Dopason is a phase I, open-label, multicenter, single-arm clinical trial designed to evaluate the safety and feasibility of intraputaminal transplantation of human embryonic stem cell-derived dopaminergic progenitor cells (DopaCells) in patients with moderately severe Parkinson's disease.
During this trial phase, a total of six participants will be included and distributed between two clinical centers. Each participant will undergo one standardized surgical procedure consisting of bilateral stereotactic intraputaminal transplantation of approximately 10 × 10⁶ cells in total (about 5 × 10⁶ cells per putamen), aiming to achieve an estimated survival of 200,000 neurons. All procedures will be performed using the same surgical delivery system and an almost identical operative protocol across both sites to ensure procedural consistency. After transplantation, patients will receive immunosuppressive therapy for a duration of one year and will be systematically followed for study outcomes for at least 12 months.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
6
DopaCells are dopamine-producing progenitor cells generated from human embryonic stem cells through differentiation under GMP-compliant conditions.
Basiliximab, Tacrolimus, Prednisolone
For intraputaminal delivery of DopaCell
Shiraz University of Medical Sciences
Shiraz, Iran
RECRUITINGIran University of Medical Sciences
Tehran, Iran
RECRUITINGRoyan Institute
Tehran, Iran
RECRUITINGSafety
Incidence and severity of treatment-emergent adverse events (TEAEs)
Time frame: Baseline to 12 Months Post-Transplant
Safety
Evidence of any post-transplantation anatomical changes at the transplant site suggestive of tumor formation, as assessed by MRI imaging.
Time frame: Baseline to 12 Months Post-Transplant
Feasibility
Successful completion of a single-session intraputaminal transplantation with of the intended cell dose
Time frame: Baseline to 12 Months Post-Transplant
Efficacy
Changes in the duration of OFF and ON periods following intracerebral DopaCells transplantation as assessed by the Hauser diary
Time frame: Baseline to 12 Months Post-Transplant
Efficacy
Changes in the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III score from baseline following intracerebral DopaCells transplantation during ON and OFF periods ( MDS-UPDRS Part III ranges from 0 to 132, where higher scores indicate worse motor impairment.)
Time frame: Baseline to 12 Months Post-Transplant
Efficacy
Graft survival following intracerebral DopaCells transplantation based on TRODAT SPECT imaging findings.
Time frame: Baseline to 12 Months Post-Transplant
Continuous Safety
Continuous Safety: The number and severity of treatment-emergent adverse events
Time frame: Baseline to 60 Months Post-Transplant
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