General Information |
| Summary |
Indication: Patients who were diagnosed with Parkinson's disease ≥ 5 years ago.
Purpose: To find the maximum tolerable dose and evaluate the safety and exploratory efficacy of allogenic embryonic stem cell-derived A9 dopamine progenitor cell (A9-DPC) therapy in patients who were diagnosed with Parkinson's disease ≥ 5 years ago, as a treatment for delaying or stopping the progression of Parkinson's disease or inducing recovery of damaged brain.
Number of Subjects: Up to 12 subjects. [Low dose] 3.15X10^6 cells/body: 6 subjects. [High dose] 6.30X10^6 cells/body: 6 subjects.
Study Design: Single center, open, single dosing, dose-escalation, phase 1/2a study
Endpoints:
[Primary Safety Endpoints]
Occurrence of treatment-emergent adverse events (TEAEs) after administration of the IP
Failure or rejection of transplantation and occurrence of bleeding and infection at Week 12 (3months), Week 24 (6months), Week 48 (12months) and Week 96 (24months) after administration of the IP
Occurrence of adverse event of special interest (AESI)* after administration of the IP
AESI: a) death, b) generation of a neoplasm or malignant tumor in tissues or organs, c) onset of an immune reaction including worsening of a previous autoimmune disease or new occurrence, and d) other delayed adverse events related to this embryonic stem cell treatment. |
| Description |
Study Period: Approximately 35 months from the date of approval by the Institutional Review Board (IRB) (However, it can be extended depending on the subject enrollment period or the time to study closure)
Indication: Patients who were diagnosed with Parkinson's disease ≥ 5 years ago
Purpose: To find the maximum tolerable dose and evaluate the safety and exploratory efficacy of allogenic embryonic stem cell-derived A9 dopamine progenitor cell (A9-DPC) therapy in patients who were diagnosed with Parkinson's disease ≥ 5 years ago, as a treatment for delaying or stopping the progression of Parkinson's disease or inducing recovery of damaged brain.
Number of Subjects: Up to 12 subjects [Low dose] Dose: 3.15X10^6 cells/body Study group(A9-DPC): 6 subjects [High dose] Dose: 6.30X10^6 cells/body Study group(A9-DPC): 6 subjects
Study Design: Single center, open, single dosing, dose-escalation, phase 1/2a study
Endpoints:
[Primary Safety Endpoints]
Occurrence of treatment-emergent adverse events (TEAEs) after administration of the IP
Failure or rejection of transplantation and occurrence of bleeding and infection at Week 12 (3 months), Week 24 (6 months), Week 48 (12 months) and Week 96 (24 months) after administration of the IP
Occurrence of adverse event of special interest (AESI)* after administration of the IP *AESI: a) death, b) generation of a neoplasm or malignant tumor in tissues or organs, c) onset of an immune reaction including worsening of a previous autoimmune disease or new occurrence, and d) other delayed adverse events related to this embryonic stem cell treatment.
[Exploratory Efficacy Endpoints]
1. Change in the following clinical endpoints at Week 4 (1 month), Week 12 (3 months), Week 24 (6 months), Week 36 (9 months), Week 48 (12 months), Week 72 (18 months) and Week 96 (24 months) after administration of the IP compared to screening
MDS-UPDRS Total Score, part Ⅲ & part Ⅳ (defined on/off)
K-MMSE
Seoul Neuropsychological screening battery (SNSB, Screening & Week 96 (24 months))
Hoehn & Yahr scale
2. Change in the following clinical endpoints at Week 4 (1 month), Week 12 (3 months), Week 24 (6 months), Week 36 (9 months), Week 48 (12 months), Week 72 (18 months) and Week 96 (24 months) after administration of the IP compared to baseline
K-MoCA
Parkinson's Questionnaire (PDQ-39)
Schwab and England ADL scale (SEADL) 4Non-Motor Symptoms Scale for Parkinson's Disease (NMS)
3. Change in Graft size through MRI at Week 12 (3 months), Week 24 (6 months), Week 48 (12 months) and Week 96 (24 months) after administration of the IP compared to baseline 4. Change in Cerebral FDG uptake and Striatal FDG uptake at Week 48 (12 months) and Week 96 (24 months) after administration of the IP compared to baseline 5. Change in density of dopamine transporters as measured by FP-CIT PET at Week 48 (12 months) and Week 96 (24 months) after administration of the IP compared to screening 6. Percentage of subjects who used concomitant medication related to Parkinson-mobility or Parkinson-Non-mobility during the whole clinical trial period and change in dose of each concomitant medication (per component) at 12 week intervals compared to the dose of each concomitant medication (per component) from the date of administration of IP to Week 12.
7. Changes in the following clinical evaluation variables confirmed through the Parkinson's Disease diary at 48 weeks (12 months), 72 weeks (18 months), and 96 weeks (24 months) after administration of the investigational drug compared to baseline:
Total waking time
Total on-time
Total off-time
Total dyskinesia time
[Other Safety Endpoints]
Vital signs
Laboratory tests
Physical examination |
| Clinical trials phase |
Phases 1/2 |
| Start date (estimated) |
2023-05-09 |
| End date (estimated) |
2026-02-07 |
| Clinical feature |
| Label |
Parkinson's disease |
| Link |
http://purl.obolibrary.org/obo/DOID_14330 |
| Description |
A synucleinopathy that has_material_basis_in degeneration of the central nervous system that often impairs motor skills, speech, and other functions.; Xref MGI.
OMIM mapping confirmed by DO. [SN]. |
|
Administrative Information |
| NCT number |
NCT05887466 |
| ICTRP weblink |
https://trialsearch.who.int/Trial2.aspx?TrialID=NCT05887466 |
| Other study identifiers |
| Name |
KCT0009343 |
| Description |
Clinical Research Information Service (CRIS) of Republic of Korea |
|
| Source weblink |
https://clinicaltrials.gov/ct2/show/NCT05887466 |
| Public contact |
| Email |
dwkim2@yuhs.ac |
| Public email |
dwkim2@yuhs.ac |
| First name |
Dong-Wook |
| Last name |
Kim |
| Phone |
+82 10-7122-5208 |
| Street |
50 Yonsei-ro |
| Postcode |
03722 |
| City |
Seoul |
| Country |
|
|
| Sponsors |
S.Biomedics
|
| Collaborators |
|
Cells |
| Which differentiated cell type is used |
| Label |
dopaminergic neuron |
| Link |
http://purl.obolibrary.org/obo/CL_0000700 |
| Description |
A neuron that releases dopamine as a neurotransmitter. |
|
Recruitment |
| Recruitment Status |
Active, not recruiting |
