Phase 1/2a First-in-human Trial Evaluating Autologous Gene-edited Muscle Stem Cells in Limb Girdle Muscular Dystrophies (GenPHSats-bASKet)
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 6
- 主要终点
- Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
研究概览
简要总结
This study is an investigator initiated first-in-human interventional open label phase 1/2a clinical trial investigating an ATMP in the orphan disease LGMD to evaluate safety and efficacy.
详细描述
This trial is directed towards a first-in-human application of GenPHSats; gene edited primary human satellite cell derived muscle stem cells as a new Advanced Therapy Medicinal Product (ATMP) in a phase 1/2a clinical trial with Gene edited PHSats (GenPHSats) initiating healthy muscle development in patients with LGDM. The trial is set up to verify if GenPHSats can provide an therapy option for LGDM patients as there is currently no therapy available. The GenPHSats are an autologous product comprised of primary human satellite cell derived muscle stem cells obtained from the patient's own muscle tissue and gene edited in vitro prior to transplantation.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 14 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •LGDM diagnosed,
- •Identified gene defect location and gene editing proved feasible,
- •Age ≥14 years,
- •Patient in treatment in the department at Charité, Universitätsmedizin Berlin, Muscle Research Unit and Outpatient Clinic for Muscle Disorders,
- •Signed informed consent
排除标准
- •Acute or chronic inflammatory local or systemic disease
- •Coagulation disorder
- •Known complications due to local anesthesia,
- •Congenital heart defect, cardiac arrhythmia,
- •Pathology of the airways such as micrognathia
- •Pierre Robin Sequence
- •Central hypoventilation syndrome/Ondine syndrome
- •Significant other medical or psychiatric illness
- •Positive serology for HIV and/or hepatitis A, B, C
- •Pregnant or lactating women
- •Known allergic reaction to constituents of the cryopreservation medium
结局指标
主要结局
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
时间窗: Through study completion, an average of 1 year
Characterization of type, incidence, severity, duration, reversibility, treatability of adverse events recorded at least at visit 1, 2, 3 and 4 (end of trial).
次要结局
- Rate of muscle biopsy post-injection structure(3 month post injection)
研究者
Simone Spuler, MD
Clinical Professor
Charite University, Berlin, Germany