Long-term safety and effectiveness follow-up study of AB-1005 gene therapy in patients with Parkinson’s disease or multiple system atrophy

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What is this study about?

This study involves people with Parkinson’s Disease or Multiple System Atrophy, which are conditions that affect movement and other body functions due to problems with certain brain cells. The study uses a treatment called AB-1005, which is a gene therapy designed to deliver a protective protein to specific areas of the brain. During the treatment procedure, Carbidopa tablets are given by mouth and Fluorodopa (18F) is given through a vein as an injection. AB-1005 is given as a single injection directly into a part of the brain called the putamen.

The purpose of this study is to learn more about the long-term safety and how well AB-1005 works in people with these movement disorders. This is a follow-up study for people who are currently taking part in or have previously taken part in another study testing AB-1005. The study will track participants over many years to watch for any side effects and to see how their symptoms change over time.

During the study, participants will have regular check-ups where doctors will look for any unwanted effects of the treatment and measure how well participants can move and perform daily activities. For people with Parkinson’s Disease, the study will track their motor symptoms, how much medication they need, and will use a special brain scan called 18F-DOPA PET to see how the brain is working. For people with Multiple System Atrophy, the study will measure movement abilities and quality of life using standard rating scales. The study will continue for several years to gather information about the long-term effects of this gene therapy treatment.

1 Enrollment and consent

This study is designed for individuals who are currently enrolled or were previously enrolled in a treatment study involving AB-1005.

AB-1005 is a gene therapy treatment that uses a modified virus to deliver a specific gene into brain cells. This gene helps produce a protein that may protect nerve cells.

Your participation in this follow-up study will begin after you provide your consent to be monitored for long-term safety and effectiveness.

2 Long-term monitoring visits

The study will monitor your health and condition over an extended period, until approximately December 2037.

During this time, you will attend regular follow-up visits where your safety and how well the treatment is working will be assessed.

The visits will track any side effects or health changes that may occur, whether they are serious or non-serious.

3 Assessment of motor function and daily activities

If you have Parkinson’s disease, your movement symptoms and daily functioning will be evaluated using standardized rating scales.

These assessments will measure your motor abilities both when your medication is working well (called ON state) and when it is not working as well (called OFF state).

You will be asked to keep a diary recording the time you spend in different states: feeling good with medication working, experiencing involuntary movements while on medication, and time when medication is not working effectively.

The amount of levodopa and similar medications you take daily will be recorded. Levodopa is a medication commonly used to treat Parkinson’s disease symptoms.

4 Assessment for multiple system atrophy

If you have multiple system atrophy, a different set of rating scales will be used to assess your symptoms and quality of life.

These assessments will evaluate your daily activities, motor function, and overall well-being specific to this condition.

5 Brain imaging procedures

If you have Parkinson’s disease, you may undergo brain imaging scans using a technique called 18F-DOPA PET.

For this scan, a small amount of a radioactive substance called Fluorodopa (18F) will be given to you through a vein (intravenously).

This imaging helps measure the activity of certain brain cells that are affected in Parkinson’s disease.

Before the scan, you may be asked to take Carbidopa tablets by mouth. Carbidopa helps the imaging substance work better by preventing its breakdown outside the brain.

6 Ongoing safety monitoring

Throughout the entire study period, any health problems or side effects you experience will be carefully recorded and evaluated.

This monitoring will continue for several years to understand the long-term safety profile of the gene therapy treatment you received.

Who Can Join the Study?

You must have Parkinson’s Disease, which is a condition that affects movement and can cause tremors and stiffness, or Multiple System Atrophy-Parkinsonian subtype, which is a rare condition that causes similar movement problems along with other symptoms affecting the body’s automatic functions
You must be currently participating in or have previously participated in a research study testing the medication called AB-1005
You must be willing and able to provide informed consent, which means you understand the study and agree to take part in it by signing a consent form
The study accepts both male and female participants
The study includes adults and elderly participants

Who Cannot Join the Study?

The source data does not provide specific exclusion criteria, which are reasons why a patient cannot participate in the study
Without detailed exclusion criteria listed, it is not possible to specify which medical conditions, medications, or other factors would prevent participation
Exclusion criteria typically include things like other serious illnesses, certain medications that might interfere with the study drug, pregnancy, or recent participation in other studies, but these specific details are not provided in the available information

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Specjalistyczna Praktyka Lekarska prof. Grzegorz Opala	Katowice	Poland

Other Sites

Site Name	City	Country	Status
Copernicus Podmiot Leczniczy Sp. z o.o.	Gdansk	Poland

Trial status

Country	Status	Recruitment Start
Poland	Not yet recruiting	01.12.2026

Trial locations

Investigated drugs:

AB-1005 is a gene transfer therapy being studied for people with Parkinson’s Disease or Multiple System Atrophy. This treatment works by delivering genetic material into the body to help manage the symptoms and progression of these movement disorders. The therapy is designed to provide long-term benefits by modifying how certain cells in the brain function.

Investigated diseases:

Parkinson’s Disease – Parkinson’s Disease is a progressive disorder of the nervous system that affects movement control. The disease develops when certain nerve cells in the brain gradually break down or die, leading to a shortage of a chemical messenger called dopamine. Common symptoms include trembling of the hands, arms, legs, or face, stiffness of the limbs and trunk, slowness of movement, and problems with balance and coordination. As the disease progresses, symptoms become more pronounced and may interfere with daily activities. People with this condition may also experience changes in speech, writing difficulties, and reduced automatic movements such as blinking or swinging arms while walking. The progression of symptoms varies from person to person, and the disease typically worsens gradually over many years.

Multiple System Atrophy – Multiple System Atrophy is a rare progressive disorder that affects multiple parts of the nervous system, including areas that control movement, blood pressure, and other automatic body functions. The disease occurs when certain brain cells deteriorate and die, particularly those involved in coordinating movement and regulating involuntary body processes. The Parkinsonian subtype of this condition primarily affects movement, causing symptoms similar to Parkinson’s Disease such as slow movement, stiff muscles, tremors, and balance problems. As the disease advances, people may develop difficulties with coordination, speech, swallowing, and bladder control. Blood pressure may drop significantly when standing up, causing dizziness or fainting. The condition progresses more rapidly than Parkinson’s Disease, with symptoms becoming increasingly severe over time.

Trial ID:

2025-522653-19-00

Protocol code:

ASK-PD0-CS002

NCT ID:

NCT07081841

Trial Phase:

Therapeutic confirmatory (Phase III)

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Long-term safety and effectiveness follow-up study of AB-1005 gene therapy in patients with Parkinson’s disease or multiple system atrophy

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?