NCT06687837

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

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

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 8 (estimated)
• Sponsor: Jeffrey S. Schweitzer, MD, PhD · Academic / Other
• Sex: All
• Age: 45 Years – 80 Years
• Healthy volunteers: Not accepted

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.

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.

Conditions

• Parkinson Disease

Interventions

Type	Name	Description
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)

Timeline

• Start date: 2025-04-29
• Primary completion: 2027-12-01
• Completion: 2028-12-01
• First posted: 2024-11-14
• Last updated: 2026-03-25

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study