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

NCT06687837 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: 2024P001089
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Parkinson Disease

Outcome Measures

Primary Outcomes (1)

• Safety and Tolerability

  Incidence and severity of adverse events and serious adverse events

  2 years from time of implantation

Secondary Outcomes (7)

• 18-F DOPA PET uptake

  2 years from time of implantation

• Change in motor function

  2 years from time of implantation

• Change in "off" hours

  2 years from time of implantation

• Change in dyskinesia

  2 years from time of implantation

• Change in PD medication usage

  2 years from time of implantation

Study Arms (2)

Low dose administration

EXPERIMENTAL

4 million autologous dopaminergic cells will be implanted into the putamen on each side of the brain

Biological: autologous dopaminergic cell implantation

High dose administration

EXPERIMENTAL

8 million autologous dopaminergic cells will be implanted into the putamen on each side of the brain

Biological: autologous dopaminergic cell implantation

Interventions

autologous dopaminergic cell implantation BIOLOGICAL

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)

High dose administration; Low dose administration

Eligibility Criteria

• Age: 45 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Jeffrey S. Schweitzer, MD, PhD: lead

Study Sites (1)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

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

Jeffrey S Schweitzer, MD, PhD

CONTACT

6177261799; JSCHWEITZER1@mgh.harvard.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: George A. Lopez, MD Endowed Chair in Neurosurgery

Model Details: Open label, two dosage tiers

Study Record Dates

• First Submitted: October 24, 2024
• First Posted: November 14, 2024
• Study Start: April 29, 2025
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2028
• Last Updated: March 25, 2026

Record last verified: 2026-03

Locations

US (1)