Evaluation of Therapeutic Response in Spinal Muscular Atrophy Using Multispectral Optoacoustic Tomography (MSOT) and Magnetic Resonance Imaging (MRI)

NCT04262570 | Unknown

• 1 other identifier: 453_19B
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Muscular Diseases; Spinal Muscular Atrophy

Keywords

Muscular Diseases; Spinal Muscular Atrophy (SMA); Multispectral Optoacoustic Tomography (MSOT); Magnetic Resonance Imaging (MRI)

Outcome Measures

Primary Outcomes (1)

• 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

  3 time points (at 0,2, and 12 months)

Secondary Outcomes (17)

• Muscular lipid content

  3 time points (at 0,2, and 12 months)

• Muscular collagen content

  3 time points (at 0,2, and 12 months)

• Muscular hemo-/myoglobin content

  3 time points (at 0,2, and 12 months)

• Muscular de-/oxygenated hemo-/myoglobin content

  3 time points (at 0,2, and 12 months)

• Ratio of lipid to hemo/myoglobin signal or collagen to hemo/myoglobin signal

  3 time points (at 0,2, and 12 months)

Study Arms (2)

SMA patients (therapy arm)

EXPERIMENTAL

* 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)

SMA patients (control arm)

ACTIVE COMPARATOR

* 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)

Interventions

Multispectral Optoacoustic Tomography (MSOT) and Magnetic Resonance Imaging (MRI) DEVICE

Non-invasive transcutaneous imaging of molecular muscle components

SMA patients (control arm); SMA patients (therapy arm)

Eligibility Criteria

• Age: 14 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Genetically confirmed SMA type III
• From age 14
• Willingness and ability to participate, sufficient knowledge of the german language to understand the declaration of consent, or if not possible, information of the patient in his/her mother tongue or English
• High probability that the patients will be able to fully participate in the study (defined by the ability to lie still for about 1 hour and follow any breathing commands) For therapy arm: • Medical indication for Spinraza® therapy; start of study before first administration Spinraza® administration For control arm: • No medical indication for Spinraza® therapy

You may not qualify if:

• Pregnancy
• Tattoo on the skin area to be examined
• General contraindications for MRT examinations
• Electrical implants like pacemakers or perfusion pumps
• Pronounced claustrophobia
• Study participants with ferromagnetic or electrically conductive implants such as aneurysm clips, surgical clips, prostheses, artificial hearts, heart valves with metal parts, metal splinters, tattoos next to the eye, symmetrical tattoos on the extremities or steel implants must consult the study physician; they may not be able to be examined (relative contraindications for MRI).
• Non-approved concomitant medication: strongly sedating medication must be excluded, as intensive monitoring of bodily functions during ongoing imaging cannot be guaranteed and the active participation of the test person might be necessary.

Sponsors & Collaborators

• University of Erlangen-Nürnberg Medical School: lead

Study Sites (1)

University Hospital Erlangen

Erlangen, 91054, Germany

RECRUITING

MeSH Terms

Conditions

Muscular Diseases; Muscular Atrophy, Spinal

Interventions

Magnetic Resonance Imaging

Condition Hierarchy (Ancestors)

Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Spinal Cord Diseases; Central Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases

Intervention Hierarchy (Ancestors)

Tomography; Diagnostic Imaging; Diagnostic Techniques and Procedures; Diagnosis

Study Officials

• Ferdinand Knieling, MD

  University Hospital Erlangen, Department of Pediatric and Adolescent Medicine

  PRINCIPAL INVESTIGATOR

• Matthias Türk, MD

  University Hospital Erlangen, Department of Neurology

  PRINCIPAL INVESTIGATOR

• Armin Nagel, MD

  University Hospital Erlangen, Department of Radiology

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Alexandra Wagner, MD

CONTACT

+49 9131 85 33118; alexandra.l.wagner@uk-erlangen.de

Ferdinand Knieling, MD

CONTACT

+49 9131 85 33118; ferdinand.knieling@uk-erlangen.de

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: January 27, 2020
• First Posted: February 10, 2020
• Study Start: February 25, 2020
• Primary Completion: December 1, 2021
• Study Completion: February 1, 2022
• Last Updated: February 28, 2020

Record last verified: 2020-02

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.

Locations

DE (1)