Clinical trial NCT06438965: MSOT as Non-invasive Biomarker for Diagnosis and Monitoring of Neuromuscular Diseases
Official title: Multispectral Optoacoustic Imaging (MSOT) as Non-invasive Biomarker for Diagnosis and Monitoring of Neuromuscular Diseases (MSOT-NMD)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Neuromuscular Diseases
Lead sponsor: Children's Hospital of Eastern Ontario
Sponsor class: OTHER
First posted: 2024-06-03
Enrollment: 240
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Children with various neuromuscular diseases who are seen at the Children's Hospital of Eastern Ontario will be offered to participate in the study as part of their diagnostic work-up and at regular intervals during their ongoing care.

Brief 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.


Eligibility criteria: 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

Primary outcomes:
measure: Comparison of the quantitative proportion of collagen, lipid, and hemo/myoglobin signal
timeFrame: 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 outcomes:
measure: Comparison of the quantitative amount of lipid and collagen signal
timeFrame: 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
measure: Comparison of the quantitative proportion of oxygenated/deoxygenated hemo/myoglobin signal
timeFrame: 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
measure: Comparison of the quantitative amount of oxygenated/deoxygenated hemo/myoglobin signal
timeFrame: 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
measure: Comparison of the MSOT-determined quantitative amount of hemo/myoglobin, lipid, and collagen signal at different positions /muscle groups intraindividually
timeFrame: 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
measure: Comparison of the quantitative proportion of oxygenated/deoxygenated hemo/myoglobin, lipid, and collagen signal determined by MSOT at different positions /muscle groups intraindividually
timeFrame: 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
measure: Correlation of the collagen signal
timeFrame: 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
measure: Correlation of the oxygenated/deoxygenated hemo/myoglobin signal determined with MSOT clinical assessments
timeFrame: 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
measure: Correlation of the collagen signal determined with MSOT with routine laboratory tests
timeFrame: 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
measure: Correlation of the oxygenated/deoxygenated hemo/myoglobin signal determined by MSOT with routine laboratory tests
timeFrame: 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
measure: Correlation of the collagen signal determined with MSOT with patient reported outcome measures.
timeFrame: 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
measure: Correlation of the oxygenated/deoxygenated hemo/myoglobin signal determined by MSOT with patient reported outcome measures.
timeFrame: 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
measure: Correlation of the collagen signal determined with MSOT with the current treatment regimen
timeFrame: 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
measure: Correlation of the oxygenated/deoxygenated hemo/myoglobin signal determined by MSOT with the current treatment regimen
timeFrame: 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
Other outcomes:
Locations:
city: Ottawa
country: Canada
facility: CHEO
state: Ontario
Study officials:
References: