RecruitingPHASE2INTERVENTIONAL

A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)

This research study, called REGENERATE-PD, is looking into a new treatment for people with moderate Parkinson's disease. The treatment is a gene therapy called AAV2-GDNF, which is given directly to a specific part of the brain called the putamen during a single surgical procedure. The main goals are to find out how safe this new therapy is and how well it works to reduce Parkinson's symptoms. To do this, some participants will receive the gene therapy, while others will have a control surgery. Doctors will not know who gets which treatment to ensure the results are fair. This is a Phase 2 study, which means it builds on earlier research and aims to gather more information on effectiveness and safety.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

AskBio Inc

Enrolment target

127

Start

11 Jun 2024

Estimated completion

31 Aug 2028

Parkinson Disease

What is this study about?

This study is called REGENERATE-PD, and it's designed to investigate a new treatment for people living with Parkinson's disease. Specifically, it focuses on those with moderate symptoms. Parkinson's is a condition where certain brain cells that produce a chemical called dopamine gradually die. This leads to problems with movement, such as tremor, stiffness, and slow movement. Existing treatments can help manage symptoms, but researchers are always looking for new ways to slow down or even reverse the progression of the disease.

The new treatment being tested is a 'gene therapy' called AAV2-GDNF. Gene therapy aims to introduce new genetic material into cells to help them function better. In this case, AAV2-GDNF is designed to help brain cells produce a special protein called GDNF. GDNF is believed to support the survival and function of dopamine-producing cells, which could potentially slow down the progression of Parkinson's disease or improve symptoms. This therapy is delivered directly into a specific area of the brain called the putamen during a single surgical procedure.

This is a Phase 2 study, meaning it's a step after initial safety tests. The main goals are to see how safe the AAV2-GDNF gene therapy is and how well it helps people with their Parkinson's symptoms. To ensure fair results, the study is 'randomised,' which means participants are put into groups by chance. It's also 'double-blinded,' so neither the participants nor their doctors will know whether they're receiving the active gene therapy or a control procedure. This helps prevent any bias when assessing the results.

Key takeaways

This study is testing a new gene therapy for moderate Parkinson's disease.
It aims to see if the therapy is safe and improves symptoms.
Participation involves a single brain surgery.
The study compares the gene therapy to a control surgery.
Results will help understand if this new treatment could be a future option.

Who may be eligible?

To join this study, you need to be an adult between 45 and 75 years old. Doctors will check if you've been diagnosed with Parkinson's disease between 4 and 10 years ago. You also need to have certain movement difficulties like slow movement, stiffness, tremor, or balance issues, and your symptoms should change throughout the day. It's important that your current Parkinson's medications have been stable for at least 4 weeks before you join, and that your symptoms improve when you take your levodopa medication.

There are also some reasons why you might not be able to join. For example, if you have other serious medical, genetic, or brain conditions that could be confused with Parkinson's, or severe heart problems, you wouldn't be suitable. People with issues like significant memory problems, severe depression or anxiety that aren't well controlled, or a history of psychosis (seeing or hearing things that aren't there) or impulse control problems (like gambling addiction) also can't take part. If you've had certain types of brain surgery before, like deep brain stimulation, or if you can't have an MRI scan, you won't be able to participate. Also, if you’re currently taking medication to suppress your immune system (outside of normal anti-rejection meds) or have had certain types of cancer (excluding some skin cancers), you won’t be included.

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 45 and 75 years old?
Were you diagnosed with Parkinson's disease 4 to 10 years ago?
Are your Parkinson's medications stable for at least 4 weeks?
Do your Parkinson's symptoms improve when you take levodopa?
Do you have movement changes throughout the day?
Do you have no history of brain surgery or severe heart problems?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you'll first go through a screening process to make sure you're a good fit. If you are, you'll be randomly assigned to either receive the AAV2-GDNF gene therapy or a control surgery. Both involve a single surgical procedure where a special substance is delivered to a specific part of your brain. During the recovery period and for a period afterwards, you will have regular visits with the study team. These visits will involve various tests and assessments to check your health, Parkinson's symptoms, and how you're responding to the treatment. This will include movement tests, questionnaires, and possibly brain scans (MRIs). You'll continue to take your regular Parkinson's medications throughout the study, and your doctors will monitor for any changes. The total duration of your participation in the study, including follow-up, will be several years to ensure all safety and effectiveness data can be collected.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. For potential benefits, this study is testing a new gene therapy that might help improve Parkinson's symptoms or slow the disease's progression. However, because it's a new treatment, it's not guaranteed to work, and we don't fully know all its effects yet. Potential risks include those associated with brain surgery, such as infection, bleeding, or other complications. There might also be side effects from the gene therapy itself. The study team will carefully monitor your health throughout. You have the right to withdraw from the study at any time, for any reason, without affecting your regular medical care.

Locations (39)

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

UCI Alpha Clinic (Neurology)
Verified postcode
Irvine, United States· Recruiting
Loma Linda University (Neurology)
Verified postcode
Loma Linda, United States· Recruiting
Loma Linda University (Surgical)
Verified postcode
Loma Linda, United States· Active not recruiting
University of California San Francisco (Neurology)
Verified postcode
San Francisco, United States· Recruiting
University of San Francisco (Surgical)
Verified postcode
San Francisco, United States· Active not recruiting
CenExel Rocky Mountain Clinical Research (Neurology)
Verified postcode
Englewood, United States· Recruiting
Georgetown University (Neurology)
Verified postcode
Washington D.C., United States· Recruiting
Parkinson's Disease and Movement Disorders Center of Boca Raton (Neurology)
Verified postcode
Boca Raton, United States· Recruiting
Parkinson's Disease Treatment Center of Southwest Florida (Neurology)
Verified postcode
Port Charlotte, United States· Active not recruiting
Emory University (Surgical)
Verified postcode
Atlanta, United States· Active not recruiting
Rush University Medical Center (Neurology)
Verified postcode
Chicago, United States· Recruiting
University of Kansas Medical Center (Neurology)
Verified postcode
Kansas City, United States· Recruiting

Common questions

What is gene therapy?

Gene therapy is a type of treatment that aims to add new genetic material into cells, which can help them work better or produce a helpful protein that your body needs.

What is the putamen?

The putamen is a small, deep part of your brain that plays an important role in controlling your movements.

What does 'double-blinded' mean?

Double-blinded means that neither you nor your doctor will know whether you are receiving the active gene therapy or the control surgery. This helps ensure fair and unbiased results.

Will I still take my usual Parkinson's medicines?

Yes, you will continue to take your regular Parkinson's medications throughout the study, and these will be carefully monitored by the study team.

Is surgery involved in this study?

Yes, a single surgical procedure is involved for all participants, whether they receive the active gene therapy or the control procedure.

How to find out more

Nisha Chhabria, MD

askfirst@askbio.com 919-388-1040 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.