New Imaging Biomarkers for Muscular Diseases - Multispectral Optoacoustic Imaging in Spinal Muscular Atrophy
MSOT_SMA
1 other identifier
interventional
20
1 country
1
Brief Summary
This study aims to refine the capability of MSOT to characterise muscle tissue and to determine non-invasive, quantitative biomarkers for the disease assessment in patients with spinal muscular atrophy (SMA) using Multispectral Optoacoustic Tomography (MSOT).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for not_applicable
Started Nov 2019
Shorter than P25 for not_applicable
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 2, 2019
CompletedFirst Posted
Study publicly available on registry
October 4, 2019
CompletedStudy Start
First participant enrolled
November 7, 2019
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 30, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
January 30, 2020
CompletedNovember 18, 2020
November 1, 2020
3 months
October 2, 2019
November 17, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Spectral profile of muscle tissue
Spectral profile of muscle tissue determined by multispectral optoacoustic tomography (MSOT) of patients with spinal muscular atrophy compared to healthy volunteers units: arbitrary units (a.u.)
Single time point (1 day)
Secondary Outcomes (14)
Muscular lipid content
Single time point (1 day)
Muscular collagen content
Single time point (1 day)
Muscular myo-/hemoglobin content
Single time point (1 day)
Muscular de-/oxygenated myo-/hemoglobin content
Single time point (1 day)
Correlation of lipid signal with clinical data (age/disease duration)
Single time point (1 day)
- +9 more secondary outcomes
Study Arms (2)
Healthy Volunteers (HV)
ACTIVE COMPARATOR* Multispectral Optoacoustic Tomography (MSOT) and B-Mode Ultrasound of muscles (left and right, total 8 sites) leg proximal: Musculus quadriceps, distal: Musculus triceps surae arm proximal: Musculus biceps, distal: forearm flexors; * physical assessment/milestones: Hammersmith Infant Neurological Examination (HINE)/ expanded Hammersmith functional motor scale (HFMSE)/ The Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP Intend)/ Upper Limb Module (ULM)
Spinal Muscular Atrophy (SMA) patients
EXPERIMENTAL* Multispectral Optoacoustic Tomography (MSOT) and B-Mode Ultrasound of muscles (left and right, total 8 sites) leg proximal: Musculus quadriceps, distal: Musculus triceps surae arm proximal: Musculus biceps, distal: forearm flexors; * physical assessment/milestones: Hammersmith Infant Neurological Examination (HINE)/ expanded Hammersmith functional motor scale (HFMSE)/ The Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP Intend)/ Upper Limb Module (ULM)
Interventions
Non-invasive transcutaneous imaging of subcellular muscle components
Eligibility Criteria
You may qualify if:
- genetically proven SMA
You may not qualify if:
- Pregnancy
- Tattoo on skin to be examined
- For healthy volunteers only: suspected muscular disease/myopathia
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Department of Pediatrics and Adolescent Medicine
Erlangen, Bavaria, 91054, Germany
Related Publications (1)
Nedoschill E, Wagner AL, Danko V, Buehler A, Raming R, Jungert J, Neurath MF, Waldner MJ, Rother U, Woelfle J, Trollmann R, Knieling F, Regensburger AP. Monitoring spinal muscular atrophy with three-dimensional optoacoustic imaging. Med. 2024 May 10;5(5):469-478.e3. doi: 10.1016/j.medj.2024.02.010. Epub 2024 Mar 25.
PMID: 38531362DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Ferdinand Knieling, MD
University Hospital Erlangen, Department of Pediatric and Adolescent Medicine
- PRINCIPAL INVESTIGATOR
Regina Trollmann, MD
University Hospital Erlangen, Department of Pediatric and Adolescent Medicine
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- DIAGNOSTIC
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 2, 2019
First Posted
October 4, 2019
Study Start
November 7, 2019
Primary Completion
January 30, 2020
Study Completion
January 30, 2020
Last Updated
November 18, 2020
Record last verified: 2020-11
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP
- 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.
Individual participant data that underlie the results reported in the primary publication, after deidentification (text, tables, figures, and appendices)