A Phase 1 interventional study of Autologous midbrain dopamine neurons in Parkinson Disease, sponsored by Penelope J. Hallett, Ph.D.. Enrolling by invitation at 1 site in United States. Open to participants aged 55 Years to 80 Years. Per ClinicalTrials.gov, last updated 2026-09-15.
Sponsored by Penelope J. Hallett, Ph.D. · Phase 1, Interventional, and Treatment
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.
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.
4,487 studies on the registry are indexed under Parkinson Disease; 1,082 are open to participants now.
This study's planned enrollment of 12 is below the median of 40 across 3,294 interventional studies indexed under Parkinson Disease.
Browse Parkinson Disease studies →This is the only study on the registry with Penelope J. Hallett, Ph.D. as lead sponsor.
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Exclusion Criteria:
Subjects with active cardiovascular and cerebrovascular disease within 6 months prior to signing the informed consent form:
Biological: Autologous midbrain dopamine neurons
The autologous midbrain dopamine neurons are an experimental cryopreserved cell product derived from human autologous induced pluripotent stem cells. The autologous midbrain dopamine neurons are surgically administered into the putamen. Six participants received unilateral transplantation into one putamen. Six participants will receive bilateral transplantation into both putamina during a single surgical session.
Safety: number and severity of adverse events and serious adverse events
To assess the safety of autologous transplantation of cryopreserved midbrain dopamine neurons into the putamen of subjects with Parkinson's disease by measuring (1) the incidence of serious adverse events at 12 months and 18 months post-transplantation and (2) the incidence and severity of all intervention emergent adverse events.
Time frame: Baseline to 12 months post-transplant and baseline to 18 months post-transplant
Change in UPDRS Part III
Change in Movement Disorder Society Unified Parkinson's Disease Rating Scale (UPDRS) motor (Part III) compared to the baseline. UPDRS Part III assesses motor function and is measured in both "Off" and "On" states. UPDRS Part III score range: 0-132. A lower score is associated with milder Parkinson's disease motor symptoms.
Time frame: 18 months following transplantation
Change in ON time without troublesome dyskinesia
Change in ON time without troublesome dyskinesia is measured using a Parkinson's disease patient diary card.
Time frame: 18 months following transplantation
Change in OFF time
Change in OFF time is measured using a Parkinson's disease patient diary card.
Time frame: 18 months following transplantation
Change in baseline Levodopa Equivalent Daily Dose
Change in Levodopa Equivalent Daily Dose (LEDD) which measures Parkinson's medications.
Time frame: 18 months following transplantation
Change in Unified Dyskinesia Rating Scale
The Unified Dyskinesia Rating Scale evaluates dyskinesia in patients with Parkinson's disease (range 0-104). A lower score indicates less dyskinesia.
Time frame: 18 months following transplantation
Change in UPDRS Part II
Change in Movement Disorder Society Unified Parkinson's Disease Rating Scale (UPDRS) Part II, which assesses the motor aspects of experiences of daily living in the week prior to the visit. Score range: 0-52. A lower score is associated with less disability.
Time frame: 18 months following transplantation
Change in MoCA
Change in Montreal Cognitive Assessment (MoCA), which measures various aspects of cognitive function. Score range 0-30. A higher score is associated with better cognitive function.
Time frame: 18 months following transplantation
Change in DaTscan
DaTscan (SPECT neuroimaging for dopamine transporter, DAT) imaging is performed to assess changes in dopamine neuron function in the putamen (the transplanted area of the brain).
Time frame: Baseline to 18 months following transplantation
Plan to share: No
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