A phase 2, multicenter, randomized, double-blind, placebo-controlled study to assess the safety and efficacy of VQ-101 in participants with Parkinson’s disease carrying a heterozygous mutation of GBA1
This clinical trial is testing a new experimental tablet called VQ-101 for adults living with Parkinson’s disease. It is specifically for people who carry a change in a gene known as GBA1, which is linked to Parkinson’s. The main goal is to find out if VQ-101 can safely slow down the progression of the condition. Participants are randomly chosen to take either the real medicine or a dummy tablet (placebo) every day. Neither the patients nor the doctors will know who gets which treatment during the study. Over about 78 weeks, doctors will monitor participants closely using memory and thinking tests, blood tests, and heart traces (ECGs). This study will help researchers understand whether VQ-101 is safe, well-tolerated, and effective at helping people manage Parkinson's symptoms over the long term.
At a glance
What is this study about?
Parkinson’s disease is a condition that affects the brain, causing movement problems such as tremor, stiffness, and slowness. In some people, a specific change in a gene called GBA1 plays a role in how the condition develops. Scientists are searching for targeted treatments that can help protect brain cells in people with this particular genetic link.
This study tests a new medicine named VQ-101. Researchers want to discover if VQ-101 can slow down how quickly Parkinson’s symptoms worsen over time. They will also look closely at how the medicine affects everyday thinking, memory, and general well-being.
To make sure the results are fair and reliable, this is a 'placebo-controlled' study. This means some people will take VQ-101, while others will take an identical-looking dummy pill that contains no active medicine. By comparing the two groups, doctors can clearly see if any improvements or side effects are truly caused by VQ-101.
Key takeaways
- Tests a new daily tablet (VQ-101) for Parkinson's disease linked to the GBA1 gene.
- Aims to find out if the treatment safely slows down symptom progression.
- Compares the active drug against a dummy pill (placebo) over 78 weeks.
- Includes regular hospital check-ups, memory checks, blood tests, and heart monitoring.
- Taking part is voluntary, and you can withdraw at any time.
Who may be eligible?
This trial is open to adults aged 18 and older who have been diagnosed with Parkinson's disease. To qualify, you must carry a specific change in the GBA1 gene. If you are not sure whether you have this genetic variation, the study team or your specialist can arrange a simple test to find out.
Both men and women can take part. However, your overall health and any other medications you take will be reviewed carefully to ensure it is completely safe for you to join.
People who do not carry the GBA1 gene change, or who have certain other serious medical conditions that might interfere with the trial results, will not be able to participate. The trial team will go through the full checklist with you.
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.
- You are aged 18 or older.
- You have a confirmed diagnosis of Parkinson’s disease.
- You carry a known change in the GBA1 gene (or are willing to be tested for it).
- You are able to take daily tablets by mouth.
- You are willing to attend regular hospital appointments over roughly 18 months.
What does participation involve?
If you take part, you will be asked to take study tablets every day. Because this is a randomised study, you will have an equal chance of receiving either the active medicine (VQ-101) or a placebo dummy pill. You will take part in the study for at least 78 weeks (around a year and a half).
Throughout the study, you will visit a specialist hospital clinic regularly. During these appointments, the medical team will check your vital signs, take blood samples, and perform heart traces (ECGs) to monitor your physical health. You will also complete routine memory, puzzle, and word tests, as well as questionnaires about your mood and daily symptoms.
The doctors will also monitor whether your usual Parkinson’s medications need adjusting while you are on the trial.
Potential risks and benefits
Locations (4)
- —UnverifiedNetherlands
- —UnverifiedFrance
- —UnverifiedSpain
- —UnverifiedGermany
Common questions
What is the GBA1 gene?
GBA1 is a gene that helps cells clear waste. Some people carry a natural change in this gene that increases the risk of developing Parkinson’s disease.
What is a placebo?
A placebo is a dummy pill that looks exactly like the real medicine but contains no active ingredients. It helps doctors measure whether the real drug is truly effective.
Will I have to pay to take part?
No, clinical trials in the UK provide the study medicine and tests free of charge, and travel costs are often reimbursed.
Can I keep taking my normal Parkinson’s medication?
Yes, standard treatments are usually continued, but the trial doctors will review all your prescriptions to make sure they are safe to combine.
What happens if I change my mind?
You can leave the trial at any point, for any reason, and your regular medical team will continue to look after you as normal.
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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