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MABs Therapy m.3243A>G Mutation Carriers

NCT05063721 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2024-01-17

No results posted yet for this study

Summary

Rationale: Mitochondrial disorders are progressive, often fatal multisystem disorders, in 20-25% of the cases caused by heteroplasmic mutations in the mitochondrial DNA (mtDNA). At this moment, there is no effective treatment known to influence the disease process or manifestation. Myogenic stem cell-based therapies complementing defective muscle cells and fibres, are highly promising to combat the myopathy and exercise intolerance which affect \>50% of heteroplasmic mtDNA mutation carriers. Myogenic stem cells called mesoangioblasts (MABs), are currently the only myogenic precursors that fulfil all criteria to be used as advanced therapy medicinal product (ATMP) for systemic treatment. The researchers have demonstrated that MABs of most m.3243A\>G carriers contain no or only a low amount (\<10%) of the mtDNA mutation, allowing direct ex vivo expansion of patient-derived MABs. The overall aim is to induce muscle regeneration using these autologous MABs with a mutation load of \<10%, as an advanced therapy medicinal product (ATMP).

Objective: The phase I trial will consist of an intra-arterial injection (via catheter in femoral artery) of the autologous MABs in the left lower leg of 5 m.3243A\>G patients.

Conditions

  • Mitochondrial Myopathies

Interventions

BIOLOGICAL

autologous mesoangioblasts

intra-arterial administration of autologous mesoangioblasts in lower leg (50x10E6/kg)

Sponsors & Collaborators

  • Maastricht University

    lead OTHER

Principal Investigators

  • Karin Faber, Prof. PhD MD · Maastricht University Medical Center

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-03-01
Primary Completion
2022-09-16
Completion
2022-12-01

Countries

  • Netherlands

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05063721 on ClinicalTrials.gov