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

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.

Timeline

Start

2023-09-14

Primary completion

2029-02-07

Completion

2029-06-29

Publications

• 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.
• Background 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.
• 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.
• 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.
• 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)]

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