Stereotactic Intracerebral Injection of Allogenic IPSC-DAPs in Patients With Parkinson's Disease

Not Applicable

Not yet recruiting

• Conditions: Parkinson Disease
• Interventions: Biological: ALC01 therapy

• Registration Number: NCT07212088
• Lead Sponsor: iCamuno Biotherapeutics Ltd.

Brief Summary

Parkinson's disease is a progressive neurodegenerative disorder characterized by high morbidity due to the limited regenerative capacity of dopaminergic neurons in the brain. Current drug treatments primarily manage symptoms but do not halt or reverse neuronal loss. Cellular replacement therapy has emerged as a potential strategy to restore dopaminergic function and address the underlying neuronal deficits. This study aims to evaluate the safety, feasibility, and efficacy of transplanting dopaminergic neurons into the brain to improve motor function and quality of life in patients with advanced Parkinson's disease.

Detailed Description

Patients with Parkinson's disease will be treated with allogenic induced pluripotent stem cell-derived dopamine progenitor cells (iPSC-DAPs). These cells will be transplanted directly into the striatum to restore dopamine-producing capacity. Patients will be evaluated at 1, 3, 6, 9 and 12 months after transplantation for safety, feasibility, and efficacy.

Study Timeline

• Status Verified: 2025-09
• Study First Submitted: 2025-09-30
• Study First Submitted QC: 2025-09-30
• Last Update Submitted: 2025-09-30
• Study First Posted: 2025-10-08
• Last Update Posted: 2025-10-08
• Study Start: 2025-11-15
• Primary Completion: 2027-11-15
• Study Completion: 2028-06-15

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

• Able to sign informed consent and comply with the study protocol
• 55-75 years of age, at the time of signing informed consent
• Diagnosed to be Parkinson's disease patients over 5 years
• Taking levodopa, but with complications of therapy such as wearing off and/or dyskinesia
• At least 3 hours accumulative "off" time per day
• Hoehn and Yahr Stage 3 - 4 in the off state at screening
• Dopamine drug responsiveness demonstrated by a positive "on/off" test with at least a 30% improvement on UPDRS III (motor) scale

Exclusion Criteria

• Patients with the following concomitant conditions or disorders: Epilepsy；Multiple sclerosis；Unable to give consent due to dementia；Atypical Parkinsonism；Genetic Parkinson's disease；Suicidal ideation associated with intent or plan in the past 12 months；History of psychosis；History of subarachnoid hemorrhage；History of stroke or transient ischemic attack
• Patient with unstable vital sign at screening and/or prior to the surgery
• Estimated Glomerular Filtration Rate (eGFR) < 60 ml/min/1.73m2
• Liver dysfunction, as evidenced by enzymes (AST and ALT) greater than three times the ULN.
• Hematologic abnormality: hemoglobin <10 mg/dL or platelet count < 100,000/mL
• International normalized ratio (INR) ≥ 1.3 not due to a reversible cause
• Patients with autoimmune disorders
• Patients with HIV and/or active HBV or HCV
• Patients who are unable to undergo MRI and PET/CT
• Patients with an expected life expectancy of <1 year
• Patients who have had active malignancies
• Patients currently receive levodopa-carbidopa intestinal gel or apomorphine treatment
• Patients who have history of pallidotomy or thalamotomy or deep brain stimulation (DBS) surgery
• Received cell or gene therapy (autologous or allogeneic) within the previous 12 months
• Participation in an investigational therapeutic or device trial within 30 days of consent
• Women who are pregnant or breast-feeding
• Other conditions that researchers consider not suitable to participate in this study

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Low Dose ALC01 therapy	ALC01 therapy	Stereotactic Intracerebral Injection of iPSC-DAPs
High Dose ALC01 therapy	ALC01 therapy	Stereotactic Intracerebral Injection of iPSC-DAPs

Primary Outcome Measures

Name	Time	Method
Incidence and servility of Treatment-Emergent Adverse Events	From baseline to 12 months post surgery	Incidence of adverse event (AE), serious adverse event(SAE) is defined as the composite of number and severity of adverse events, regardless of causality, clinical laboratory abnormalities, clinical meaningful changes from baseline

Secondary Outcome Measures

Name	Time	Method
Changes in motor function	From baseline to 12 months post surgery	Changes in motor function that measured by Movement and Disorders Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) part III. TheMDA- UPDRS Part III score ranges from 0 to 132, with higher score indicating greater disability
Changes in the Hoehn and Yahr scale	From baseline to 12 months post surgery	Hoehn and Yahr scale is used to provide a general estimate of clinical function of PD patients, combining functional deficits (disability) and objective signs (impairment). The Hoehn and Yahr score ranges from 0 to 5, with higher score indicating higher dysfunction of PD patients
Changes in quality of life (QoL)	From baseline to 12 months post surgery	Measured by Parkinson's Disease Quality of Life Rating Scale (PDQ-39) from baseline to 12 months post surgery.; PDQ-39 score ranges from 0 to 156 and provides the evidence of the quality of life of a PD patient. The higher the score, the lower the quality of life of PD patients.
Change in PD medication usage	From baseline to 12 months post surgery	Change in the Parkinson's disease medication usage as defined by the levodopa equivalent daily dose (LEDD)
18-F DAT PET uptake	Baseline, 6 and 12 months post surgery	Changes in putamen 18F DAT PET uptake that measured by PET/CT

Trial Locations

Locations (1)

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