NCT03490214

Brief Summary

This pilot study aims to assess subcellular muscle structure in patients with Duchenne X-linked progressive Duchenne muscular dystrophy (DMD) in comparison to healthy volunteers using multispectral optoacoustic tomography (MSOT). During MSOT, a transducer is placed on the skin similar to a conventional sonography and instead of sound, energy is supplied to the tissue by means of light flashes. This leads to a constant change of minimal expansions and contractions (thermoelastic expansion) of individual tissue constituents or molecules. The resulting sound waves can then be detected by the same examination unit.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Jun 2018

Shorter than P25 for not_applicable

Geographic Reach
1 country

1 active site

Status
completed

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

March 19, 2018

Completed
18 days until next milestone

First Posted

Study publicly available on registry

April 6, 2018

Completed
2 months until next milestone

Study Start

First participant enrolled

June 1, 2018

Completed
2 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2018

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2018

Completed
Last Updated

December 9, 2019

Status Verified

December 1, 2019

Enrollment Period

2 months

First QC Date

March 19, 2018

Last Update Submit

December 5, 2019

Conditions

Duchenne Muscular DystrophyMuscular DystrophiesMuscular Dystrophy, Duchenne

Keywords

OptoacousticsMSOTMultispectral Optoacoustic Tomography

Outcome Measures

Primary Outcomes (2)

  • Muscular lipid content

    Quantitative lipid signal derived by transcutaneous Multispectral Optoacoustic Tomography (MSOT) in patients with DMD compared to healthy control Units: arbitrary units (a.u.)

    Single time point (1 day)

  • Muscular collagen content

    Quantitative collagen signal derived by transcutaneous Multispectral Optoacoustic Tomography (MSOT) in patients with DMD compared to healthy control Units: arbitrary units (a.u.)

    Single time point (1 day)

Secondary Outcomes (10)

  • Muscular myo-/hemoglobin content

    Single time point (1 day)

  • Correlation of lipid signal with age/disease duration

    Single time point (1 day)

  • Correlation of myo-/hemoglobin signal with age/disease duration

    Single time point (1 day)

  • Correlation of lipid signal with 6MWT

    Single time point (1 day)

  • Correlation of lipid signal with MRC

    Single time point (1 day)

  • +5 more secondary outcomes

Study Arms (2)

Muscular Dystrophia

EXPERIMENTAL

Multispectral Optoacoustic Tomography (MSOT) of muscles (left and right, total 8 sites) leg proximal: Musculus quadriceps, distal: Musculus triceps surae arm proximal: Musculus biceps, distal: Musculus brachioradialis

Device: Multispectral Optoacoustic Tomography

Healthy Volunteer

ACTIVE COMPARATOR

Multispectral Optoacoustic Tomography (MSOT) of muscles (left and right, total 8 sites) leg proximal: Musculus quadriceps, distal: Musculus triceps surae arm proximal: Musculus biceps, distal: Musculus brachioradialis

Device: Multispectral Optoacoustic Tomography

Interventions

Multispectral Optoacoustic TomographyDEVICE

Non-invasive transcutaneous imaging of subcellular muscle components

Healthy VolunteerMuscular Dystrophia

Eligibility Criteria

Age3 Years - 10 Years
Sexmale(Gender-based eligibility)
Gender Eligibility DetailsMale
Healthy VolunteersYes
Age GroupsChild (0-17)

You may qualify if:

  • Histologic or genetically proven DMD
  • Age 3-10 years

You may not qualify if:

  • Healthy controls
  • Male
  • Age 3-10 years
  • Suspected muscular disease/myopathia
  • missing informed consent

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Department of Pediatrics and Adolescent Medicine, University Hospital Erlangen

Erlangen, Bavaria, 91054, Germany

Location

Related Publications (1)

  • Regensburger AP, Fonteyne LM, Jungert J, Wagner AL, Gerhalter T, Nagel AM, Heiss R, Flenkenthaler F, Qurashi M, Neurath MF, Klymiuk N, Kemter E, Frohlich T, Uder M, Woelfle J, Rascher W, Trollmann R, Wolf E, Waldner MJ, Knieling F. Detection of collagens by multispectral optoacoustic tomography as an imaging biomarker for Duchenne muscular dystrophy. Nat Med. 2019 Dec;25(12):1905-1915. doi: 10.1038/s41591-019-0669-y. Epub 2019 Dec 2.

    PMID: 31792454RESULT

MeSH Terms

Conditions

Muscular Dystrophy, DuchenneMuscular Dystrophies

Condition Hierarchy (Ancestors)

Muscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Ferdinand Knieling, Dr.

    Department of Pediatrics and Adolescent Medicine, University Hospital Erlangen

    PRINCIPAL INVESTIGATOR

  • Regina Trollmann, Prof. Dr.

    Department of Pediatrics and Adolescent Medicine, University Hospital Erlangen

    PRINCIPAL INVESTIGATOR

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

March 19, 2018

First Posted

April 6, 2018

Study Start

June 1, 2018

Primary Completion

August 1, 2018

Study Completion

September 1, 2018

Last Updated

December 9, 2019

Record last verified: 2019-12

Data Sharing

IPD Sharing
Will not share

Locations

DE(1)