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Intra-arterial autologous myogenic stem cell therapy for m.3243A>G mutation carriers

Intra-arterial autologous myogenic stem cell therapy for m.3243A>G mutation carriers - MABs therapy m.3243A>G mutation carriers

Status
Unknown
Phases
Unknown
Study type
Interventional
Source
NL-OMON
Registry ID
NL-OMON42959
Enrollment
5
Registered
2017-05-24
Start date
Unknown
Completion date
Unknown
Last updated
2024-04-23

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

mitochondrial myopathy

Interventions

A vastus lateralis muscle biopsy will be collected for isolation and ex vivo expansion of mesoangioblasts. Intra-arterial administration of autologous mesoangioblasts in right lower leg. Total dosis

Sponsors

Erasmus MC, Universitair Medisch Centrum Rotterdam
Lead Sponsor

Eligibility

Age
18 Years to 99 Years

Inclusion criteria

Inclusion criteria: Adult carriers of a m.3243A>G mutation in muscle >30%

Exclusion criteria

Exclusion criteria: Significant concurrent illness 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 Any other factor that in the opinion of the investigator excludes the patient from the study Allergy contrast fluid

Design outcomes

Primary

MeasureTime frame
Assess safety—

Secondary

MeasureTime frame
Assess effectiveness (strength tibialis anterior muscle, myogenesis (NCAM/eMHC+ fibers), the m.3243A>G mutation load in newly formed muscle (NCAM/eMHC+) fibers, OXPHOS capacity and muscle+mitochondrial morphology) and homing (inflammation markers blood) of the ATMP.—

Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)