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

NCT ID: NCT05882487

Last Updated: 2023-05-31

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: EARLY_PHASE1
• Total Enrollment: 9 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-06-01
• Study Completion Date: 2026-06-30

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.

Conditions

Parkinson Disease

Keywords

Parkinson Disease; KL002; Putamen; Stereotactic brain surgery

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

KL002 injection solution

single dose, neurosurgically infused, bilaterally into the striatum

Group Type: EXPERIMENTAL

KL002 injection solution

Intervention Type: GENETIC

Neurosurgical delivery of KL002 to the brain

Interventions

KL002 injection solution

Neurosurgical delivery of KL002 to the brain

Intervention Type: GENETIC

Eligibility Criteria

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.

• Minimum Eligible Age: 40 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

R&D Kanglin Biotech

OTHER

Sponsor Role: collaborator

Second Affiliated Hospital, School of Medicine, Zhejiang University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Baorong Zhang, M.D.

Role: PRINCIPAL_INVESTIGATOR

The Second Affiliated Hospital Zhejiang University School of Medicine Hangzhou, Zhejiang province, China, 310009

Locations

Second Affiliated Hospital of Zhejiang University School of Medicine

Hangzhou, Zhejiang, China

Countries

China

Central Contacts

Yang Ruan, M.D.

Role: CONTACT

Phone: +86 18858736151

Email: 11818236@zju.edu.cn

Jiali Pu, Ph.D.

Role: CONTACT

Email: jialipu@zju.edu.cn

Other Identifiers

2023-0013

Identifier Type: -

Identifier Source: org_study_id