mitochondrial myopathy
Conditions
Interventions
3x intra-arterial administration (4-6 weeks interval) of 50 million
mesoangioblasts/kg in left arm (m.3243A>G
Sponsors
Medisch Universitair Ziekenhuis Maastricht
Eligibility
Age
18 Years to 64 Years
Inclusion criteria
Inclusion criteria: - Written informed consent - Age: 18-64 year - Sex: male/female - Patients with the heteroplasmic m.3243A>G mutation.
Exclusion criteria
Exclusion criteria: - 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Assessment of muscle strength and fatigue in treated and untreated BB muscle will enable to assess efficacy of three intra-arterial administrations of autologous MABs as ATMP to induce muscle regeneration. To assess the co-primary endpoint safety, we will check for (S)AEs, vascular obstructions and neurological vital signs. | — |
Secondary
| Measure | Time frame |
|---|---|
| Assess muscle mass in treated and untreated BB muscle at baseline and after last administration. Analyse muscle morphology, m.3243A>G mutation load and mitochondrial respiratory capacity in BB muscle biopsies of treated arm before and after last treatment. | — |
Countries
Netherlands
Outcome results
None listed