certain other medical conditions, would not be able to participate.
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 35 and 80 years old?
- Have you been diagnosed with Parkinson's disease between 1 and 7 years ago?
- Are your Parkinson's symptoms generally stable (not too severe)?
- Are you currently taking your usual Parkinson's medications?
- Are you willing to have a genetic test for the GBA1 gene change?
- Are you able to attend regular clinic appointments for over 1.5 years?
What does participation involve?
If you join this study, it's divided into two main parts. The first part involves a genetic screening to check for the GBA1 gene change. If you have it, you might be invited to the second part, which involves the treatment.
In the treatment part, you'll go through a screening period of up to 5 weeks to make sure you meet all the requirements. After that, you'll be randomly assigned to receive either one of the two doses of BIA 28-6156 or a placebo (a dummy drug). Neither you nor your doctor will know which one you're receiving. This treatment period will last for up to 78 weeks (about 1.5 years). You will continue taking your regular Parkinson's medications throughout this time.
Throughout the study, you'll have regular visits to the clinic for assessments. These will include check-ups, tests of your movement and thinking, and blood tests to monitor your health and how the drug is working. After the 78-week treatment period, there will be a final safety follow-up for about a month. The total duration of your involvement could be around 80 weeks.
Potential risks and benefits
Locations (95)
- Barrow Neurological InstituteVerified postcodePhoenix, United States
- University of California San DiegoVerified postcodeLa Jolla, United States
- Cedars-SinaiVerified postcodeLos Angeles, United States
- University of ColoradoVerified postcodeAurora, United States
- Parkinson's Center and Movement Disorders of Boca RatonVerified postcodeBoca Raton, United States
- University of Miami, Dept. of NeurologyVerified postcodeMiami, United States
- Renstar Medical ResearchVerified postcodeOcala, United States
- Morehouse School of MedicineVerified postcodeAtlanta, United States
- Northwestern UniversityVerified postcodeChicago, United States
- Rush University Medical CenterVerified postcodeChicago, United States
- University of Iowa Hospitals and ClinicsVerified postcodeIowa City, United States
- University of Kansas Medical CenterVerified postcodeKansas City, United States
Common questions
What is the GBA1 gene change?
It's a specific change in a gene called GBA1 that has been linked to an increased risk of developing Parkinson's disease for some people.
What is a placebo?
A placebo is a 'dummy drug' that looks exactly like the actual medication but contains no active ingredients. It's used to fairly compare the real drug's effects.
Will I have to stop my current Parkinson's medication?
No, you will continue to take your usual Parkinson's medications at a stable dose throughout the study.
What does 'double-blind' mean?
It means that neither you nor your study doctor will know whether you are receiving the active drug or the placebo, ensuring unbiased results.
How long will I be in the study?
The treatment part of the study lasts up to 78 weeks (about 1.5 years), followed by a short safety check. The total commitment could be around 80 weeks.
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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