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Parkinson DiseasePhase 1NCT06687837

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

A Phase 1 study testing autologous dopaminergic cell implantation in people with Parkinson Disease, run by Jeffrey S. Schweitzer, MD, PhD at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

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. Read more about this study

Who is it for?
Ages 45 to 80 years
Conditions the study is for
Parkinson Disease
What is being tested?
autologous dopaminergic cell implantation
Could I receive a placebo?
No, everyone receives an active treatment
How many people
8 participants
Where
1 siteUnited States
Study dates
29 Apr 2025to Dec 2028
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 45 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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%
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  • 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:
  • Platelets > 100×109/L (transfusion independent)
  • Prothrombin time / partial thromboplastin time in normal range and international normalized ratio ≤ 1.3
  • Aspartate aminotransferase and alanine aminotransferase < 2.5x the upper limit of normal
  • Serum creatinine ≤ 1.5mg/dL
  • White blood cell count < 12×109/L.
  • 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.

Who cannot join

  • 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.
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  • 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

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Jeffrey S Schweitzer, MD, PhD contact

6177261799

JSCHWEITZER1@mgh.harvard.edu

United States1 site, 1 recruiting

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

    Jeffrey S Schweitzer, MD, PhD, 617-726-1799, jschweitzer1@mgh.harvard.edu

    Dolores Smith, 617-726-1799, vprathivadi@mgh.harvard.edu

    Recruiting

Other trials in United States

About this study

Official title: 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.

Read the 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.

Being tested
autologous dopaminergic cell implantation
Medical subject headings
Parkinson Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • autologous dopaminergic cell implantationBiological

    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)

Study groups in NCT06687837
Study groupRoleWhat they receive
Low dose administrationExperimental4 million autologous dopaminergic cells will be implanted into the putamen on each side of the brain
Receives: Biological: autologous dopaminergic cell implantation
High dose administrationExperimental8 million autologous dopaminergic cells will be implanted into the putamen on each side of the brain
Receives: Biological: autologous dopaminergic cell implantation

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Safety and Tolerability

    Incidence and severity of adverse events and serious adverse events

    Measured: 2 years from time of implantation

Secondary outcomes (7)

  1. 18-F DOPA PET uptake

    Change in putaminal 18F DOPA PET uptake from baseline to 12 and 24-month follow-up.

    Measured: 2 years from time of implantation

  2. Change in motor function

    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.

    Measured: 2 years from time of implantation

  3. Change in "off" hours

    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.

    Measured: 2 years from time of implantation

  4. Change in dyskinesia

    Change in the frequency and severity of dyskinesia as assessed by the MDS-UDysRS from baseline to 12 and 24-month follow-up.

    Measured: 2 years from time of implantation

  5. Change in PD medication usage

    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.

    Measured: 2 years from time of implantation

  6. Change in Parkinson's disease related quality of life

    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.

    Measured: 2 years from time of implantation

  7. Global impression of change

    Patient and Clinician Global Impression of Change (PGI-I and CGI-I) assessed at 12 and 24-months after implantation

    Measured: 2 years from time of implantation

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted14 Nov 2024
  2. Study start29 Apr 2025
  3. Primary completionDec 2027 (estimated)
  4. Study completionDec 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Model description
Open label, two dosage tiers
Masking
None (open label)
Enrolment
8 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06687837
Sponsor's protocol ID
2024P001089
Status last verified
Mar 2026
First submitted
24 Oct 2024
Last updated
25 Mar 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Jeon J, Cha Y, Hong YJ, Lee IH, Jang H, Ko S, Naumenko S, Kim M, Ryu HL, Shrestha Z, Lee N, Park TY, Park H, Kim SH, Yoon KJ, Song B, Schweitzer J, Herrington TM, Kong SW, Carter B, Leblanc P, Kim KS. Pre-clinical safety and efficacy of human induced pluripotent stem cell-derived products for autologous cell therapy in Parkinson's disease. Cell Stem Cell. 2025 Mar 6;32(3):343-360.e7. doi: 10.1016/j.stem.2025.01.006. Epub 2025 Feb 13.

    PubMed 39952239

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing autologous dopaminergic cell implantation in people with Parkinson Disease, run by Jeffrey S. Schweitzer, MD, PhD at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 18 inclusion and 24 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 25 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Jeffrey S Schweitzer, MD, PhD (6177261799, JSCHWEITZER1@mgh.harvard.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Safety and Tolerability (time frame: 2 years from time of implantation). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06687837, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Mar 2026.

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