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

June 20, 2023 updated by: Xuanwu Hospital, Beijing

Safety and Efficacy of Autologous Induced Neural Stem Cell-derived Dopaminergic Precursor Cells in the Treatment of Parkinson's Disease

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Parkinson's Disease (PD) is the second most common neurodegenerative disease, caused by progressive depletion of midbrain dopaminergic neurons in the substantia nigra pars compacta. This clinical study will include the preparation of dopaminergic neural precursor cells derived from neural stem cells through reprogramming of patient's peripheral blood mononuclear cells (PBMCs), and transplantation of the obtained cells into the brains of PD patients by stereotaxic injection. Safety, tolerability, evidence of cell survival (using PET scan), and the efficacy on PD symptoms will be assessed at different time points up to 12 months post-transplantation.

Study Type

Interventional

Enrollment (Estimated)

10

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • China
    • Beijing
      • Beijing, Beijing, China, 100053
        • Recruiting
        • Xuanwu Hospital Capital Medical University
        • Contact:
        • Principal Investigator:
          • Zhiguo Chen, Ph.D.
        • Principal Investigator:
          • Biao Chen, M.D., Ph.D.
        • Principal Investigator:
          • Guoguang Zhao, M.D., Ph.D.
        • Principal Investigator:
          • Jie Lu, M.D., Ph.D.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

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%).

Exclusion Criteria:

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Autologous induced neural stem cell-derived DA precursor cells
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
The autologous induced neural stem cell-derived DA precursor cells will be stereotactically implanted into the striatum of PD patients.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence and severity of transplant-related adverse events.
Time Frame: Within 12 months post-transplantation
Within 12 months post-transplantation
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.
Time Frame: Within 12 months post-transplantation
Minimum score: 0; Maximum score: 132; Higher scores mean a worse outcome.
Within 12 months post-transplantation

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Assessment of changes in the daily total medication "off" time in comparison with baseline values.
Time Frame: Within 12 months post-transplantation
Within 12 months post-transplantation
Assessment of changes in daily levodopa-equivalent dose in comparison with baseline values.
Time Frame: Within 12 months post-transplantation
Within 12 months post-transplantation
Assessment of changes in Parkinson's Disease Questionnaire (PDQ-39) in comparison with baseline values.
Time Frame: Within 12 months post-transplantation
Within 12 months post-transplantation
Assessment of changes in AV133 PET imaging in comparison with baseline values.
Time Frame: Within 12 months post-transplantation
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 I, in comparison with baseline values.
Time Frame: Within 12 months post-transplantation
Minimum score: 0; Maximum score: 52; Higher scores mean a worse outcome.
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.
Time Frame: Within 12 months post-transplantation
Minimum score: 0; Maximum score: 52; Higher scores mean a worse outcome.
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.
Time Frame: Within 12 months post-transplantation
Minimum score: 0; Maximum score: 24; Higher scores mean a worse outcome.
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.
Time Frame: Within 12 months post-transplantation
Within 12 months post-transplantation
Assessment of changes in Unified Dyskinesia Rating Scale in comparison with baseline values.
Time Frame: Within 12 months post-transplantation
Minimum score: 0; Maximum score: 104; Higher scores mean a worse outcome.
Within 12 months post-transplantation
Assessment of changes in International Parkinson and Movement Disorder Society - Non-Motor Rating Scale (NMSS), in comparison with baseline values.
Time Frame: Within 12 months post-transplantation
Minimum score: 0; Maximum score: 960; Higher scores mean a worse outcome.
Within 12 months post-transplantation

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Xuanwu Hospital, Beijing

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 13, 2023

Primary Completion (Estimated)

December 31, 2025

Study Completion (Estimated)

December 31, 2026

Study Registration Dates

First Submitted

June 5, 2023

First Submitted That Met QC Criteria

June 5, 2023

First Posted (Actual)

June 13, 2023

Study Record Updates

Last Update Posted (Actual)

June 22, 2023

Last Update Submitted That Met QC Criteria

June 20, 2023

Last Verified

May 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CTC001

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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