Parkinson Disease
Conditions
Keywords
Parkinson Disease, KL002, Putamen, Stereotactic brain surgery
Brief summary
This is a non-randomized, open label, dose-escalation study to evaluate the safety and efficacy of KL002 in the treatment of advanced primary Parkinson's disease. Condition or disease: Parkinson's Disease Intervention/treatment: Drug: KL002 Phase: NA
Detailed description
9 patients will be enrolled to this open-label dose escalation study to evaluate up to three dose levels of KL002 of three patients each. All patients will receive bilateral intraputaminal injections of KL002 through stereotactic brain surgery. The safety and potential clinical responses to KL002 will be assessed by clinical evaluations of Parkinson's disease, cognitive tests, laboratory blood tests and neuroimaging.
Interventions
GENETICKL002 injection solution
Neurosurgical delivery of KL002 to the brain
Sponsors
R&D Kanglin Biotech
Second Affiliated Hospital, School of Medicine, Zhejiang University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
40 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
1. Males/females between 40 and 70 years (inclusive) 2. Diagnosed with idiopathic Parkinson's disease, Hoehn and Yahr stage 4 and 5 3. Disease duration at least 5 years 4. MDS UPDRS Part III (total motor) score ≥ 25 and a maximum of 60 in the OFF state 5. Good compliance and can complete all follow-up in accordance with the protocol requirements
Exclusion criteria
1. Prior brain surgery including deep brain stimulation or abnormal brain imaging. 2. Presence of depression as measured by Hamilton Depression Scale ≥20 3. History of brain injury or central nervous system infection. 4. Cognitive impairment score\<26 on MoCA and ≤ 20 on MMSE dementia scale 5. Evidence of significant medical or psychosis, such as dementia, psychosis, and a history of drug abuse 6. Atypical or secondary parkinsonism, including but not limited to symptoms believed to be due to trauma, brain tumor, infection, cerebrovascular disease, other neurological disease, or to drugs, chemicals, or toxins. 7. Prior gene therapy. 8. Chronic immunosuppressive therapy, including chronic steroids, immunotherapy, cytotoxic therapy and chemotherapy 9. Any other conditions that the investigator believed unsuitable for participation in this study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of Treatment Emergent Adverse Events | Day 0 through month 24 | Safety and Tolerability of KL002 assessed by Treatment Emergent Adverse Events |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Parkinson's Symptoms | Day 0 through month 24 | Effect of KL002 on Parkinson's symptoms as recorded in subject diaries, neurological, motor, and non-motor assessments, quality of life surveys and changes to Parkinson's medications. |
| PET Scan Imaging | Day 0 through month 24 | Relationship between KL002 distribution in the brain and change in dopamine expression as indicated in PET imaging. |
Countries
China
Contacts
Primary ContactYang Ruan, M.D.
Backup ContactJiali Pu, Ph.D.
Outcome results
None listed