NCT06422208

Autologous iPSC-Derived Dopamine Neuron Transplantation for Parkinson's Disease

A Phase 1 Clinical Trial of Autologous iPSC-Derived Dopamine Neuron Transplantation for Parkinson's Disease

• Status: Enrolling By Invitation
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 6 (estimated)
• Sponsor: Penelope J. Hallett, Ph.D. · Academic / Other
• Sex: All
• Age: 55 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

This research study is evaluating an investigational cell product called autologous induced pluripotent stem cell (iPSC)-derived dopamine neurons. This research study is a single-center Phase 1/2a clinical trial, which will test the safety of injecting the investigational cell product into the brain of subjects with Parkinson's disease.

Detailed description

The goal of this research study is to test a new treatment for Parkinson's disease. Parkinson's disease is a progressive disease that causes people to lose specific brain cells called midbrain dopamine neurons. When these dopamine neurons are lost, it leads to a lack of dopamine in the brain. When there is not enough dopamine, people with Parkinson's disease experience problems with their movement. This trial will test whether new dopamine neurons made from blood cells from subjects with Parkinson's disease are safe when surgically injected into the area of the brain affected (called the putamen) of the same subjects (called autologous transplantation). The trial will assess the safety of the injected cells and will also measure the effects of the transplanted autologous dopamine neurons on Parkinson's disease symptoms. At this time, autologous iPSC-derived midbrain dopamine neurons are available only through participation in the ongoing Phase 1/2a clinical trial. Expanded access or compassionate use outside of the trial is not available.

Conditions

• Parkinson Disease

Interventions

Type	Name	Description
BIOLOGICAL	Autologous midbrain dopamine neurons	The autologous midbrain dopamine neurons are a experimental cryopreserved cell product derived from human autologous induced pluripotent stem cells. The autologous midbrain dopamine neurons will be surgically administered into the putamen, unilaterally, in a single surgical session.

Timeline

• Start date: 2024-08-07
• Primary completion: 2026-12-01
• Completion: 2027-12-01
• First posted: 2024-05-20
• Last updated: 2025-09-11

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study