Safety and Efficacy of Autologous iNSC-DAP in the Treatment of Parkinson's Disease

NCT05901818 | Recruiting Phase 1

• 1 other identifier: CTC001
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

This is a phase I, interventional, single arm, open-label, clinical study to evaluate the safety and efficacy of the striatal transplantation of autologous induced neural stem cell-derived DA precursor cells in Parkinson's Disease patients.

Conditions

Parkinson's Disease

Keywords

Autologous; iNSC; DA precursor cells; Parkinson's Disease; Cell therapy; Stereotaxic injection

Outcome Measures

Primary Outcomes (2)

• Assessment of changes during the medication "off" time in Movement Disorder Society-Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS), part III, in comparison with baseline values.

  Minimum score: 0; Maximum score: 132; Higher scores mean a worse outcome.

  Within 12 months post-transplantation

• Incidence and severity of transplant-related adverse events.

  Within 12 months post-transplantation

Secondary Outcomes (10)

• Assessment of changes in Movement Disorder Society-Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS), part I, in comparison with baseline values.

  Within 12 months post-transplantation

• Assessment of changes in Movement Disorder Society-Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS), part II, in comparison with baseline values.

  Within 12 months post-transplantation

• Assessment of changes in Movement Disorder Society-Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS), part IV, in comparison with baseline values.

  Within 12 months post-transplantation

• Assessment of changes during the medication "off" time in motor function by using Purdue Pegboard test and gait analyzer, in comparison with baseline values.

  Within 12 months post-transplantation

• Assessment of changes in the daily total medication "off" time in comparison with baseline values.

  Within 12 months post-transplantation

Study Arms (1)

Autologous induced neural stem cell-derived DA precursor cells

EXPERIMENTAL

The patients will receive transplantation of autologous induced neural stem cell-derived DA precursor cells.

Drug: Autologous induced neural stem cell-derived DA precursor cells

Interventions

Autologous induced neural stem cell-derived DA precursor cells DRUG

The autologous induced neural stem cell-derived DA precursor cells will be stereotactically implanted into the striatum of PD patients.

Autologous induced neural stem cell-derived DA precursor cells

Eligibility Criteria

• Age: 30 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Ages between 30 and 85 years, males or females; Diagnosed to be Parkinson's disease patients according to MDS Parkinson's disease diagnostic criteria; Disease history over 3 years; Hoehn and Yahr Stage less than or equal to 4 during the medication "on" time; Responsive to levodopa treatment (Maximum rate of improvement in MDS-UPDRS, part 3, is over 30%).

You may not qualify if:

• Atypical Parkinsonian syndrome or secondary Parkinsonian syndrome; Accompanied with other central nervous system diseases; With other severe systemic diseases or dysfunction; With severe psychiatric disorders; Subjects are using hormone or cytotoxic drugs and cannot stop taking the drug during the trial; With cognitive disorders (MMSE\<24); With severe dyskinesia (MDS-UPDRS part 4, score in 4.1/4.2 ≥ 2); Subjects have undergone previous brain surgery; Subjects are long-term user of anticoagulant; Subjects have intracranial lesions which may affect the surgery or follow-up studies as assessed by imaging; Subjects are unable to undergo MRI or AV133 PET examination; Pregnancy or in preparation for pregnancy; Not suitable to participate in this clinical trial as assessed by the study investigators/physicians.

Sponsors & Collaborators

• Xuanwu Hospital, Beijing: lead

Study Sites (1)

Xuanwu Hospital Capital Medical University

Beijing, Beijing Municipality, 100053, China

RECRUITING

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Central Study Contacts

Zhiguo Chen, Ph.D.

CONTACT

+86-10-83198889; chenzhiguo@gmail.com

Jinghong Ma, M.D.

CONTACT

+86-10-83198677; jinghongma@163.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Single Group Assignment

Study Record Dates

• First Submitted: June 5, 2023
• First Posted: June 13, 2023
• Study Start: June 13, 2023
• Primary Completion: December 31, 2025
• Study Completion (Estimated): December 31, 2026
• Last Updated: June 22, 2023

Record last verified: 2023-05

Locations

CN (1)