Assess effect and safety of intra-arterial autologous mesoangioblasts administration to the upper arm of m.3243A>G mutation carriers

Phase 2

• Conditions: mitochondrial myopathy; mitochondrial muscular disease; 10028302

• Registration Number: NL-OMON55910
• Lead Sponsor: Medisch Universitair Ziekenhuis Maastricht

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Last Update Posted: 9 April 2024

Recruitment & Eligibility

• Status: Pending
• Sex: Not specified
• Target Recruitment: 20

Inclusion Criteria

- Written informed consent - Age: 18-64 year - 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

- 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

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Assessment of muscle strength and fatigue in treated and untreated BB muscle<br /><br>will enable to assess efficacy of three intra-arterial administrations of<br /><br>autologous MABs as ATMP to induce muscle regeneration. To assess the co-primary<br /><br>endpoint safety, we will check for (S)AEs, vascular obstructions and<br /><br>neurological vital signs.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Assess muscle mass in treated and untreated BB muscle at baseline and after<br /><br>last administration. Analyse muscle morphology, m.3243A>G mutation load and<br /><br>mitochondrial respiratory capacity in BB muscle biopsies of treated arm before<br /><br>and after last treatment.</p><br>