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
Plain-Language Summary
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.