Muscular DystrophiesNCT04045158
Diaphragm Ultrasound in Neuromuscular Disorders
An observational study of Muscular Dystrophies, run by Centre d'Investigation Clinique et Technologique 805 at 1 site in France.
Trial 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) . Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Muscular Dystrophies
- How many people
- 1,000 participants
- Where
- 1 siteFrance
- Study dates
- 15 May 2019to 10 May 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Child, Adult, Older adult
Study population: Patients with neuromuscular diseases admitted for a cardio-respiratory function evaluation
Who may be able to join
- Duchenne muscular dystrophy,
- Becker muscular dystrophy,
- LGMD,
- myotonic dystrophy type 1 and type 2,
- metabolic myopathy,
- mitochondrial myopathy,
- FSHD1 and 2,
- myasthenia
Who cannot join
- history of cardiac surgery
- history of thoracic surgery
- brain traumatism
Trial locations and contacts
1 site in 1 country.
About this study
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
- Conditions
- Muscular Dystrophies
- Keywords
- diaphragm, ultrasound, echocardiography
- Medical subject headings
- Muscular Dystrophies
What is being measured
The primary outcome is the main question the study is designed to answer.
diaphragm ultrasound motion
using echography
Measured: 1 year
Secondary outcomes (4)
correlation between diaphragm motion (mm) and forced vital capacity(ml)
using echography and FPT
Measured: 1 year
correlation between diaphragm motion (mm) and inspiratory capacity (ml)
using echography and FPT
Measured: 1 year
diaphragm thickness (mm)
using echography
Measured: 1 year
correlation between diaphargm thickness and maximal inspiratory pressure (cmH20)
using echography and FPT
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Aug 2019
- Study start15 May 2019
- Primary completion28 Dec 2019 (estimated)
- Study completion10 May 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Muscular DystrophiesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 1,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Centre d'Investigation Clinique et Technologique 805
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Abdallah FAYSSOIL, Centre d'Investigation Clinique et Technologique 805
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04045158
- Sponsor's protocol ID
- DIAPH-NMD
- Status last verified
- Aug 2019
- First submitted
- 22 Jul 2019
- Last updated
- 5 Aug 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
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.
PubMed 34618930
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Diaphragm Ultrasound in Neuromuscular Disorders
- ClinicalTrials.gov ID
- NCT04045158
- Acronym / sponsor ID
- DIAPH-NMD
- Conditions
- Muscular Dystrophies
- Keywords
- diaphragm, ultrasound, echocardiography
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 1,000 participants (estimated)
- Lead sponsor
- Centre d'Investigation Clinique et Technologique 805University, hospital or other
- Study start
- 15 May 2019 (actual)
- Primary completion
- 28 Dec 2019 (estimated)
- Study completion
- 10 May 2020 (estimated)
- First posted
- 5 Aug 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with neuromuscular diseases admitted for a cardio-respiratory function evaluation
Key inclusion criteria (8 listed)
- Duchenne muscular dystrophy,
- Becker muscular dystrophy,
- LGMD,
- myotonic dystrophy type 1 and type 2,
- metabolic myopathy,
Key exclusion criteria (3 listed)
- history of cardiac surgery
- history of thoracic surgery
- brain traumatism
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
diaphragm ultrasound motion
Time frame: 1 year
using echography
4 secondary outcomes
correlation between diaphragm motion (mm) and forced vital capacity(ml)
Time frame: 1 year
correlation between diaphragm motion (mm) and inspiratory capacity (ml)
Time frame: 1 year
diaphragm thickness (mm)
Time frame: 1 year
correlation between diaphargm thickness and maximal inspiratory pressure (cmH20)
Time frame: 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Aug 2019.
- Derived from this study: PubMed 34618930Fayssoil 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Muscular Dystrophies, run by Centre d'Investigation Clinique et Technologique 805 at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 5 Aug 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
diaphragm ultrasound motion (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04045158, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Aug 2019.