AAV2-GDNF gene therapy for Parkinson's (NCT06285643)
Gene therapy reduces movement fluctuations
- Trial ID
- NCT06285643
- Official Title
- A Phase 2, Randomized, Double-blind, Sham Surgery-controlled Study of the Efficacy and Safety of Intraputaminal AAV2-GDNF in the Treatment of Adults With Moderate Stage Parkinson's Disease
- Goal
- Gene therapy reduces movement fluctuations
- Phase
- PHASE2
- Status
- RECRUITING
- Sponsor
- AskBio Inc
- Study Type
- INTERVENTIONAL
- Enrollment
- 127 participants
- Conditions
- Parkinson Disease
- Interventions
- AAV2-GDNF gene therapy, control surgery
Summary For Families
The goal is to see whether boosting production of GDNF in the putamen can protect or restore dopamine neurons and improve motor symptoms and troublesome on/off fluctuations in people with moderate Parkinson's. The approach uses an AAV2 viral vector injected into the putamen so local brain cells make GDNF, the procedure involves stereotactic surgery with a sham surgery control to maintain blinding, and the treatment is meant to complement levodopa rather than replace it since participants must be levodopa responsive. The trial is looking for adults 45 to 75 years old diagnosed 4 to 10 years ago who have motor fluctuations, a stable Parkinson medication regimen, and clear levodopa responsiveness, while excluding those with prior brain surgery, significant vascular or cognitive/psychiatric issues, MRI contraindications, chronic immunosuppression, or other conditions that could mimic Parkinson's.
Locations
- Mayo Clinic, Phoenix, Arizona, United States
- UCI Alpha Clinic (Neurology), Irvine, California, United States
- Loma Linda University (Neurology), Loma Linda, California, United States
- Loma Linda University (Surgical), Loma Linda, California, United States
- University of California San Francisco (Neurology), San Francisco, California, United States
- University of San Francisco (Surgical), San Francisco, California, United States
- CenExel Rocky Mountain Clinical Research (Neurology), Englewood, Colorado, United States
- Georgetown University (Neurology), Washington D.C., District of Columbia, United States
- Parkinson's Disease and Movement Disorders Center of Boca Raton (Neurology), Boca Raton, Florida, United States
- University of Florida, Gainesville, Florida, United States
- University of Miami, Miami, Florida, United States
- Orlando Health Neuroscience Institute, Orlando, Florida, United States
- Parkinson's Disease Treatment Center of Southwest Florida (Neurology), Port Charlotte, Florida, United States
- Emory University (Surgical), Atlanta, Georgia, United States
- Rush University Medical Center (Neurology), Chicago, Illinois, United States
- University of Kansas Medical Center (Neurology), Kansas City, Kansas, United States
- Massachusetts General Hospital (Neurology), Boston, Massachusetts, United States
- Massachusetts General Hospital (Surgical), Boston, Massachusetts, United States
- Michigan State University (Neurology), East Lansing, Michigan, United States
- Quest Research Institute (Neurology), Farmington Hills, Michigan, United States
And 26 more locations.
Frequently Asked Questions
- What is this trial testing?
- This trial is studying AAV2-GDNF gene therapy. The goal is to see whether boosting production of GDNF in the putamen can protect or restore dopamine neurons and improve motor symptoms and troublesome on/off fluctuations in people with moderate Parkinson's. The approach uses an AAV2 viral vector injected into the putamen so local brain cells make GDNF, the procedure involves stereotactic surgery with a sham surgery control to maintain blinding, and the treatment is meant to complement levodopa rather than replace it since participants must be levodopa responsive. The trial is looking for adults 45 to 75 years old diagnosed 4 to 10 years ago who have motor fluctuations, a stable Parkinson medication regimen, and clear levodopa responsiveness, while excluding those with prior brain surgery, significant vascular or cognitive/psychiatric issues, MRI contraindications, chronic immunosuppression, or other conditions that could mimic Parkinson's.
- Who can participate?
- Participants must be between 45 Years and 75 Years.
- Where is this trial located?
- This trial is recruiting at 46 locations.
- Does it cost anything to join?
- You are never charged a fee to join a legitimate clinical trial, and the study treatment and study-specific tests are provided by the trial sponsor at no charge. In the US, routine care you would receive anyway can still be billed to you or your insurance; coverage rules differ by country, and trials in the UK and Canada usually run through public health systems. Travel, parking, and time are real costs, and some trials reimburse them while others do not. Before enrolling, ask the study coordinator to explain in writing what is and is not covered.
- How long does the trial last?
- This Phase 2 trial is estimated to last approximately 4 years and 3 months.
- Can I leave the trial if I change my mind?
- Yes. You can withdraw from any clinical trial at any time, for any reason, without affecting your standard medical care. Trials are voluntary by law. The team may ask if you are willing to do a brief exit visit so they can collect safety information, but you are not obligated.
- Can my spouse or care partner come with me to visits?
- In most cases yes, and it is often encouraged. Care partners can help with notes, questions, and getting home safely after a long visit. Some study assessments do need to happen one on one, but care partners are usually welcome for the rest of the appointment.
- Will I get a placebo in this trial?
- This trial has 2 groups, and 1 is a sham (a pretend version of the procedure) group. Because assignment is random, you have about a 1 in 2 chance (roughly 50%) of being in the sham group, assuming the groups are filled equally. Ask the coordinator to confirm the exact assignment ratio.
- What procedures are involved in this trial?
- Based on the protocol, this trial involves: Surgical procedure (invasive). Confirm the full schedule with the study coordinator.