Clinical trial • Phase I/II • Endocrinology|Musculoskeletal|Other|Rare Disease

AUTOLOGOUS MUSCLE PRECURSOR CELLS for Mitochondrial myopathy|m.3243A>G mitochondrial disease

Phase I/II trial of AUTOLOGOUS MUSCLE PRECURSOR CELLS for Mitochondrial myopathy|m.3243A>G mitochondrial disease. None/Not specified-controlled.

Overview

Trial Therapeutic Area
Endocrinology|Musculoskeletal|Other|Rare Disease
Trial Disease
Mitochondrial myopathy|m.3243A>G mitochondrial disease
Trial Stage
Phase I/II
Drug Modality
Cell therapy

Key dates

Initial CTIS Submission Date
25-10-2024
First CTIS Authorization Date
19-11-2024

Trial design

None/Not specified-controlled Phase I/II trial across 1 site in Netherlands.

Comparator
None/Not specified
Target Sample Size
20

Eligibility

Recruits 20 No vulnerable population selected. Trial enrols adults aged 18-64; written informed consent is required. Participants with psychiatric or other disorders likely to impact informed consent are explicitly excluded. Subject information and informed consent form documents are provided (PIF documents listed)..

Pregnancy Exclusion
Pregnant or lactating women
Vulnerable Population
No vulnerable population selected. Trial enrols adults aged 18-64; written informed consent is required. Participants with psychiatric or other disorders likely to impact informed consent are explicitly excluded. Subject information and informed consent form documents are provided (PIF documents listed).

Inclusion criteria

  • {"criterion_text":"- Written informed consent"}
  • {"criterion_text":"- Age: 18-64"}
  • {"criterion_text":"- Sex: male/female"}
  • {"criterion_text":"- Patients with the hetroplasmic m.3243A>G mutation."}

Exclusion criteria

  • {"criterion_text":"- Use of dabigatran, apixaban, edoxaban or rivaroxaban (DOACs) as anti-coagulants"}
  • {"criterion_text":"- Have a weekly alcohol intake of ≥ 35 units (men) or ≥ 24 units (women)"}
  • {"criterion_text":"- Current history of drug abuse"}
  • {"criterion_text":"- Deficient immune system or autoimmune disease"}
  • {"criterion_text":"- Significant concurrent illness"}
  • {"criterion_text":"- Ongoing participation in other clinical trials with intervention"}
  • {"criterion_text":"- Pregnant or lactating women"}
  • {"criterion_text":"- Psychiatric or other disorders likely to impact on informed consent"}
  • {"criterion_text":"- Patients unable and/or unwilling to comply with treatment and study instructions"}
  • {"criterion_text":"- A history of strokes with signs of extra-pyramidal or pyramidal syndrome"}
  • {"criterion_text":"- Allergy for contrast fluid"}
  • {"criterion_text":"- Peripheral signs of ischemia or vasculopathy"}
  • {"criterion_text":"- Any other factor that in the opinion of the investigator excludes the patient from the study"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Assessment of safety parameters (angiography, 8h post-procedure monitoring of neurological vital signs, (S)AE) and assessment of changes in muscle strength and muscle fatigue of treated and untreated biceps brachii muscle using Biodex dynamometer measurements at baseline and 4-6 weeks after the third ATMP administration.","definition_or_measurement_approach":"Safety: angiography, 8-hour post-procedure neurological vital signs monitoring, (S)AE reporting. Efficacy: changes in muscle strength and muscle fatigue measured by Biodex dynamometer at baseline and 4-6 weeks after the third ATMP administration."}

Secondary endpoints

  • {"endpoint_text":"- Assess muscle mass, morphology, m.3243A>G mutation load and mitochondrial respiratory capacity in muscle biopsies of treated BB muscle","definition_or_measurement_approach":"Evaluation performed on muscle biopsy samples of treated biceps brachii muscle to assess muscle mass, morphology, m.3243A>G mutation load and mitochondrial respiratory capacity."}

Recruitment

Planned Sample Size
20
Recruitment Window Months
31
Consent Approach
Written informed consent required from each participant. Participants are adults (18-64); no assent process described. Those with psychiatric or other disorders likely to impact informed consent are excluded. Subject information and informed consent form documents are listed in the trial documents (PIF_redacted and PIF_unredacted_cl).

Geography

Total Number Of Sites
1
Total Number Of Participants
20

Netherlands

Earliest CTIS Part Ii Submission Date
08-11-2024
Latest Decision Or Authorization Date
08-04-2025
Processing Time Days
151
Number Of Sites
1
Number Of Participants
20

Sites

Site Name
Academisch Ziekenhuis Maastricht
Department Name
Neurology
Contact Person Name
Janneke Hoeijmakers
Contact Person Email
j.hoeijmakers@mumc.nl
Number Of Participants
20

Sponsor

Primary sponsor

Full Name
Academisch Ziekenhuis Maastricht
Organisation Type
Hospital/Clinic/Other health care facility
Country Of Registered Address
Netherlands

Investigational products

Investigational Product Name
Autologous mesoangioblasts
Active Substance
AUTOLOGOUS MUSCLE PRECURSOR CELLS
Modality
Cell therapy
Routes Of Administration
Intra-arterial
Route
Intra-arterial
Frequency
Three intra-arterial administrations

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