Treating Parkinson's Disease Through Transplantation of Autologous Stem Cell-Derived Dopaminergic Neurons

March 22, 2026 updated by: Jeffrey S. Schweitzer, MD, PhD

Phase I Trial of Autologous Induced Pluripotent Stem Cell-derived Dopaminergic Progenitor Cell Transplantation for Parkinson's Disease

The goal of this clinical trial is to assess the safety and tolerability of the surgical transplantation of dopaminergic progenitor cells into the brains of participants with Parkinson's disease. The transplanted dopaminergic cells will be derived from the participant's own skin cells.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

This Phase I, open-label clinical trial aims to assess the feasibility and safety of autologous midbrain dopaminergic progenitor cell (mDAP) transplantation for the treatment of Parkinson's disease. mDAPs will be produced for each participant from a fibroblast sample and then transplanted bilaterally into the putamen under general anesthesia. The study will assess the safety and tolerability of the cell transplant procedure through clinical assessments and neuroimaging (CT, MRI and 18F-DOPA PET) over a 2-year follow-up period.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
8

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Diagnosis of Parkinson's Disease consistent with the Movement Disorders Society 2015 Parkinson's diagnostic criteria.
  • Age 45 - 80 years
  • English proficiency sufficient to understand the consent form and participate in a discussion of risks and benefits
  • At least 5 years since Parkinson's disease motor symptom onset
  • Modified Hoehn and Yahr stage 3-4 in "off"-medication state
  • Motor symptoms responsive to levodopa and/or dopamine agonist, defined as taking at least 300 mg/day of levodopa and exhibiting improvement between "off" and "on" MDS-UPDRS of at least 30%
  • At least 3 hours of cumulative "off" time per day
  • Stable regimen of Parkinson's medications, including levodopa and dopamine agonists, for at least 4 weeks prior to screening.

  • Acceptable surgical laboratory values including:

    1. Platelets > 100×109/L (transfusion independent)
    2. Prothrombin time / partial thromboplastin time in normal range and international normalized ratio ≤ 1.3
    3. Aspartate aminotransferase and alanine aminotransferase < 2.5x the upper limit of normal
    4. Serum creatinine ≤ 1.5mg/dL
    5. White blood cell count < 12×109/L.
    6. Estimated glomerular filtration rate ≥ 30 mL/min/1.73m2
  • Subject agrees to defer elective neurological surgery, including deep brain stimulation or lesional procedure for PD, invasive treatments, including levodopa or apomorphine infusion, or pump- pump-administered levodopa intestinal gel, until after the study's primary outcome is completed.
  • Findings on baseline 18F-DOPA PET imaging consistent with dopaminergic denervation of the putamen
  • Subject is willing and able to comply with all study visits and procedures in the opinion of the Investigator.

Exclusion Criteria:

  • Subjects unable to give consent due to dementia or psychosis.
  • Montreal Cognitive Assessment (MoCA) score < 26
  • Subjects with a first-degree relative with Parkinson's disease or with a known genetic mutation predisposing to the development of Parkinson's disease (i.e. this initial study is confined to the more common "sporadic" vs a "genetic" form of the disease).
  • Atypical Parkinsonism (Parkinson's-Plus syndrome, secondary parkinsonism)
  • Moderate or severe levodopa-induced dyskinesias in any body segment (such patients were found to be more prone to graft-induced dyskinesias in the fetal tissue studies that are proof of priniciple for this therapy)
  • Neurologic history or imaging demonstrating brain pathology not directly related to Parkinson's disease that is likely to interfere with study compliance or assessment of Parkinson's related motor disability.
  • History of stroke or transient ischemic attack
  • History of subarachnoid hemorrhage
  • Presence or history of psychosis within 12 months of screening
  • Suicidal ideation associated with intent or plan in the past 12 months (an answer of "yes" to C-SSRS questions 4 or 5) or with a previous history of suicide attempts in the past 5 years.
  • History of intracranial surgery including deep brain stimulation, focused ultrasound, stereotactic or radiosurgical lesion therapy
  • History of malignancy within 5 years. Exceptions will be made for treated cutaneous squamous cell or basal cell carcinoma without evidence of metastasis.
  • Use of anticoagulation / antiplatelet agents that cannot be stopped for one week in advance of and two days following surgery without significant risk to the subject
  • Use of chronic immunosuppressive therapy including chronic steroids
  • Contraindication to MRI or MRI contrast agents
  • Pregnant or nursing women
  • Subjects with active cardiovascular and cerebrovascular disease within 6 months prior to signing the informed consent form.
  • History of severe heart failure (congestive heart failure of New York Heart Association Class II or above or left ventricular ejection fraction < 35% by any examination method), unstable angina pectoris and myocardial infarction/
  • Severe arrhythmia
  • History of cardiovascular surgery (cardiac, vascular stent surgery, angioplasty)
  • Patients with major vascular diseases (aortic aneurysm, aortic dissecting aneurysm, internal carotid artery stenosis)
  • Hypertensive patients with poorly controlled blood pressure (defined as blood pressure consistently above 160/100 mmHg despite treatment with antihypertensive drugs) and patients with severe postural hypotension
  • Diabetic patients with poorly controlled blood glucose (glycosylated hemoglobin > 9.0%, or fasting plasma glucose (FPG) ≥ 11.1 mmol/L);
  • Subjects with alcohol or drug addiction

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: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Low dose administration
4 million autologous dopaminergic cells will be implanted into the putamen on each side of the brain
Biological: autologous dopaminergic cell implantation
Dopaminergic progenitor cells derived from autologous induced pluripotent stem cells will be injected into the brain in two cohorts of Parkinson's patients, one receiving low dose and the other high dose (4 and 8 million cells, respectively)
Experimental: High dose administration
8 million autologous dopaminergic cells will be implanted into the putamen on each side of the brain
Biological: autologous dopaminergic cell implantation
Dopaminergic progenitor cells derived from autologous induced pluripotent stem cells will be injected into the brain in two cohorts of Parkinson's patients, one receiving low dose and the other high dose (4 and 8 million cells, respectively)

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Safety and Tolerability
Time Frame: 2 years from time of implantation
Incidence and severity of adverse events and serious adverse events
2 years from time of implantation

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
18-F DOPA PET uptake
Time Frame: 2 years from time of implantation
Change in putaminal 18F DOPA PET uptake from baseline to 12 and 24-month follow-up.
2 years from time of implantation
Change in motor function
Time Frame: 2 years from time of implantation
Change in the on-state and practically defined off-state motor function as assessed by the MDS-UPDRS part III from baseline to 12 and 24-month follow-up.
2 years from time of implantation
Change in "off" hours
Time Frame: 2 years from time of implantation
Change in the number of waking hours spent in the "off" state as measured using a self-reported symptom diary, comparing baseline to 12 and 24-month follow-up.
2 years from time of implantation
Change in dyskinesia
Time Frame: 2 years from time of implantation
Change in the frequency and severity of dyskinesia as assessed by the MDS-UDysRS from baseline to 12 and 24-month follow-up.
2 years from time of implantation
Change in PD medication usage
Time Frame: 2 years from time of implantation
Change in the Parkinson's disease medication usage as defined by the levodopa equivalent daily dose (LEDD) from baseline to 12 and 24-month follow-up.
2 years from time of implantation
Change in Parkinson's disease related quality of life
Time Frame: 2 years from time of implantation
Change in the Parkinson's disease-related quality of life as assessed by the change in PDQ-39 from baseline to 12 and 24-month follow-up.
2 years from time of implantation
Global impression of change
Time Frame: 2 years from time of implantation
Patient and Clinician Global Impression of Change (PGI-I and CGI-I) assessed at 12 and 24-months after implantation
2 years from time of implantation

Collaborators and Investigators

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

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Jeffrey S. Schweitzer, MD, PhD

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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 29, 2025

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 1, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2028

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 24, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 12, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 14, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 25, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 22, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 2024P001089

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

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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