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Parkinson's DiseaseNCT06477744

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

A study testing Allogenic embryonic stem cellderived A9 dopamine progenitor cell (A9-DPC)_Low Dose and Allogenic embryonic stem cellderived A9 dopamine progenitor cell (A9-DPC)_High Dose in people with Parkinson's Disease, run by S.Biomedics Co., Ltd. at 1 site in South Korea.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 50 to 75 years
Conditions the study is for
Parkinson's Disease
What is being tested?
Allogenic embryonic stem cellderived A9 dopamine progenitor cell (A9-DPC)_Low Dose and Allogenic embryonic stem cellderived A9 dopamine progenitor cell (A9-DPC)_High Dose
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
1 siteSouth Korea
Study dates
14 Sep 2023to 29 Jun 2029
Phase
Interventional
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 50 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • Not Applicable

Trial locations and contacts

1 site in 1 country.

South Korea1 site
  • Yonsei Universitiy Health System, Severance Hospital

    Seoul, 03722

Other trials in South Korea

About this 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

  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.

Read the 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
  1. 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.
  1. Drug under Long-term Follow-up ; Allogenic embryonic stem cell-derived A9 dopamine progenitor cell (A9-DPC)
  2. 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
  1. 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.
  2. 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.

Being tested
Allogenic embryonic stem cellderived A9 dopamine progenitor cell (A9-DPC)_Low Dose, Allogenic embryonic stem cellderived A9 dopamine progenitor cell (A9-DPC)_High Dose
Medical subject headings
Parkinson Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Allogenic embryonic stem cellderived A9 dopamine progenitor cell (A9-DPC)_Low DoseBiological

    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.
  • Allogenic embryonic stem cellderived A9 dopamine progenitor cell (A9-DPC)_High DoseBiological

    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.
Study groups in NCT06477744
Study groupRoleWhat they receive
Low Dose GroupExperimental1. 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)
Receives: Biological: Allogenic embryonic stem cellderived A9 dopamine progenitor cell (A9-DPC)_Low Dose
High Dose GroupExperimental1. 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)
Receives: Biological: Allogenic embryonic stem cellderived A9 dopamine progenitor cell (A9-DPC)_High Dose

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Adverse Event of Special Interests (AESIs)

    Review occurrence of adverse event of special interests (AESIs)

    Measured: 5 years after IP administration

Secondary outcomes (3)
  1. 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).

    • Defined-on condition: condition that the most positive functional effect, as agreed by the subject and the tester, after treatment with drugs for controlling the symptoms of Parkinson's disease
    • Defined-off condition: condition after 12 hours off drugs for controlling the symptoms of Parkinson's disease

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

  2. 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).

    • Defined-on condition: condition that the most positive functional effect, as agreed by the subject and the tester, after treatment with drugs for controlling the symptoms of Parkinson's disease
    • Defined-off condition: condition after 12 hours off drugs for controlling the symptoms of Parkinson's disease

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

  3. 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).

    • Defined-on condition: condition that the most positive functional effect, as agreed by the subject and the tester, after treatment with drugs for controlling the symptoms of Parkinson's disease
    • Defined-off condition: condition after 12 hours off drugs for controlling the symptoms of Parkinson's disease

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted27 Jun 2024
  2. Study start14 Sep 2023
  3. Primary completion7 Feb 2029 (estimated)
  4. Study completion29 Jun 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase N/A
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
12 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06477744
Sponsor's protocol ID
SB-PD-002
Status last verified
Jun 2024
First submitted
12 Jun 2024
Last updated
26 Jul 2024
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • 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.

    PubMed 15872020
  • 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.

    PubMed 32402162
  • 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.

    PubMed 29113472
  • 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.

    PubMed 28858313
  • 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.

    PubMed 19025984
  • National Institute of Food and Drug Safety, Ministry of Food and Drug Safety, Guideline for Long-term Follow-up of Advanced Biopharmaceuticals 2020.12

  • Korea National Institute for Bioethics Policy, Instruction for Protection of Vulnerable Subjects 2019.7

  • MDS-UPDRS Korean Official Translation. Last updated May 18, 2020.

  • NCI-CTCAE v5.0 [Common Terminology Criteria for Adverse Events (CTCAE) (cancer.gov)]

Questions about this trial

What is this clinical trial studying?

A study testing Allogenic embryonic stem cellderived A9 dopamine progenitor cell (A9-DPC)_Low Dose and Allogenic embryonic stem cellderived A9 dopamine progenitor cell (A9-DPC)_High Dose in people with Parkinson's Disease, run by S.Biomedics Co., Ltd. at 1 site in South Korea.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 26 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Adverse Event of Special Interests (AESIs) (time frame: 5 years after IP administration). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06477744, which also lists 9 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jul 2024.

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