AuthorisedTherapeutic use (Phase IV)Interventional

(ASK-PD0-CS002) Long-Term Safety and Efficacy Follow-up of AB-1005 Gene Transfer Study Participants with Parkinson’s Disease or Multiple System Atrophy

This ongoing study is following up with individuals who have Parkinson's disease or Multiple System Atrophy and previously received a gene therapy called AAV2-GDNF. The main goal is to carefully monitor the long-term safety of this treatment by checking for any side effects, both mild and serious. Researchers are also looking at how well the treatment is working over time. For those with Parkinson's, this includes tracking their daily symptoms, how much medication they need, and using special brain scans. For individuals with Multiple System Atrophy, the study assesses how their symptoms are managed and their overall quality of life. Participants will continue to receive close medical attention to understand the lasting effects of the gene therapy.

At a glance

Status

Authorised

Phase

Therapeutic use (Phase IV)

Sponsor

Askbio Inc.

Enrolment target

Start

22 Dec 2025

Multiple System Atrophy Parkinson's Disease

What is this study about?

Imagine you or someone you know has Parkinson's disease or Multiple System Atrophy. Researchers have been developing a special gene therapy, called AAV2-GDNF, to help manage these conditions. This particular study isn't about giving new treatments; instead, it's a very important follow-up to check on people who have already received this therapy in previous studies.

The main purpose of this study is to see how safe and effective the AAV2-GDNF gene therapy has been over a long period. Think of it like checking in on someone after they've had a significant medical treatment to make sure they're doing well and to understand the lasting effects. The medical team will be closely monitoring participants for any new or ongoing side effects, whether they are minor or more serious. This helps them understand the full picture of the treatment's safety.

Beyond safety, the study also looks at how well the treatment helps with symptoms. For those with Parkinson's, this means carefully tracking their daily 'on' and 'off' times, how their movement is, and if they need adjustments to their regular medication. They might also have special brain scans. For people with Multiple System Atrophy, the study will assess how their condition affects them in daily life and their general well-being. By gathering all this information, researchers hope to learn more about the long-term impact of this gene therapy and help advance future treatments.

Key takeaways

This study is following up on a gene therapy previously given for Parkinson's or Multiple System Atrophy.
The main goals are to assess long-term safety and how effective the treatment has been.
It's only for people who have already received the specific gene therapy (AAV2-GDNF).
Participation involves ongoing health check-ups and symptom monitoring.
Data collected will help understand the long-term effects of this gene therapy.
You have the right to withdraw from the study at any point.

Who may be eligible?

To be part of this follow-up study, you must have previously taken part in one of the clinical trials where you received the AAV2-GDNF gene therapy.

Participants need to be at least 18 years old. There's no upper age limit mentioned, meaning older adults are welcome to participate if they meet the other criteria.

This study is open to both men and women who meet the above conditions.

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.

Did you previously receive the AAV2-GDNF gene therapy in another study?
Are you 18 years old or older?
Are you able to attend appointments for follow-up assessments?
Are you currently diagnosed with Parkinson's Disease or Multiple System Atrophy?

Answer every question to see your result.

What does participation involve?

If you are already part of this study, your participation involves ongoing check-ups. The study team will monitor your health carefully, including any changes in your symptoms or how you're feeling. They will be looking for any side effects, both those that are minor and those that are more serious, to understand the long-term safety of the gene therapy. For participants with Parkinson's, assessments might include logging your daily 'on' and 'off' times, checking your movement and thinking abilities, and sometimes having special brain scans (called 18F-DOPA PET) to see how your brain is responding. For those with Multiple System Atrophy, assessments will focus on your symptoms and quality of life. You'll likely have scheduled visits with the study team to complete these assessments, and the total duration of your continued follow-up will depend on the study's plan.

Potential risks and benefits

The main benefit of taking part in this follow-up is contributing valuable information that could help others with Parkinson's disease or Multiple System Atrophy in the future. While this study does not offer new treatment, you will receive close medical monitoring. The main risk involved would be any discomfort or small risks associated with the assessments themselves, such as blood tests or scans. There is always a risk that new, unexpected side effects from the gene therapy could emerge over time. Remember, you can choose to stop participating in the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Poland

Common questions

What is the main purpose of this study?

The main purpose is to check the long-term safety and how well a gene therapy, called AAV2-GDNF, has worked for people with Parkinson's disease or Multiple System Atrophy who received it in earlier studies.

Am I receiving new treatment in this study?

No, this study is a follow-up for people who have already received the AAV2-GDNF gene therapy. It's about monitoring how you're doing after that treatment.

Who can take part in this follow-up study?

You can take part if you previously received the AAV2-GDNF gene therapy in another clinical trial, are 18 years or older, and can be male or female.

What will I have to do if I'm in this study?

You'll have regular medical check-ups and assessments to track your symptoms, health, and any side effects. This might include questionnaires, physical exams, and sometimes scans.

Can I stop being in the study if I change my mind?

Yes, you are free to leave the study at any time, for any reason, and it will not affect your ongoing medical care.