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

Withdrawn

• Conditions: mitochondrial myopathy; mitochondrial muscular disease; 10028302

• Registration Number: NL-OMON42959
• Lead Sponsor: Erasmus MC, Universitair Medisch Centrum Rotterdam

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 23 April 2024

Recruitment & Eligibility

• Status: Withdrawn
• Sex: Not specified
• Target Recruitment: 5

Inclusion Criteria

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

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

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Assess safety </p><br>

Secondary Outcome Measures

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