SAFE-iPS - France's First Hospital-based Platform for the Production of Induced Pluripotent Stem Cells for the Regenerative Medicine of the Future
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Successful generation of functional induced pluripotent stem cell (iPSC) lines
研究概览
简要总结
The objective of the study is to establish the first French hospital-based Good Manufacturing Practice (GMP)-compliant platform for the production of clinical-grade induced pluripotent stem cells (iPSCs) for future regenerative medicine applications.
The primary objective is to generate, via the SAFE-iPS platform, 4 clinical-grade iPSC lines derived from peripheral blood samples taken from:
- Two healthy male volunteers
- Two healthy female volunteers
These 4 iPSC lines will undergo comprehensive quality assessment including:
- Expression of pluripotency markers
- Genomic stability assessment
- Absence of residual Sendai viral integration
- Technical reproducibility assessment
- Manufacturing efficiency assessment
- Preliminary medico-economic evaluation This project aims to demonstrate that the SAFE-iPS manufacturing process can be successfully transferred to a hospital Good Manufacturing Practice (GMP) environment and can reproducibly generate clinical-grade iPSC lines meeting international quality standards for future regenerative medicine applications. It also aims to establish the first French public hospital platform dedicated to routine GMP production of clinical-grade iPSCs.
详细描述
Background :
Induced pluripotent stem cells (iPSCs) represent a major advance in regenerative medicine because they can be generated from easily accessible autologous somatic cells and differentiated into virtually any cell type. Their potential clinical applications include hematology, neurology, rare diseases, oncology, and regenerative medicine. However, translation of this technology into clinical practice requires Good Manufacturing Practice (GMP)-compliant production processes and stringent quality standards for Advanced Therapy Medicinal Products (ATMPs).
Currently, no French public hospital has a validated and routinely operational GMP platform dedicated to the production of clinical-grade iPSC lines. The SAFE-iPS platform at Montpellier University Hospital combines dedicated clean-room facilities, an ISO 13485-certified quality management system, and more than ten years of expertise in iPSC production and characterization, providing the necessary infrastructure to establish such a platform.
The central hypothesis of the study is that peripheral blood mononuclear cell (PBMC) reprogramming using a non-integrating Sendai viral vector expressing OCT4, KLF4, SOX2 and c-MYC (OKSM) can be successfully implemented in a hospital GMP environment and reproducibly generate clinical-grade iPSC lines fulfilling international quality requirements for future clinical applications.
Objectives :
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Basic Science
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 35 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Age ≥ 18 years and ≤ 35 years
- •No medical contraindication to a 54 mL blood sample collection
- •Negative viral serology for:
- •Human Immunodeficiency Virus (HIV)
- •Hepatitis B Virus (HBV)
- •Hepatitis C Virus (HCV)
- •Human T-lymphotropic Virus type 1 (HTLV-1)
- •A participant who agrees to have their anonymized biological samples and cell lines stored in a biological sample collection for other research purposes (including genetic research)
排除标准
- •Any active medical condition
- •Any ongoingl treatment
- •Subjects who cannot read and/or write
- •Inability to ensure participant during the study period
- •Participation in another clinical trial or administration of an off-label medication that could interfere with blood test results.
- •Participation in another research protocol involving biological sampling that limits the number of mL to be collected.
- •Failure to obtain informed consent
- •Not enrolled in a social security program,
- •Subject participating in another research study with an ongoing exclusion period
- •Protected populations under the French Public Health Code (women who are giving birth, breastfeeding, or pregnant; subjects deprived of liberty by judicial or administrative decision; adults under legal protection (under any form of guardianship))
结局指标
主要结局
Successful generation of functional induced pluripotent stem cell (iPSC) lines
时间窗: upon completion of reprogramming and full validation, within ≤ 60 days post-transfection
The primary outcome of the study is the successful production of four (4) functional human induced pluripotent stem cell (iPSC) lines, comprising two (2) male-derived and two (2) female-derived lines, validated according to international pluripotency standards
次要结局
- Process Reproducibility(Month 3)
- Quality of induced pluripotent stem cel (iPSC) lines(Day 21 to Day 30 post-reprogramming)
- Genomic Safety(Day 45 to Day 60 post-reprogramming)
- Operational Efficiency(Through production phase, less than 30 days)
- Economic Evaluation(Month 3)