Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Severance Hospital | OTHER |
Not provided
Not provided
Not provided
Not provided
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.
Population of the Long-term Follow-up : Subjects who have participated in SB-PD-001 Study and received A9-DPC (about 12 subjects)
Long-term Follow-up Period ; Approximately 72 months from the date of approval by the Institutional Review Board (IRB)
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)]
[Late-onset AESIs (up to 5 years after the study drug treatment)]
There is no delayed AESIs confirmed so far, and when any additional AESIs are detected in the subsequent follow-up, they will be added.
Drug under Long-term Follow-up ; Allogenic embryonic stem cell-derived A9 dopamine progenitor cell (A9-DPC)
Inclusion Criteria ;
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.
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.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Low Dose Group | Experimental |
|
|
| High Dose Group | Experimental |
|
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Allogenic embryonic stem cellderived A9 dopamine progenitor cell (A9-DPC)_Low Dose | Biological |
|
| Measure | Description | Time Frame |
|---|---|---|
| Adverse Event of Special Interests (AESIs) | Review occurrence of adverse event of special interests (AESIs) | 5 years after IP administration |
| Measure | Description | Time Frame |
|---|---|---|
| MDS-UPDRS Total Score (defined On/Off) | Present descriptive statistics (number of subjects, average, standard deviation, median, minimum, maximum) by each dose group about changes in the MDS-UPDRS Total Scores, up to 96 Weeks (24 months) after IP administration compared to baseline (-Day 14 to -Day 4).
|
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Phil-hyu Lee, M.D.,Ph.D | Yonsei Universitiy Health System, Severance Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Yonsei Universitiy Health System, Severance Hospital | Seoul | 03722 | South Korea |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 15872020 | Background | Mendez I, Sanchez-Pernaute R, Cooper O, Vinuela A, Ferrari D, Bjorklund L, Dagher A, Isacson O. Cell type analysis of functional fetal dopamine cell suspension transplants in the striatum and substantia nigra of patients with Parkinson's disease. Brain. 2005 Jul;128(Pt 7):1498-510. doi: 10.1093/brain/awh510. Epub 2005 May 4. | |
| 32402162 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
| Allogenic embryonic stem cellderived A9 dopamine progenitor cell (A9-DPC)_High Dose | Biological |
|
|
| -Day 14 to -Day 4, 5 years after IP administration |
| MDS-UPDRS part Ⅲ (defined On/Off) | Present descriptive statistics (number of subjects, average, standard deviation, median, minimum, maximum) by each dose group about changes in the MDS-UPDRS part Ⅲ (defined On/Off) up to 96 Weeks (24 months) after IP administration compared to baseline (-Day 14 to -Day 4).
| -Day 14 to -Day 4, 5 years after IP administration |
| MDS-UPDRS Ⅳ score | Present descriptive statistics (number of subjects, average, standard deviation, median, minimum, maximum) by each dose group about changes in the MDS-UPDRS Ⅳ score up to 96 Weeks (24 months) after IP administration compared to baseline (-Day 14 to -Day 4).
| -Day 14 to -Day 4, 5 years after IP administration |
| Schweitzer JS, Song B, Herrington TM, Park TY, Lee N, Ko S, Jeon J, Cha Y, Kim K, Li Q, Henchcliffe C, Kaplitt M, Neff C, Rapalino O, Seo H, Lee IH, Kim J, Kim T, Petsko GA, Ritz J, Cohen BM, Kong SW, Leblanc P, Carter BS, Kim KS. Personalized iPSC-Derived Dopamine Progenitor Cells for Parkinson's Disease. N Engl J Med. 2020 May 14;382(20):1926-1932. doi: 10.1056/NEJMoa1915872. |
| 29113472 | Background | Yasuhara T, Kameda M, Sasaki T, Tajiri N, Date I. Cell Therapy for Parkinson's Disease. Cell Transplant. 2017 Sep;26(9):1551-1559. doi: 10.1177/0963689717735411. |
| 28858313 | Background | Kikuchi T, Morizane A, Doi D, Magotani H, Onoe H, Hayashi T, Mizuma H, Takara S, Takahashi R, Inoue H, Morita S, Yamamoto M, Okita K, Nakagawa M, Parmar M, Takahashi J. Human iPS cell-derived dopaminergic neurons function in a primate Parkinson's disease model. Nature. 2017 Aug 30;548(7669):592-596. doi: 10.1038/nature23664. |
| 19025984 | Background | Goetz CG, Tilley BC, Shaftman SR, Stebbins GT, Fahn S, Martinez-Martin P, Poewe W, Sampaio C, Stern MB, Dodel R, Dubois B, Holloway R, Jankovic J, Kulisevsky J, Lang AE, Lees A, Leurgans S, LeWitt PA, Nyenhuis D, Olanow CW, Rascol O, Schrag A, Teresi JA, van Hilten JJ, LaPelle N; Movement Disorder Society UPDRS Revision Task Force. Movement Disorder Society-sponsored revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): scale presentation and clinimetric testing results. Mov Disord. 2008 Nov 15;23(15):2129-70. doi: 10.1002/mds.22340. |
| Background | National Institute of Food and Drug Safety, Ministry of Food and Drug Safety, Guideline for Long-term Follow-up of Advanced Biopharmaceuticals 2020.12 |
| Background | Korea National Institute for Bioethics Policy, Instruction for Protection of Vulnerable Subjects 2019.7 |
| Background | MDS-UPDRS Korean Official Translation. Last updated May 18, 2020. |
| Background | NCI-CTCAE v5.0 [Common Terminology Criteria for Adverse Events (CTCAE) (cancer.gov)] |
| ID | Term |
|---|---|
| D010300 | Parkinson Disease |
| ID | Term |
|---|---|
| D020734 | Parkinsonian Disorders |
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D009069 | Movement Disorders |
| D000080874 | Synucleinopathies |
| D019636 | Neurodegenerative Diseases |
Not provided
Not provided