An interventional study of Multispectral Optoacoustic Tomography (MSOT) and Magnetic Resonance Imaging (MRI) in Muscular Diseases and Spinal Muscular Atrophy, sponsored by University of Erlangen-Nürnberg Medical School. Status unknown at 1 site in Germany. Open to participants aged 14 Years and older. Per ClinicalTrials.gov, last updated 2020-02-28.
Sponsored by University of Erlangen-Nürnberg Medical School · Not applicable, Interventional, and Diagnostic
This study aims to refine the capability of Multispectral Optoacoustic Tomography (MSOT) and Magnet Resonance Imaging (MRI) to characterise the molecular composition of muscle tissue non-invasively and to evaluate the therapeutic response in patients with spinal muscular atrophy (SMA) over time.
SMA is an autosomal-recessive disorder, characterized by progressive muscle weakness and atrophy with an incidence of 1/10,000. The condition is caused by a homozygous deletion or mutation in the survival motor neuron 1 (SMN1), resulting in reduced expression of the survival motor neuron (SMN) protein. This leads to the degeneration of motor neurons in the spinal cord and brain stem. A nearby related gene, survival motor neuron 2 (SMN2), could partially compensate the loss of SMN1. Individuals with a higher copy number of SMN2 do in general have a milder phenotype. New therapeutic approaches, e.g. nusinersen (spinraza©), an antisense oligonucleotide medication that modulates pre-messenger RNA splicing of the survival motor neuron 2 (SMN2) gene, are promising to help the formerly incurable disease. However, most clinical trials lack primary outcomes other than clinical testing. Preliminary work shows that new methods such as multispectral optoacoustic tomography (MSOT) and magnetic resonance imaging (MRI) detect tissue changes very sensitively. Multispectral optoacoustic tomography (MSOT) is capable of visualizing the distribution of endogenous absorbers by initiating laser-induced thermoelastic expansion and detection of resulting pressure waves. This imaging technique enables the label-free detection and quantification of different endogenous chromophores. In addition to this technology, MRI imaging has advanced in the field of muscle diseases, with 23Na-MRI being the first example. With both methods, the molecular composition of muscle tissue can be determined non-invasively and quantitatively at the same time. In this first pilot study on patients with SMA, the investigators will now assess whether the differences in the muscle composition of SMA patients with or without therapy can be quantified and whether they can be used simultaneously as markers during therapy with nusinersen (spinraza©) . Ideally, both techniques can complement or validate each other. In the future, this could generate a completely new, non-invasive method for evaluating endogenous biomarkers for therapy response.
282 studies on the registry are indexed under Muscular Diseases; 63 are open to participants now.
This study's planned enrollment of 10 is below the median of 34 across 158 interventional studies indexed under Muscular Diseases.
Browse Muscular Diseases studies →University of Erlangen-Nürnberg Medical School is the lead sponsor of 275 studies on the registry; 52 are open to participants now.
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Exclusion Criteria:
* Multispectral Optoacoustic Tomography (MSOT) of muscles (left and right, total 4 sites) leg proximal: Musculus quadriceps, distal: Musculus triceps surae arm proximal: Musculus biceps, distal: forearm flexors; * Magnetic Resonance Imaging (MRI) of lower leg * Physical assessment/milestones: expanded Hammersmith functional motor scale (HFMSE)/ Revised Upper Limb Module (RULM)/ 6-minute-walking-test (6-MWT)
Device: Multispectral Optoacoustic Tomography (MSOT) and Magnetic Resonance Imaging (MRI)
* Multispectral Optoacoustic Tomography (MSOT) of muscles (left and right, total 4 sites) leg proximal: Musculus quadriceps, distal: Musculus triceps surae arm proximal: Musculus biceps, distal: forearm flexors; * Magnetic Resonance Imaging (MRI) of lower leg * Physical assessment/milestones: expanded Hammersmith functional motor scale (HFMSE)/ Revised Upper Limb Module (RULM)/ 6-minute-walking-test (6-MWT)
Device: Multispectral Optoacoustic Tomography (MSOT) and Magnetic Resonance Imaging (MRI)
Non-invasive transcutaneous imaging of molecular muscle components
Evaluation of muscle structure under therapy and change from baseline over time
Comparison of the molecular muscle structure determined by MSOT and MRI in patients with SMA with and without treatment and evaluation of changes from baseline over time
Time frame: 3 time points (at 0,2, and 12 months)
Muscular lipid content
Quantitative lipid signal derived by transcutaneous Multispectral Optoacoustic Tomography (MSOT) in patients with SMA with and without therapy and change over time Units: arbitrary units (a.u.)
Time frame: 3 time points (at 0,2, and 12 months)
Muscular collagen content
Quantitative collagen signal derived by transcutaneous Multispectral Optoacoustic Tomography (MSOT) in patients with SMA with and without therapy and change over time Units: arbitrary units (a.u.)
Time frame: 3 time points (at 0,2, and 12 months)
Muscular hemo-/myoglobin content
Quantitative hemo/myoglobin signal derived by transcutaneous Multispectral Optoacoustic Tomography (MSOT) in patients with SMA with and without therapy and change over time Units: arbitrary units (a.u.)
Time frame: 3 time points (at 0,2, and 12 months)
Muscular de-/oxygenated hemo-/myoglobin content
Quantitative de-/oxygenated hemo-/myoglobin signal derived by transcutaneous Multispectral Optoacoustic Tomography (MSOT) in patients with SMA with and without therapy and change over time Units: arbitrary units (a.u.)
Time frame: 3 time points (at 0,2, and 12 months)
Ratio of lipid to hemo/myoglobin signal or collagen to hemo/myoglobin signal
Ratio of quantitative lipid signal to hemo/myoglobin signal or collagen signal to hemo/myoglobin signal derived by transcutaneous Multispectral Optoacoustic Tomography (MSOT) in patients with SMA with and without therapy and change over time
Time frame: 3 time points (at 0,2, and 12 months)
T1 relaxation time
T1 relaxation time determined by Magnetic Resonance Imaging (MRI) in patients with SMA with and without therapy and change over time
Time frame: 3 time points (at 0,2, and 12 months)
T2 relaxation time
T2 relaxation time determined by Magnetic Resonance Imaging (MRI) in patients with SMA with and without therapy and change over time
Time frame: 3 time points (at 0,2, and 12 months)
Fat-water percentage
Fat-water percentage determined by Magnetic Resonance Imaging (MRI) in patients with SMA with and without therapy and change over time
Time frame: 3 time points (at 0,2, and 12 months)
Sodium concentration
Sodium concentration determined by Magnetic Resonance Imaging (MRI) in patients with SMA with and without therapy and change over time
Time frame: 3 time points (at 0,2, and 12 months)
Correlation of MSOT data with therapy status
Correlation of the quantitative lipid/collagen/hemo/myoglobin and de-/oxygenated hemo-/myoglobin content determined by MSOT in patients with and without therapy and evaluation of change over time
Time frame: 3 time points (at 0,2, and 12 months)
Correlation of MSOT data with clinical data (age/disease duration)
Correlation of lipid/collagen/haemo/myoglobin and de-/oxygenated hemo-/myoglobin content determined by MSOT with disease duration/patient age and evaluation of change over time
Time frame: 3 time points (at 0,2, and 12 months)
Correlation of MSOT data with physical assessment (HFMSE/RULM/6-MWT)
Correlation of lipid/collagen/haemo/myoglobin and de-/oxygenated hemo-/myoglobin content determined by MSOT with HFMSE/Revised Upper Limb Module/6-MWT and evaluation of change over time
Time frame: 3 time points (at 0,2, and 12 months)
Correlation of MRI data with therapy status
Correlation of T1 relaxation time/T2 relaxation time/fat water portion/sodium concentration in patients with and without therapy and evaluation of change over time
Time frame: 3 time points (at 0,2, and 12 months)
Correlation of MRI data with clinical data (age/disease duration)
Correlation of T1 relaxation time/T2 relaxation time/fat water portion/sodium concentration with disease duration and patient age and evaluation of change over time
Time frame: 3 time points (at 0,2, and 12 months)
Correlation of MRI data with physical assessment (HFMSE/RULM/6-MWT)
Correlation of T1 relaxation time/T2 relaxation time/fat water portion/sodium concentration with HFMSE/Revised Upper Limb Module/6-MWT and evaluation of change over time
Time frame: 3 time points (at 0,2, and 12 months)
Correlation of MSOT data and MRI data
Correlation of MSOT determined lipid/collagen/haemo/myoglobin and de-/oxygenated hemo-/myoglobin content and MRI derived T1 relaxation time/T2 relaxation time/fat water portion/sodium concentration and evaluation of change over time
Time frame: 3 time points (at 0,2, and 12 months)
Side differences
Measurement of the signal differences in right / left comparison derived by Multispectral Optoacoustic Tomography (MSOT) and Magnetic Resonance Imaging (MRI) and evaluation of change over time
Time frame: 3 time points (at 0,2, and 12 months)
Documents are hosted by the registry — open the source record to download them.
Plan to share: Yes — Individual participant data that underlie the results reported in the primary publication, after deidentification (text, tables, figures, and appendices)
Supporting information: Study protocol, Statistical analysis plan
Time frame: Beginning 9 months and ending 36 months following article publication.
Access criteria: The data sets generated during and/or analyzed during the current study are available from the corresponding author on reasonable request as follows: Individual participant data will not be available Study Protocol and Statistical Analysis Plan will be available The data will be available beginning 9 months and ending 36 months following article publication. The data will be available to researchers who provide a methodologically sound proposal. The data will be available for individual participant data meta-analysis, only. Proposals may be submitted up to 36 months following article publication. After 36 months the data will be available in our University's data warehouse but without investigator support other than deposited metadata. Information regarding submitting proposals and accessing data may be found at https://www.uk-erlangen.de. Restrictions may apply due to patient privacy and the General Data Protection Regulation.
Started Feb 2020 (actual). Primary completion is estimated for Dec 2021, 22 months after the start.
Of 48 non-industry interventional studies with no phase under Muscular Diseases, half reached primary completion within 18 months of starting (the median), counting those still running; a quarter within 5 and three quarters within 40. This study's planned 22 months is above that.
Counting every sponsor, half of 49 studies reached it within 16 months; a quarter within 5 and three quarters within 40.
How these figures are worked out, and which studies are left out of them.
This study is status unknown, as verified in Feb 2020. You cannot join it, but the record below documents what was studied.
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