NCT06477744

Long Term Follow-up of Patients With Parkinson's Disease Who Had Administered of A9-DPC in SB-PD-001 Study

Long Term Follow-up of Patients With Parkinson's Disease Who Had Administered of Allogenic Embryonic Stem Cell-derived A9 Dopamine Progenitor Cell (A9-DPC) in SB-PD-001 Study

• Status: Active Not Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: S.Biomedics Co., Ltd. · Industry
• Sex: All
• Age: 50 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

1. Long-term follow-up period: Approximately 72 months from the date of approval by the Institutional Review Board (IRB)( Study Period: From the A9-DPC treatment date of the first subject in SB-PD-001 Study\* up to 5 years after the A9-DPC treatment of the last subject ) 2. Objectives: This study aims to evaluate the long-term safety of A9-DPC by following up on the occurrence of adverse event of special interest, (AESI)\* for 5 years from A9-DPC treatment date in subjects who have participated in SB-PD-001 Study and received A9-DPC. In addition, it will determine motor and non-motor symptoms over time following A9-DPC treatment, as measured by MDS-UPDRS. 3. Methods of the Long-term Follow-up : This is a single center, open-label, 5-year long-term follow-up to evaluate the long-term safety in subjects receiving A9-DPC in SB-PD-001 Study. Among subjects who have received A9-DPC, those who have provided voluntary written informed consent for participation of the long-term follow-up will be included. The occurrence of AESIs is investigated for 5 years from A9-DPC treatment, and MDS-UPDRS will be conducted for the efficacy assessment if the study can be conducted. To avoid any missing data about AESIs, a phone or site visit will be performed at least once yearly from the start of the follow-up.

Detailed description

1. Population of the Long-term Follow-up : Subjects who have participated in SB-PD-001 Study and received A9-DPC (about 12 subjects) 2. Long-term Follow-up Period ; Approximately 72 months from the date of approval by the Institutional Review Board (IRB) * Study Period: From the A9-DPC treatment date of the first subject in SB-PD-001 Study\* up to 5 years after the A9-DPC treatment of the last subject * Study Duration for Individual Subject: 5 years from A9-DPC treatment date \* SB-PD-001 Study: Phase 1/2a study of A9-DPC 3. Objectives and Endpoints of the Long-term Follow-up ; This study aims to evaluate the long-term safety of A9-DPC by following up on the occurrence of adverse event of special interest, (AESI)\* for 5 years from A9-DPC treatment date in subjects who have participated in SB-PD-001 Study and received A9-DPC. In addition, it will determine motor and non-motor symptoms over time following A9-DPC treatment, as measured by MDS-UPDRS. \* AESI is a minimum investigation item to be observed for long-term follow-up of stem cell treatment among the adverse events (AEs) that occur in the subjects receiving treatment. In accordance with the Guideline for Long-term Follow-up of Advanced Biopharmaceuticals distributed by the Ministry of Food and Drug Safety in 2020, it is designated as below in this long-term follow-up. AESIs refer to the serious adverse events (SAEs) in the guideline. Adverse Event of Special Interests (AESIs) \[Early-onset AESIs (up to 2 years\* after the study drug treatment)\] * Infectious diseases * Complications related to the associated surgical procedures \[Late-onset AESIs (up to 5 years after the study drug treatment)\] * Death * Generation of a neoplasm or malignant tumor in tissues or organs * Onset of an immune reaction including worsening of a previous autoimmune disease or new occurrence * Other delayed AESIs related to the treatment of embryonic stem cell-derived therapeutics. There is no delayed AESIs confirmed so far, and when any additional AESIs are detected in the subsequent follow-up, they will be added. 4. Drug under Long-term Follow-up ; Allogenic embryonic stem cell-derived A9 dopamine progenitor cell (A9-DPC) 5. Inclusion Criteria ; * Persons who have participated in SB-PD-001 Study and received A9-DPC * Persons who have provided written informed consent for this long-term follow-up 6. Methods of the Long-term Follow-up ; This is a single center, open-label, 5-year long-term follow-up to evaluate the long-term safety in subjects receiving A9-DPC in SB-PD-001 Study. Among subjects who have received A9-DPC, those who have provided voluntary written informed consent for participation of the long-term follow-up will be included. The occurrence of AESIs is investigated for 5 years from A9-DPC treatment, and MDS-UPDRS will be conducted for the efficacy assessment if the study can be conducted. To avoid any missing data about AESIs, a phone or site visit will be performed at least once yearly from the start of the follow-up. 7. Analysis Methods ; Adverse Event of Special Interests (AESIs) For AESIs, the number of subjects affected, incidence rate, its exact 95% confidence interval (CI), and the number of events will be provided. In addition, the events will be coded using the Medical Dictionary For Regulatory Activities (MedDRA) with System Organ Class (SOC) and Preferred Term (PT), and the number of subjects affected, incidence rate, and the number of events will be provided. MDS-UPDRS Total Score(defined On/Off), part Ⅲ (defined on/off) and IV score For changes at each time point after the IP treatment from baseline, descriptive statistics (number of subjects, mean, standard deviation, median, minimum, and maximum) will be provided.

Conditions

• Parkinson's Disease

Interventions

Type	Name	Description
BIOLOGICAL	Allogenic embryonic stem cellderived 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 cellderived 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.

Timeline

• Start date: 2023-09-14
• Primary completion: 2029-02-07
• Completion: 2029-06-29
• First posted: 2024-06-27
• Last updated: 2024-07-26

Locations

1 site across 1 country: South Korea