General Information |
| Summary |
XellSmart Biomedical (Suzhou) Co., Ltd. has closely collaborated with Shanghai East Hospital(East Hospital Affiliated To Tongji University) , using iPSC seed cells, on the development and establishment of a dopaminergic neural precursor cell production system. These iPSC seed cells are sourced from the National Stem Cell Translational Resource Bank in Shanghai East Hospital (East Hospital Affiliated To Tongji University), and the construction of the cell bank is carried out by Shanghai East Hospital. The iPSC seed cell preparation method and testing standards have been certified by National Institutes for Food and Drug Control in China.
This study is conducted under collaboration between Shanghai East Hospital and XellSmart Biomedical (Suzhou) Co., Ltd. It is a distinctive stem cell therapy research targeting Parkinson's disease patients, characterized by the following:
1. The first instance in China of using autologous iPSC-derived subtype-specific dopaminergic neural precursor cells for alternative transplantation therapy in Parkinson's disease.
2. Before the initiation of this study, there was only one reported case around the world involving the use of laboratory-grade autologous iPSC-induced dopaminergic neural precursor cells for Parkinson's disease treatment in an American Caucasian individual. Positive preliminary clinical research results were obtained and published in the New England Journal of Medicine in 2020. Since then, no similar studies have been reported.
It has great significance to conduct this research. It will fill two critical clinical research gaps in stem cell therapy for Parkinson's disease in the world:
1. The use of clinical-grade autologous iPSCs differentiated into dopaminergic neural cells, and treat Parkinson's disease.
2. Evaluating the safety of using autologous iPSC-differentiated dopaminergic neural cells for Parkinson's disease treatment in individuals of Asian descent or Chinese ethnicity. |
| Description |
This study is a non-randomized, open-label, investigator-initiated interventional clinical trial aimed at exploring the safety of stem cell investigational drugs. The study plans to recruit 3 eligible PD subjects and prepare dopaminergic neural precursor cells from autologous iPSCs in advance. The PD subjects will undergo general anesthesia and, with the assistance of stereotactic technology, receive an injection of 4.5 million cells/90 microliters (50,000 cells/microliter) of autologous iPSC-derived dopaminergic neural precursor cells into the Gpi nucleus on one side, transplanted evenly at a linear distance of 1 centimeter. A Sofia drug pouch will also be installed to regularly monitor changes in neurotransmitter metabolism, including dopamine, in the brain.
Following the surgery, there will be regular observations for 6 months, during which the safety of this treatment modality will be examined and analyzed. If the test results within the first 6 months post-surgery indicate both good safety and some level of efficacy, the trial will continue for an additional 6 months. However, if the test results within the first 6 months post-surgery demonstrate good safety (i.e., no adverse events related to stem cell transplantation of grade 3 or higher occurring within 28 days after stereotactic transplantation of dopaminergic neural precursor cells) but insufficient efficacy (evaluated based on subject self-assessment, clinical symptoms, rating scales, PET/MR, changes in the dosage of levodopa, and other medications), another 4.5 million cells/90 microliters (50,000 cells/microliter) of autologous iPSC-derived dopaminergic neural precursor cells will be injected into the Gpi nucleus on the other side (using the same method). Subsequently, the subjects will be regularly observed for an additional 18 months.
Postoperative observation and follow-up for unilateral stem cell transplantation will be conducted for 12 months, while bilateral stem cell transplantation will be observed and followed for 24 months (with the first postoperative observation and follow-up at 6 months and the second at 18 months). During the study, there will be periodic reviews and summaries every 6 months, generating interim clinical reports to optimize and guide the next stage of data collection and research. |
| Clinical trials phase |
Not applicable |
| Start date (estimated) |
2023-11-23 |
| End date (estimated) |
2025-12-22 |
| 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 |
NCT06145711 |
| ICTRP weblink |
https://trialsearch.who.int/Trial2.aspx?TrialID=NCT06145711 |
| Other study identifiers |
|
| Source weblink |
https://clinicaltrials.gov/study/NCT06145711 |
| Study sites |
|
| Public contact |
| Email |
wujingwendongfang@163.com |
| Public email |
wujingwendongfang@163.com |
| First name |
Jingwen |
| Last name |
Wu |
| Phone |
+ 13585517271 |
| City |
Shanghai |
| Country |
|
|
| Sponsors |
Shanghai East Hospital
|
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 |
Not yet recruiting |
| Estimated number of participants |
3 |
