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

NCT06477744 | Active Not Recruiting

• 1 other identifier: SB-PD-002
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

1. 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. 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. 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.

Conditions

Parkinson's Disease

Outcome Measures

Primary Outcomes (1)

• Adverse Event of Special Interests (AESIs)

  Review occurrence of adverse event of special interests (AESIs)

  5 years after IP administration

Secondary Outcomes (3)

• MDS-UPDRS Total Score (defined On/Off)

  -Day 14 to -Day 4, 5 years after IP administration

• MDS-UPDRS part Ⅲ (defined On/Off)

  -Day 14 to -Day 4, 5 years after IP administration

• MDS-UPDRS Ⅳ score

  -Day 14 to -Day 4, 5 years after IP administration

Study Arms (2)

Low Dose Group

EXPERIMENTAL

1. IP Name : Allogenic embryonic stem cell-derived A9 dopamine progenitor cell (A9-DPC) 2. Study group : 6 subjects 3. Dosage: 3.15X10\^6 cells/body (6 tracks in total, 52.5X10\^4 cells per track)

Biological: Allogenic embryonic stem cellderived A9 dopamine progenitor cell (A9-DPC)_Low Dose

High Dose Group

EXPERIMENTAL

1. IP Name : Allogenic embryonic stem cell-derived A9 dopamine progenitor cell (A9-DPC) 2. Study group : 6 subjects 3. Dosage: 6.30X10\^6 cells/body (6 tracks in total, 105X10\^4 cells per track)

Biological: Allogenic embryonic stem cellderived A9 dopamine progenitor cell (A9-DPC)_High Dose

Interventions

Allogenic embryonic stem cellderived A9 dopamine progenitor cell (A9-DPC)_Low Dose BIOLOGICAL

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.

Low Dose Group

Allogenic embryonic stem cellderived A9 dopamine progenitor cell (A9-DPC)_High Dose BIOLOGICAL

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.

High Dose Group

Eligibility Criteria

• Age: 50 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• Not Applicable

Sponsors & Collaborators

• S.Biomedics Co., Ltd.: lead
• Severance Hospital: collaborator

Study Sites (1)

Yonsei Universitiy Health System, Severance Hospital

Seoul, 03722, South Korea

Location

Related Publications (9)

• 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.

  PMID: 15872020 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.

  PMID: 32402162 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.

  PMID: 29113472 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.

  PMID: 28858313 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.

  PMID: 19025984 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)]

  BACKGROUND

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Study Officials

• Phil-hyu Lee, M.D.,Ph.D

  Yonsei Universitiy Health System, Severance Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 12, 2024
• First Posted: June 27, 2024
• Study Start: September 14, 2023
• Primary Completion (Estimated): February 7, 2029
• Study Completion (Estimated): June 29, 2029
• Last Updated: July 26, 2024

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will not share

Locations

KR (1)