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