Safety and Pharmacodynamic Effects of BIIB122 in Participants With LRRK2-Associated Parkinson's Disease (LRRK2-PD)
This study is looking into a new medicine called BIIB122 for people with a particular form of Parkinson's disease that runs in families because of a specific gene (LRRK2). It's a 12-week study where some people will get BIIB122 and others will get a dummy medicine, so we can compare them. The main goals are to check if BIIB122 is safe, how well people tolerate it, and if it has any effects on the body. Understanding this could help us develop new treatments for LRRK2-related Parkinson's.
At a glance
What is this study about?
This research study is about a new drug called BIIB122, which is being investigated for a specific type of Parkinson's disease. This particular type of Parkinson's is linked to a change in a gene called LRRK2. If you have this gene change, it means there's a higher chance of developing Parkinson's, and in these cases, the disease might work a little differently.
The main aim of this study is to see if BIIB122 is safe for people with LRRK2-related Parkinson's disease and if it causes any side effects. We also want to understand how the medicine affects the body. By learning more about how BIIB122 interacts with the LRRK2 gene, we hope to find new ways to help people living with this condition.
This is a 'Phase 2' study, which means it's still in the earlier stages. Drugs often go through several phases of testing before they can be widely used. Even though it's early, studies like this are vital for discovering new and better treatments for Parkinson's disease in the future. The information we gather will help researchers decide if BIIB122 should move on to larger studies.
Key takeaways
- This study is for people with Parkinson's disease linked to the LRRK2 gene.
- It's testing a new medicine called BIIB122 for safety and how it works.
- The study lasts 12 weeks, with an option to continue afterwards.
- You might receive the new medicine or a dummy pill.
- Your health will be closely monitored throughout the study.
Who may be eligible?
To join this study, you need to be a certain age and have a specific type of Parkinson's disease. You must be between 30 and 80 years old if you have one copy of the changed LRRK2 gene, or 30 years old or more if you have two copies. You'll also need a genetic test to confirm you have this specific LRRK2 gene change, and a doctor's diagnosis of Parkinson's disease.
There are also reasons why you might not be able to join. For example, if you have other neurological problems like a stroke or certain types of memory loss, or if you have a different kind of Parkinson's that isn't typical. If you've had brain surgery for your Parkinson's, or participated in another study using a gene therapy or this particular BIIB122 drug, you won't be able to join this one.
Finally, if you have other health issues that could make it hard to properly check your movement, like very bad arthritis or certain injuries, or if you have severe breathing problems or unstable blood pressure, you might not be eligible. These rules are in place to keep participants safe and make sure the study results are clear and accurate.
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 that has been diagnosed by a doctor?
- Do I know if I have a changed LRRK2 gene?
- Am I generally between 30 and 80 years old?
- Have I had brain surgery for Parkinson's? (If yes, I might not be eligible)
- Do I have any other significant long-term health problems?
- Have I taken part in other gene therapy studies for Parkinson's or a previous BIIB122 study?
What does participation involve?
If you decide to take part in this study, you will be assigned by chance to either receive the study medicine, BIIB122, or a dummy medicine called a placebo. This is a "double-blind" study, meaning neither you nor your study doctor will know which one you are getting. The treatment period will last for 12 weeks.
Throughout the study, you will have regular visits to the clinic where doctors and nurses will monitor your health. They will check your vital signs like blood pressure and heart rate, take blood samples, and perform physical examinations. You will also have assessments to monitor your Parkinson's symptoms, likely involving movement tests. After the initial 12 weeks, there will be an 'open-label extension' period, where everyone who chooses to continue will receive the BIIB122 medicine, and your health will continue to be monitored. The total duration of your participation, including follow-up, will be explained by the study team.
Potential risks and benefits
Locations (20)
- Cedars-Sinai Department of NeurologyVerified postcodeLos Angeles, United States
- University of California San FranciscoVerified postcodeSan Francisco, United States
- Parkinson's Disease and Movement Disorders CenterVerified postcodeBoca Raton, United States
- Beth Israel Deaconess Medical CenterVerified postcodeBoston, United States
- Ichan School of Medicine at Mount Sinai/Beth Israel Downtown-Movement Disorder CenterVerified postcodeNew York, United States
- Evergreen Health LaboratoryVerified postcodeKirkland, United States
- Inland Northwest ResearchVerified postcodeSpokane, United States
- Technische Universität DresdenVerified postcodeDresden, Germany
- University of LübeckVerified postcodeLübeck, Germany
- University Hospital TübingenVerified postcodeTübingen, Germany
- Rabin Medical CenterVerified postcodePetah Tikva, Israel
- Movement Disorders Institute, Sheba Medical CenterVerified postcodeRamat Gan, Israel
Common questions
What is LRRK2-related Parkinson's disease?
It's a type of Parkinson's disease linked to a specific change in a gene called LRRK2, which can be passed down in families.
What does 'Phase 2' mean for a drug study?
Phase 2 means the drug is still in early testing. Researchers are checking its safety and how it affects the body in a small group of people.
Will I know if I'm getting the real medicine or a dummy pill?
No, this is a 'double-blind' study, so neither you nor your doctor will know which you are receiving during the initial 12 weeks.
How long will I be in the study?
The main treatment period is 12 weeks, followed by an optional longer 'open-label' period where everyone gets the study drug.
Can I leave the study if I change my mind?
Yes, you are free to withdraw from the study at any time, for any reason, and it won't affect your regular medical care.
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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