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

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

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

Study Overview

• Status: Not yet recruiting
• Conditions: Parkinson Disease
• Intervention / Treatment: Genetic: KL002 injection solution

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.

• Study Type: Interventional
• Enrollment (Estimated): 9
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Yang Ruan, M.D.; Phone Number: +86 18858736151; Email: 11818236@zju.edu.cn
• Study Contact Backup: Name: Jiali Pu, Ph.D.; Email: jialipu@zju.edu.cn

Study Locations

• China
  • Zhejiang
    • Hangzhou, Zhejiang, China, 310000
      • Second Affiliated Hospital of Zhejiang University School of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: KL002 injection solution; single dose, neurosurgically infused, bilaterally into the striatum	Genetic: KL002 injection solution; Neurosurgical delivery of KL002 to the brain

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Second Affiliated Hospital, School of Medicine, Zhejiang University
• Collaborators: R&D Kanglin Biotech
• Investigators: Principal Investigator: Baorong Zhang, M.D., The Second Affiliated Hospital Zhejiang University School of Medicine Hangzhou, Zhejiang province, China, 310009

Study record dates

Study Major Dates

• Study Start (Estimated): June 1, 2023
• Primary Completion (Estimated): April 30, 2026
• Study Completion (Estimated): June 30, 2026

Study Registration Dates

• First Submitted: May 22, 2023
• First Submitted That Met QC Criteria: May 22, 2023
• First Posted (Actual): May 31, 2023

Study Record Updates

• Last Update Posted (Actual): May 31, 2023
• Last Update Submitted That Met QC Criteria: May 22, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Keywords: Parkinson Disease; KL002; Putamen; Stereotactic brain surgery
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Parkinsonian Disorders; Basal Ganglia Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases; Parkinson Disease
• Other Study ID Numbers: 2023-0013

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No