BBM-P002 AAV gene therapy injected into both putamen for Parkinson's disease

A Single-arm, Open-label, Single-dose Clinical Study to Evaluate the Safety and Tolerability of BBM-P002 for Stereotactic Bilateral Putamen Injection in the Treatment of Idiopathic Parkinson's Disease

This trial will try a single stereotactic injection of BBM-P002 gene therapy into both putamen to see if it is safe and tolerable for people with idiopathic Parkinson's disease who have had symptoms for at least five years.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 18 (estimated)
• Ages: 40 Years to 70 Years
• Sex: All
• Sponsor: Shanghai Xinzhi BioMed Co., Ltd. (Industry-sponsored)
• Locations: 1 site (Shanghai, Shanghai Municipality)
• Trial ID: NCT07195825 on ClinicalTrials.gov

What this trial studies

This is a Phase 1, single-arm, open-label trial that delivers BBM-P002, an AAV-based gene therapy, into the bilateral putamen using stereotactic injection and then follows participants for 52 weeks for primary safety/tolerability monitoring and up to five years for long-term safety and efficacy. The protocol includes a dose-limiting toxicity observation period and serial assessments of pharmacokinetics, pharmacodynamics, immune response, and motor function. Eligible participants must have idiopathic Parkinson's disease for at least five years, be levodopa-responsive, and be on stable dopaminergic medication during screening. Safety, tolerability, and biomarker signals will guide further development rather than definitive efficacy conclusions at this stage.

Who should consider this trial

Good fit: Adults with idiopathic Parkinson's disease of at least five years' duration who are levodopa-responsive, meet the trial's Hoehn–Yahr and MDS‑UPDRS motor score requirements during OFF periods, and can maintain stable dopaminergic medications and study restrictions are ideal candidates.

Not a fit: Patients with atypical parkinsonism, unstable medical conditions, recent or planned neurosurgical interventions, inability to comply with contraceptive or follow-up requirements, or unwillingness to pause vaccinations or other investigational treatments are unlikely to benefit from participation.

Why it matters

Potential benefit: If successful, BBM-P002 could offer a longer-lasting, targeted approach to modify disease-related pathways in the putamen and potentially improve motor symptoms with a single neurosurgical injection.

How similar studies have performed: AAV-based gene therapies have shown biological activity and acceptable safety in some early trials for neurological diseases, but robust, reproducible clinical benefit in Parkinson's disease remains limited and this approach is still experimental.

Eligibility criteria

Inclusion Criteria:

1. Participants with a diagnosis of Parkinson's disease for ≥ 5 years.
2. The Hoehn-Yahr staging meets the requirements during the OFF periods.
3. The MDS-UPDRS Part III score met the requirements during the OFF periods. the levodopa challenge test was positive
4. During the screening period, participants were required to take the same stable dose of dopamine-releasing drugs (such as levodopa) regularly
5. The participants agreed to postpone the neurosurgical procedures related to the indication treatment during the main study phase
6. The participants agreed not to participate in any other therapeutic intervention studies during the trial period
7. The participants agreed not to receive the vaccine during the main study phase
8. From the screening period until at least 52 weeks after dosing, use a reliable contraceptive method
9. The participants had good compliance and were able to undergo regular follow-ups. During the follow-up period, they were able to accurately complete the diary cards. Family members, guardians or caregivers could assist the subjects in filling out the diary cards
10. Voluntarilyvoluntarily participated in the study and signed the informed consent form

Exclusion Criteria:

1. Atypical or secondary Parkinsonism
2. Have contraindications for surgery or have previously undergone brain surgery
3. The participants has an abnormal brain MRI suggestive of brain pathology other than Parkinson's disease
4. Those with severe cognitive impairments
5. Those with severe depression or severe anxiety
6. Abnormal liver function
7. Abnormal coagulation function
8. Abnormalities in infectious disease screening
9. Currently undergoing antiviral treatment for hepatitis
10. Suffering from unstable or severe diseases in the cardiovascular, respiratory, digestive, nervous, hematological, immune and other systems
11. Suffering from malignant tumors or having a history of tumors
12. Previous history of severe allergies
13. Exclude those who have participated in other clinical trials within the past three months
14. Had received gene therapy during the screening period
15. Select those who have received stem cell treatment within the past 12 months
16. Exclude those who have used other investigational drugs within the past 4 weeks
17. During the screening period, if the individual had received live vaccines within the previous 2 months, or had a history of vaccination within the last 30 days, they would be excluded.
18. Having a history of alcohol dependence or drug addiction
19. Pregnant or lactating female participants
20. The situations that were determined by the investigator to be unsuitable for inclusion in the study

Where this trial is running

Shanghai, Shanghai Municipality

• Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine — Shanghai, Shanghai Municipality, China (Recruiting)

Study contacts

• Principal investigator: Jun Liu, MD — Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
• Study coordinator: Wenshu Luo, Ph.D

Conditions PD; gene therapy; Adeno-Associated Virus