Assess efficacy of intra-arterial autologous myogenic stam cell therapy for m.3243A>G mutation carriers
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Assessment of safety parameters (angiography, 8h post-procedure monitoring of neurological vital signs, (S)AE) and assessment of changes in muscle strength and muscle fatigue of treated and untreated biceps brachii muscle using Biodex dynamometer measurements at baseline and 4-6 weeks after the third ATMP administration.
研究概览
简要总结
Assess effect of three intra-arterial administrations of autologous MABs to the BB muscle on of m.3243A>G patients by assessing strength and fatigue in BB muscles of both arms before and after last MABs administration in left arm. Assess safety of the three ATMP administrations to the upper left arm by checking for SAEs, vascular obstructions and changes in neurological vital signs.
入排标准
- 年龄范围
- 18 years 至 64 years（18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •Written informed consent, Age: 18-64, Sex: male/female, Patients with the hetroplasmic m.3243A>G mutation.
排除标准
- •Use of dabigatran, apixaban, edoxaban or rivaroxaban (DOACs) as anti-coagulants; Have a weekly alcohol intake of ≥ 35 units (men) or ≥ 24 units (women); Current history of drug abuse; Deficient immune system or autoimmune disease; Significant concurrent illness; Ongoing participation in other clinical trials with intervention; Pregnant or lactating women; Psychiatric or other disorders likely to impact on informed consent; Patients unable and/or unwilling to comply with treatment and study instructions - A history of strokes with signs of extra-pyramidal or pyramidal syndrome - Allergy for contrast fluid - Peripheral signs of ischemia or vasculopathy; Any other factor that in the opinion of the investigator excludes the patient from the study
结局指标
主要结局
Assessment of safety parameters (angiography, 8h post-procedure monitoring of neurological vital signs, (S)AE) and assessment of changes in muscle strength and muscle fatigue of treated and untreated biceps brachii muscle using Biodex dynamometer measurements at baseline and 4-6 weeks after the third ATMP administration.
Assessment of safety parameters (angiography, 8h post-procedure monitoring of neurological vital signs, (S)AE) and assessment of changes in muscle strength and muscle fatigue of treated and untreated biceps brachii muscle using Biodex dynamometer measurements at baseline and 4-6 weeks after the third ATMP administration.
次要结局
- Assess muscle mass, morphology, m.3243A>G mutation load and mitochondrial respiratory capacity in muscle biopsies of treated BB muscle
研究者
Florence van Tienen
Scientific
Academisch Ziekenhuis Maastricht