MSOT as Non-invasive Biomarker for Diagnosis and Monitoring of Neuromuscular Diseases

Neuromuscular Diseases Clinical Trial

Official title:

Multispectral Optoacoustic Imaging (MSOT) as Non-invasive Biomarker for Diagnosis and Monitoring of Neuromuscular Diseases (MSOT-NMD)

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06438965
• Other study ID #: MSOT-01
• Status: Not yet recruiting
• Start date: July 2024
• Est. completion date: July 2028

Study information

• Verified date: May 2024
• Source: Children's Hospital of Eastern Ontario
• Contact: Hanns Lochmuller, Dr.
• Phone: (613) 737-7600
• Email: hlochmuller[@]toh.ca
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The goal of this study is to learn if Multispectral Optoacoustic Tomographs (MSOT) works to diagnose and follow the course of neuromuscular diseases (NMDs) in children. MSOT scans will be obtained from muscle region to measure hemo/myoglobin, collagen and lipid content/signal and oxygenation in patients with neuromuscular diseases. No additional research activities -other than MSOT - will be done during this study. Existing clinical, laboratory and imaging data from standard-of-care procedures will be correlated with the MSOT data. The expected total duration of the study is approximately 36 months. Repeated measurements will be done to evaluate disease progression and the value of MSOT in NMD.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 240
• Est. completion date: July 2028
• Est. primary completion date: July 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 18 Years

Eligibility

Inclusion Criteria:

Patients with neuromuscular disease

• Children (from birth (infants that are born term) to 18 years of age) participants or consent through authorized guardian

• Confirmed or suspected diagnosis of a neuromuscular disease (through molecular genetics, biopsy, clinical examination)

Exclusion Criteria:

Participants:

• Diagnosis is not consistent with a confirmed or suspected neuromuscular disease

• Patients with active skin lesions (e.g. infections, trauma) or confirmed genetic disorders (e.g. epidermolysis bullosa) that predisposes to skin lesion

• Medically unstable patients

• Tattoo on skin overlying the area to be examined

• Missing consent form

• Exclusion due to safety concerns of the investigator (subject who has any condition, including any physical, psychological, or psychiatric condition, which in the opinion of the Investigator, would compromise the safety of the subject or the quality of the data and renders the subject an unsuitable candidate for the study)

• Medication leading to increased light sensitivity

Related Conditions & MeSH terms

• Neuromuscular Diseases

Intervention

Device:
• Multispectral Optoacoustic Tomography (MSOT)
MSOT scans will be obtained from two muscle region to measure hemo/myoglobin, collagen and lipid content/signal and oxygenation in patient with neuromuscular diseases.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Children's Hospital of Eastern Ontario

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Comparison of the quantitative proportion of collagen, lipid, and hemo/myoglobin signal	Comparison of the quantitative proportion of collagen, lipid, and hemo/myoglobin signal determined by MSOT in muscle tissue of patients with different neuromuscular diseases with their clinical data.	Every 6-12 month, assessed up to 36 months. Some people might be in the study for a very short time, some might be in it for 36 months depending on the time they join the study
Secondary	Comparison of the quantitative amount of lipid and collagen signal	Comparison of the quantitative amount of lipid and collagen signal determined by MSOT in muscle tissue of children with different neuromuscular diseases.	Every 6-12 month, assessed up to 36 months. Some people might be in the study for a very short time, some might be in it for 36 months depending on the time they join the study
Secondary	Comparison of the quantitative proportion of oxygenated/deoxygenated hemo/myoglobin signal	Comparison of the quantitative proportion of oxygenated/deoxygenated hemo/myoglobin signal in muscle tissue of children with different neuromuscular diseases.	Every 6-12 month, assessed up to 36 months. Some people might be in the study for a very short time, some might be in it for 36 months depending on the time they join the study
Secondary	Comparison of the quantitative amount of oxygenated/deoxygenated hemo/myoglobin signal	Comparison of the quantitative amount of oxygenated/deoxygenated hemo/myoglobin signal determined by MSOT in muscle tissue of children with different neuromuscular diseases	Every 6-12 month, assessed up to 36 months. Some people might be in the study for a very short time, some might be in it for 36 months depending on the time they join the study
Secondary	Comparison of the MSOT-determined quantitative amount of hemo/myoglobin, lipid, and collagen signal at different positions /muscle groups intraindividually	Comparison of the MSOT-determined quantitative amount of hemo/myoglobin, lipid, and collagen signal at different positions /muscle groups intraindividually in children with different neuromuscular diseases, to identify disease specific distribution patterns	Every 6-12 month, assessed up to 36 months. Some people might be in the study for a very short time, some might be in it for 36 months depending on the time they join the study
Secondary	Comparison of the quantitative proportion of oxygenated/deoxygenated hemo/myoglobin, lipid, and collagen signal determined by MSOT at different positions /muscle groups intraindividually	Comparison of the quantitative proportion of oxygenated/deoxygenated hemo/myoglobin, lipid, and collagen signal determined by MSOT at different positions /muscle groups intraindividually in children with different neuromuscular diseases, to identify disease specific distribution patterns	Every 6-12 month, assessed up to 36 months. Some people might be in the study for a very short time, some might be in it for 36 months depending on the time they join the study
Secondary	Correlation of the collagen signal	Correlation of the collagen signal determined with MSOT with clinical assessments performed on a routine level during visits.	Every 6-12 month, assessed up to 36 months. Some people might be in the study for a very short time, some might be in it for 36 months depending on the time they join the study
Secondary	Correlation of the oxygenated/deoxygenated hemo/myoglobin signal determined with MSOT clinical assessments	Correlation of the oxygenated/deoxygenated hemo/myoglobin signal determined with MSOT clinical assessments performed on a routine level during visits.	Every 6-12 month, assessed up to 36 months. Some people might be in the study for a very short time, some might be in it for 36 months depending on the time they join the study
Secondary	Correlation of the collagen signal determined with MSOT with routine laboratory tests	Correlation of the collagen signal determined with MSOT with routine laboratory tests such as blood creatine kinase (CK) levels, transaminases.	Every 6-12 month, assessed up to 36 months. Some people might be in the study for a very short time, some might be in it for 36 months depending on the time they join the study
Secondary	Correlation of the oxygenated/deoxygenated hemo/myoglobin signal determined by MSOT with routine laboratory tests	Correlation of the oxygenated/deoxygenated hemo/myoglobin signal determined by MSOT with routine laboratory tests such as blood CK levels, transaminases.	Every 6-12 month, assessed up to 36 months. Some people might be in the study for a very short time, some might be in it for 36 months depending on the time they join the study
Secondary	Correlation of the collagen signal determined with MSOT with patient reported outcome measures.	Correlation of the collagen signal determined with MSOT with patient reported outcome measures.	Every 6-12 month, assessed up to 36 months. Some people might be in the study for a very short time, some might be in it for 36 months depending on the time they join the study
Secondary	Correlation of the oxygenated/deoxygenated hemo/myoglobin signal determined by MSOT with patient reported outcome measures.	Correlation of the oxygenated/deoxygenated hemo/myoglobin signal determined by MSOT with patient reported outcome measures.	Every 6-12 month, assessed up to 36 months. Some people might be in the study for a very short time, some might be in it for 36 months depending on the time they join the study
Secondary	Correlation of the collagen signal determined with MSOT with the current treatment regimen	Correlation of the collagen signal determined with MSOT with the current treatment regimen in patients with NMD.	Every 6-12 month, assessed up to 36 months. Some people might be in the study for a very short time, some might be in it for 36 months depending on the time they join the study
Secondary	Correlation of the oxygenated/deoxygenated hemo/myoglobin signal determined by MSOT with the current treatment regimen	Correlation of the oxygenated/deoxygenated hemo/myoglobin signal determined by MSOT with the current treatment regimen in patients with NMD.	Every 6-12 month, assessed up to 36 months. Some people might be in the study for a very short time, some might be in it for 36 months depending on the time they join the study