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

Primary Purpose

Parkinson Disease

Status
Not yet recruiting
Phase
Early Phase 1
Locations
China
Study Type
Interventional
Intervention
KL002 injection solution
Sponsored by
Second Affiliated Hospital, School of Medicine, Zhejiang University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Parkinson Disease focused on measuring Parkinson Disease, KL002, Putamen, Stereotactic brain surgery

Eligibility Criteria

40 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Second Affiliated Hospital of Zhejiang University School of Medicine

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

KL002 injection solution

Arm Description

single dose, neurosurgically infused, bilaterally into the striatum

Outcomes

Primary Outcome Measures

Incidence of Treatment Emergent Adverse Events
Safety and Tolerability of KL002 assessed by Treatment Emergent Adverse Events

Secondary Outcome Measures

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.
PET Scan Imaging
Relationship between KL002 distribution in the brain and change in dopamine expression as indicated in PET imaging.

Full Information

First Posted
May 22, 2023
Last Updated
May 22, 2023
Sponsor
Second Affiliated Hospital, School of Medicine, Zhejiang University
Collaborators
R&D Kanglin Biotech
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1. Study Identification

Unique Protocol Identification Number
NCT05882487
Brief Title
Safety and Efficacy Study of KL002 in the Treatment of Advanced Primary PD
Official Title
Safety and Efficacy Study of KL002 in the Treatment of Advanced Primary Parkinson's Dissease
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
June 1, 2023 (Anticipated)
Primary Completion Date
April 30, 2026 (Anticipated)
Study Completion Date
June 30, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Second Affiliated Hospital, School of Medicine, Zhejiang University
Collaborators
R&D Kanglin Biotech

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Parkinson Disease
Keywords
Parkinson Disease, KL002, Putamen, Stereotactic brain surgery

7. Study Design

Primary Purpose
Treatment
Study Phase
Early Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
9 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
KL002 injection solution
Arm Type
Experimental
Arm Description
single dose, neurosurgically infused, bilaterally into the striatum
Intervention Type
Genetic
Intervention Name(s)
KL002 injection solution
Intervention Description
Neurosurgical delivery of KL002 to the brain
Primary Outcome Measure Information:
Title
Incidence of Treatment Emergent Adverse Events
Description
Safety and Tolerability of KL002 assessed by Treatment Emergent Adverse Events
Time Frame
Day 0 through month 24
Secondary Outcome Measure Information:
Title
Parkinson's Symptoms
Description
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.
Time Frame
Day 0 through month 24
Title
PET Scan Imaging
Description
Relationship between KL002 distribution in the brain and change in dopamine expression as indicated in PET imaging.
Time Frame
Day 0 through month 24

10. Eligibility

Sex
All
Minimum Age & Unit of Time
40 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Yang Ruan, M.D.
Phone
+86 18858736151
Email
11818236@zju.edu.cn
First Name & Middle Initial & Last Name or Official Title & Degree
Jiali Pu, Ph.D.
Email
jialipu@zju.edu.cn
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Baorong Zhang, M.D.
Organizational Affiliation
The Second Affiliated Hospital Zhejiang University School of Medicine Hangzhou, Zhejiang province, China, 310009
Official's Role
Principal Investigator
Facility Information:
Facility Name
Second Affiliated Hospital of Zhejiang University School of Medicine
City
Hangzhou
State/Province
Zhejiang
ZIP/Postal Code
310000
Country
China

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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