Safety and Efficacy Study of KL002 in the Treatment of Advanced Primary PD

Study Overview
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Brief Summary
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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
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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.

Official Title
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Safety and Efficacy Study of KL002 in the Treatment of Advanced Primary Parkinson's Dissease

Conditions
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Parkinson Disease

Intervention / Treatment
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* Genetic: KL002 injection solution


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Males/females between 40 and 70 years (inclusive) Diagnosed with idiopathic Parkinson's disease, Hoehn and Yahr stage 4 and 5 Disease duration at least 5 years MDS UPDRS Part III (total motor) score ≥ 25 and a maximum of 60 in the OFF state Good compliance and can complete all follow-up in accordance with the protocol requirements Exclusion Criteria: Prior brain surgery including deep brain stimulation or abnormal brain imaging. Presence of depression as measured by Hamilton Depression Scale ≥20 History of brain injury or central nervous system infection. Cognitive impairment score<26 on MoCA and ≤ 20 on MMSE dementia scale Evidence of significant medical or psychosis, such as dementia, psychosis, and a history of drug abuse 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. Prior gene therapy. Chronic immunosuppressive therapy, including chronic steroids, immunotherapy, cytotoxic therapy and chemotherapy Any other conditions that the investigator believed unsuitable for participation in this study.

Ages Eligible for Study
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Minimum Age: 40 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: KL002 injection solution<br>single dose, neurosurgically infused, bilaterally into the striatum | Genetic: KL002 injection solution<br>* Neurosurgical delivery of KL002 to the brain<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Treatment Emergent Adverse Events | Safety and Tolerability of KL002 assessed by Treatment Emergent Adverse Events | Day 0 through month 24 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Parkinson's Symptoms | 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. | Day 0 through month 24 | 
| PET Scan Imaging | Relationship between KL002 distribution in the brain and change in dopamine expression as indicated in PET imaging. | Day 0 through month 24 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Parkinson Disease, KL002, Putamen, Stereotactic brain surgery