Safety and Feasibility of Bilateral Striatal Transplantation of DopaCell in Parkinson's Disease

NCT07572071 | Recruiting Phase 1 DMC

• 1 other identifier: 402000063
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Parkinson Disease (PD)

Keywords

Parkinson's disease; Dopaminergic progenitor cell transplantation; Intrastriatal

Outcome Measures

Primary Outcomes (3)

• Safety

  Incidence and severity of treatment-emergent adverse events (TEAEs)

  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.

  Baseline to 12 Months Post-Transplant

• Feasibility

  Successful completion of a single-session intraputaminal transplantation with of the intended cell dose

  Baseline to 12 Months Post-Transplant

Secondary Outcomes (4)

• Efficacy

  Baseline to 12 Months Post-Transplant

• Efficacy

  Baseline to 12 Months Post-Transplant

• Efficacy

  Baseline to 12 Months Post-Transplant

• Continuous Safety

  Baseline to 60 Months Post-Transplant

Study Arms (1)

Transplantation

EXPERIMENTAL

Experimental: DopaCell Biological: DopaCell, 5 million cells per putamen Immunosuppressive Regimen: Basiliximab, Tacrolimus, Prednisolone Device: Customized microinjection device

Biological: Dopacell; Drug: Immunosuppressive Regimen; Device: Customized microinjection device

Interventions

Dopacell BIOLOGICAL

DopaCells are dopamine-producing progenitor cells generated from human embryonic stem cells through differentiation under GMP-compliant conditions.

Transplantation

Immunosuppressive Regimen DRUG

Basiliximab, Tacrolimus, Prednisolone

Transplantation

Customized microinjection device DEVICE

For intraputaminal delivery of DopaCell

Transplantation

Eligibility Criteria

• Age: 30 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age: 30-70 years
• Diagnosis of PD: MDS clinical Diagnostic Criteria for Parkinson's disease
• The disease duration more than 5 years
• Moderate Parkinson's disease, defined as a Hoehn and Yahr stage of 2 or 3 during the OFF period.
• The patient is receiving oral pharmacological therapy, and in the opinion of the Principal Investigator, the patient's symptoms remain inadequately controlled despite optimal medical management, or the patient is experiencing adverse effects related to their current treatment
• No history or only mild levodopa-induced dyskinesia, defined as a score of 2 or less on the UDysRS scale in any body region during the ON state.
• The patient demonstrates a clinically meaningful response to a therapeutic dose of levodopa, as determined by the Principal Clinical Investigator or a trained specialist under the supervision of the Principal Investigator.
• The performance of different organs based on laboratory evaluations:
• Number of neutrophils ≥2000 / microliter
• Platelet count ≥100,000 / microliter
• AST / ALT: less than or equal to three times the maximum normal value at the intervention site
• Total bilirubin less than or equal to 1.5 times the maximum normal amount at the intervention site
• eGFR \* rate: greater than or equal to 60 ml / min / 1.73 m2 \* eGFR (mL / min / 1.73 m2) = 194 X Cr \^ -1.094 X age \^ -0.287 (X 0.739 for females)
• Informed consent

You may not qualify if:

• The abnormal function of immune system
• The symptomatic brain injuries (brain atrophy, cerebral Infarct, trauma, vascular malformation) confirmed by brain MRI
• Markedly reduced or normal signal in the ventral striatum on TRO-DaT SPECT imaging.
• Any abnormal findings on brain MRI.
• Positive GBA mutation test.
• Diagnosis of dementia based on a MoCA score \< 24.
• The abnormality of thrombotic system or high risk of bleeding
• Positive for any of the following viral markers or active infections: HBsAg, HBsAb, HBcAb, anti-HIV antibodies, anti-HTLV-1\&2 antibodies, active hepatitis C infection, syphilis, or active CMV, VZV, EBV, or COVID-19 infection.
• Impossibility of MRI imaging for patients with metal in the body, pacemaker in the body, claustrophobia, with artificial heart valves that are incompatible with MRI or body weight is not within the tolerable range for MRI.
• Patients with contraindications to the study drug: Tacrolimus, Prednisolone, Basiliximab, Cotrimoxazole, MRI contrast agent.
• Patients undergoing other cell transplants, including embryonic stem cell-derived dopaminergic progenitor cells.
• Patients with a history of PD at the same time and concurrent: Malignant neoplasm, epilepsy, cerebral hemorrhage or a positive history
• Psychiatric disorders confirmed by a psychiatrist, including major depression, bipolar disorder, or schizophrenia, that are uncontrolled or treatment resistant.
• Patients with intellectual disability who, in the judgment of a psychiatrist, are unable to fully comprehend the study requirements.
• History of pallidotomy, thalamotomy, or deep brain stimulation (DBS).

Sponsors & Collaborators

• Royan Institute: lead
• Royan AtiTech Pharmed: collaborator
• Shiraz University of Medical Sciences: collaborator
• Iran University of Medical Sciences: collaborator

Study Sites (1)

Royan Institute

Tehran, Iran

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

Sarvenaz Salahi, MD

CONTACT

021-23562000; salahi13639@gmail.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 7, 2026
• First Posted: May 7, 2026
• Study Start: August 1, 2025
• Primary Completion (Estimated): August 1, 2028
• Study Completion (Estimated): August 1, 2030
• Last Updated: May 7, 2026

Record last verified: 2026-04

Locations

IR (1)