Gene therapy improves motor control
- Trial ID
- NCT07100171
- Official Title
- A Single-Center, Open-Label, Single-Arm Exploratory Study Evaluating GM101 Injection in Patients With Mid-to-Late Stage Parkinson's Disease
- Goal
- Gene therapy improves motor control
- Phase
- EARLY_PHASE1
- Status
- RECRUITING
- Sponsor
- Genemagic Biosciences Co., Ltd
- Study Type
- INTERVENTIONAL
- Enrollment
- 10 participants
- Conditions
- Parkinson's Disease
- Interventions
- Biological: GM101
Summary For Families
GM101 injection aims to help people with mid-to-late stage Parkinson's whose symptoms are no longer well controlled by medicines, focusing on improving motor function and daily control. It is a one-time, neurosurgically delivered gene therapy using an AAV8 viral vector to put a therapeutic gene into targeted brain regions, so it works alongside existing drugs rather than immediately replacing levodopa. The trial requires adults 40 to 70 years old with at least 5 years of Parkinson's, severe disability in the off state (Hoehn‑Yahr 4,5), a strong levodopa response, and a stable anti-Parkinson medication regimen. People who had prior brain surgery, prior gene or cell therapy, high AAV8 antibodies, or major medical or psychiatric conditions are excluded.
Locations
- Xiangya Hospital, Central South University, Changsha, China
Frequently Asked Questions
- What is this trial testing?
- This trial is studying Biological: GM101. GM101 injection aims to help people with mid-to-late stage Parkinson's whose symptoms are no longer well controlled by medicines, focusing on improving motor function and daily control. It is a one-time, neurosurgically delivered gene therapy using an AAV8 viral vector to put a therapeutic gene into targeted brain regions, so it works alongside existing drugs rather than immediately replacing levodopa. The trial requires adults 40 to 70 years old with at least 5 years of Parkinson's, severe disability in the off state (Hoehn‑Yahr 4,5), a strong levodopa response, and a stable anti-Parkinson medication regimen. People who had prior brain surgery, prior gene or cell therapy, high AAV8 antibodies, or major medical or psychiatric conditions are excluded.
- Who can participate?
- Participants must be between 40 Years and 70 Years.
- Where is this trial located?
- This trial is recruiting at 1 location.
- Does it cost anything to join?
- No. There is no cost to participate. Study-related care and treatment are provided at no charge.
- How long does the trial last?
- This Phase 1 trial is estimated to last approximately 1 year and 9 months.
- Will I get the real drug or a placebo?
- It depends on the trial design. Some Parkinson's trials are placebo controlled, meaning a portion of participants get an inactive comparison while others get the experimental treatment. Some use crossover designs so everyone eventually receives the active treatment at some point. Observational trials do not use a placebo at all because they are not testing a new treatment. The consent form for any specific trial spells out the design and your odds of being assigned to placebo before you enroll.
- How often will I need to visit the study site?
- Visit frequency varies by trial. Many Parkinson's studies require an in person visit every 4 to 12 weeks during the active treatment phase, with shorter or longer gaps depending on the design. The site coordinator can give you the full visit schedule before you sign anything, so you know what the time commitment looks like.
- 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.
- Will travel or parking be reimbursed?
- Many trials reimburse for parking, mileage, and sometimes lodging if the site is far from your home. Reimbursement policies vary by sponsor and site. When you contact the trial team, ask specifically what is covered and how reimbursement is processed.
- 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.