Study to Evaluate the Safety and Efficacy of ESC-derived Dopamine Progenitor Cell Therapy in PD Patients

Summary

Indication: Patients who were diagnosed with Parkinson's disease ≥ 5 years ago.

Purpose: To find the maximum tolerable dose and evaluate the safety and exploratory efficacy of allogenic embryonic stem cell-derived A9 dopamine progenitor cell (A9-DPC) therapy in patients who were diagnosed with Parkinson's disease ≥ 5 years ago, as a treatment for delaying or stopping the progression of Parkinson's disease or inducing recovery of damaged brain.

Number of Subjects: Up to 12 subjects. \[Low dose\] 3.15X10\^6 cells/body: 6 subjects. \[High dose\] 6.30X10\^6 cells/body: 6 subjects.

Study Design: Single center, open, single dosing, dose-escalation, phase 1/2a study

Endpoints:

\[Primary Safety Endpoints\]

1. Occurrence of treatment-emergent adverse events (TEAEs) after administration of the IP
2. Failure or rejection of transplantation and occurrence of bleeding and infection at Week 12 (3months), Week 24 (6months), Week 48 (12months) and Week 96 (24months) after administration of the IP
3. Occurrence of adverse event of special interest (AESI)\* after administration of the IP

* AESI: a) death, b) generation of a neoplasm or malignant tumor in tissues or organs, c) onset of an immune reaction including worsening of a previous autoimmune disease or new occurrence, and d) other delayed adverse events related to this embryonic stem cell treatment.

Conditions

• Parkinson's Disease

Interventions

BIOLOGICAL

Allogenic embryonic stem cell-derived A9 dopamine progenitor cell (A9-DPC) Low Dose

Biological/Vaccine: Allogenic embryonic stem cell-derived A9 dopamine progenitor cell (A9-DPC)\_Low Dose 1. IP Name : Allogenic embryonic stem cell-derived A9 dopamine progenitor cell (A9-DPC) 2. Main ingredients and quantities: A9-DPC -Low Dose : 7.0X10\^6 cells (Use 3.15X10\^6 cells of this) 3. Formulation: milky white cell suspension 4. Storage method: Refrigerated storage (5±3#) 5. Expiration date: within 36 hours of manufacture 6. Frequency: single dosing 7. Method: The subject pierces a burr hole in the skull on the day of surgery. Then the cells are injected at a predetermined stem cell administration point of the putamen in the brain of the subject. One side is administered in three tracks per putamen (6 tracks total brain). Do the same for the other side.

BIOLOGICAL

Allogenic embryonic stem cell-derived A9 dopamine progenitor cell (A9-DPC) High Dose

Biological/Vaccine: Allogenic embryonic stem cell-derived A9 dopamine progenitor cell (A9-DPC)\_High Dose 1. IP Name : Allogenic embryonic stem cell-derived A9 dopamine progenitor cell (A9-DPC) 2. Main ingredients and quantities: A9-DPC -High Dose : 1.4X10\^7 cells (Use 6.30X10\^6 cells of this) 3. Formulation: milky white cell suspension 4. Storage method: Refrigerated storage (5±3#) 5. Expiration date: within 36 hours of manufacture 6. Frequency: single dosing 7. Method: The subject pierces a burr hole in the skull on the day of surgery. Then the cells are injected at a predetermined stem cell administration point of the putamen in the brain of the subject. One side is administered in three tracks per putamen (6 tracks total brain). Do the same for the other side.

Sponsors & Collaborators

• Yonsei University

  collaborator OTHER

• S.Biomedics Co., Ltd.

  lead INDUSTRY

Principal Investigators

• Phil-hyu Lee, MD, Ph.D · Yonsei Universitiy Health System, Severance Hospital

Study Design

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

SEQUENTIAL

Eligibility

Min Age

50 Years

Max Age

75 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2023-05-09

Primary Completion

2025-12-10

Completion

2025-12-10

Countries

• South Korea

Study Locations