Assess safety of intra-arterial autologous myogenic stem cell therapy for m.3243A>G mutation carriers

Assess safety of intra-arterial autologous myogenic stem cell therapy for m.3243A>G mutation carriers - MABs therapy m.3243A>G mutation carriers

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON50120

Enrollment

Registered

2019-06-07

Start date

2020-11-19

Completion date

Unknown

Last updated

2024-04-15

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

Conditions

mitochondrial myopathy

Interventions

Intra-arterial administration of 5*10E7 MABs/kg in left lower leg (total dosage: 1,9*10E8 MABs for treatment of one lower leg of a 75kg individual).

Eligibility

Age

18 Years to 99 Years

Inclusion criteria

Inclusion criteria: Adult carriers of >40% m.3243A>G mutation in muscle

Exclusion criteria

Exclusion criteria: - Use of anti-coagulants, anti-thrombotics and other medication influencing coagulation - 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 - Major surgery within 4 weeks of the visit - Vaccination within 4 weeks of the visit - 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 - Stokes - 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
Primary endpoint: Safety The planned i.m. MABs injections will provide knowledge on safety of autologous MABs as asCTMP by: * Monitoring HR, BP, ECG, SaO2 to assess acute adverse events * Angiography before and after cell infusion to asses vessel obstruction * Assessment of systemic inflammation and muscle markers in venous blood samples collected within first 24 hours after infusion and after 28 days. * Assessment of local inflammation in tibialis anterior muscle biopsies taken 24 hours and 28 days after infusion.	—

Measure

Time frame

Primary endpoint: Safety The planned i.m. MABs injections will provide knowledge on safety of autologous MABs as asCTMP by: * Monitoring HR, BP, ECG, SaO2 to assess acute adverse events * Angiography before and after cell infusion to asses vessel obstruction * Assessment of systemic inflammation and muscle markers in venous blood samples collected within first 24 hours after infusion and after 28 days. * Assessment of local inflammation in tibialis anterior muscle biopsies taken 24 hours and 28 days after infusion.

—

Secondary

Measure	Time frame
- Secondary endpoints: Assess homing by quantifying the number of IC-Green labelled autologous MABs in the muscle biopsy 24 hours after i.a. administration. Assess effectiveness of intra-arterial delivered autologous MABs at the tissue level by quantifying the number of new (eMHC-positive) muscle fibers and by assessing the mtDNA mutation load in muscle biopsies after 28 days.	—

Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)