The first primary objective is to assess the effect of three intra-arterialadministrations of autologous mesoangioblasts (MABs) with respect to improving musclestrength and reduce fatigue of the treated biceps brachii (BB) compared to the untreatedBB.The second primary objective is safety of three intra-arterial administrations ofautologous MABs, which the investigators will assess by monitoring (serious) adverseevents ((S)AEs), blood flow in left arm pre- and post-intervention, and neurologicalvital signs during 8h post-intervention observation in the hospital.Secondary objectives are to assess changes in muscle mass of the treated and untreated BBmuscle, and microscopic changes and m.3243A>G mutation load at tissue level in treatedbiceps brachii (BB) muscle at baseline and after treatment.Up to 20 adult m.3243A>G patients will undergo a ~30mg m. biceps brachii muscle biopsy atvisit 1. The first six eligible patients will enroll the clinical study based on theirm.3243A>G mutation load in skeletal muscle (50-90%) and mesoangioblasts (
Not Provided
Biological: Intra-arterial delivery of autologous MABs
three times intra-arterial administration of autologous mesoangioblasts in biceps brachii
of the left arm at 4-6 week interval
Inclusion Criteria:
- Written informed consent
- Age: 18-64
- Sex: male/female
- Patients with the m.3243A>G mutation load of 50%-90% determined in skeletal muscle
or derived from age-corrected calculation of blood m.3243A>G mutation load
Exclusion Criteria:
A potential subject who meets any of the following criteria will be excluded from
participation in this study:
- 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
- Claustrophobia
- Metal implants
- Any other factor that in the opinion of the investigator excludes the patient from
the study
Maastricht University Medical Center
Maastricht, Netherlands
Investigator: Florence van Tienen, PhD
Contact: 0031433882918
Florence van Tienen, PhD
00314331995
florence.vantienen@maastrichtuniversity.nl
Bert Smeets, Prof. PhD
00314331995
bert.smeets@maastrichtuniversity.nl
Janneke Hoeijmakers, MD, PhD, Principal Investigator
Maastricht University Medical Center