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

Early Phase 1

Not yet recruiting

• Conditions: Parkinson Disease
• Interventions: Genetic: KL002 injection solution

• Registration Number: NCT05882487
• Lead Sponsor: Second Affiliated Hospital, School of Medicine, Zhejiang University

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.

Study Timeline

• Status Verified: 2023-05
• Study First Submitted: 2023-05-22
• Study First Submitted QC: 2023-05-22
• Last Update Submitted: 2023-05-22
• Study First Posted: 2023-05-31
• Last Update Posted: 2023-05-31
• Study Start: 2023-06-01
• Primary Completion: 2026-04-30
• Study Completion: 2026-06-30

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 9

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
KL002 injection solution	KL002 injection solution	single dose, neurosurgically infused, bilaterally into the striatum

Primary Outcome Measures

Name	Time	Method
Incidence of Treatment Emergent Adverse Events	Day 0 through month 24	Safety and Tolerability of KL002 assessed by Treatment Emergent Adverse Events

Secondary Outcome Measures

Name	Time	Method
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.
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.

Trial Locations

Locations (1)

Second Affiliated Hospital of Zhejiang University School of Medicine; 🇨🇳 Hangzhou, Zhejiang, China