NCT04045158

Brief Summary

Muscular dystrophies are neurologic disease with higher morbidity and mortality. Cardiac function and respiratory muscles can be affected, in addition with skeletal muscle deficiency . Lung function is classically assessed with functionnal pulmonary tests (FPT) . Ultrasound recently emerged as a non-invasive tool to assess diaphragm function. The aims of the study are:

  • to provide the spectrum of diaphragm ultrasound in patients with muscular dystrophies
  • to assess the correlation between diaphragm ultrasound and FPT
  • to assess the correlation between diaphragm ultrasound and cardiac function

Trial Health

43
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
1,000

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started May 2019

Shorter than P25 for all trials

Geographic Reach
1 country

1 active site

Status
unknown

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

Study Start

First participant enrolled

May 15, 2019

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

July 22, 2019

Completed
14 days until next milestone

First Posted

Study publicly available on registry

August 5, 2019

Completed
5 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 28, 2019

Completed
4 months until next milestone

Study Completion

Last participant's last visit for all outcomes

May 10, 2020

Completed
Last Updated

August 5, 2019

Status Verified

August 1, 2019

Enrollment Period

8 months

First QC Date

July 22, 2019

Last Update Submit

August 2, 2019

Conditions

Keywords

diaphragmultrasoundechocardiography

Outcome Measures

Primary Outcomes (1)

  • diaphragm ultrasound motion

    using echography

    1 year

Secondary Outcomes (4)

  • correlation between diaphragm motion (mm) and forced vital capacity(ml)

    1 year

  • correlation between diaphragm motion (mm) and inspiratory capacity (ml)

    1 year

  • diaphragm thickness (mm)

    1 year

  • correlation between diaphargm thickness and maximal inspiratory pressure (cmH20)

    1 year

Eligibility Criteria

Sexall
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodProbability Sample
Study Population

Patients with neuromuscular diseases admitted for a cardio-respiratory function evaluation

You may qualify if:

  • Duchenne muscular dystrophy,
  • Becker muscular dystrophy,
  • LGMD,
  • myotonic dystrophy type 1 and type 2,
  • metabolic myopathy,
  • mitochondrial myopathy,
  • FSHD1 and 2,
  • myasthenia

You may not qualify if:

  • history of cardiac surgery
  • history of thoracic surgery
  • brain traumatism

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hopital Raymond Poincare

Garches, 92380, France

Location

Related Publications (1)

  • Fayssoil A, Nguyen LS, Stojkovic T, Prigent H, Carlier R, Amthor H, Bergounioux J, Zini J, Damez-Fontaine S, Wahbi K, Laforet P, Nicolas G, Behin A, Bassez G, Leturcq F, Ben Yaou R, Mansencal N, Annane D, Lofaso F, Orlikowski D. Determinants of diaphragm inspiratory motion, diaphragm thickening, and its performance for predicting respiratory restrictive pattern in Duchenne muscular dystrophy. Muscle Nerve. 2022 Jan;65(1):89-95. doi: 10.1002/mus.27432. Epub 2021 Oct 25.

MeSH Terms

Conditions

Muscular Dystrophies

Condition Hierarchy (Ancestors)

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

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
RETROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD PhD

Study Record Dates

First Submitted

July 22, 2019

First Posted

August 5, 2019

Study Start

May 15, 2019

Primary Completion

December 28, 2019

Study Completion

May 10, 2020

Last Updated

August 5, 2019

Record last verified: 2019-08

Locations