Neuromuscular DiseasesWOB&MR_PedNCT05051254
Respiratory Muscles and Work of Breathing in Children
A study testing Esogastric pressure measurement in people with Neuromuscular Diseases and Congenital Diaphragmatic Hernia, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
Respiratory muscle testing allows a quantitative assessment of inspiratory and expiratory muscles in children of any age with primary or secondary respiratory muscle impairment, in order to better understand the pathophysiology of respiratory impairment and guide therapeutic management. Read more about this study
- Who is it for?
- Ages 0 to 17 years
- Conditions the study is for
- Neuromuscular Diseases, Congenital Diaphragmatic Hernia, Lung Diseases
- What is being tested?
- Esogastric pressure measurement
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 550 participants
- Where
- 1 siteFrance
- Study dates
- 19 Jan 2022to Jan 2028
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
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 0 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patients aged less than 18 years old with primary or secondary impairment of respiratory muscles and followed at Necker Hospital
- Patients under spontaneous breathing or noninvasive or invasive mechanical ventilation
- Written informed consent
Who cannot join
- No social insurance
- Significant psychomotor retardation
- Absence of cooperation
- Significant agitation
- Hemodynamic instability
- Acute condition or temporary drug treatments that may interfere with the results of the respiratory muscle explorations
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
France1 site, 1 recruiting
- Recruiting
Hôpital Necker-Enfants Malades
Paris, 75015
Brigitte FAUROUX, MD, PhD, 1 71 19 60 92 ext. +33, brigitte.fauroux@aphp.fr
Lucie GRIFFON, MD
About this study
Respiratory muscle testing allows a quantitative assessment of inspiratory and expiratory muscles in children of any age with primary or secondary respiratory muscle impairment, in order to better understand the pathophysiology of respiratory impairment and guide therapeutic management. The use of an invasive technique (esogastric probe) makes it possible to specifically explore the diaphragm, the accessory inspiratory muscles and the expiratory muscles in order to detect dysfunction or paralysis of these muscles, and to estimate the work of breathing in order to better guide the respiratory management.
The primary objective of the study is to evaluate the respiratory effort in children with primary or secondary impairment of the respiratory muscles during spontaneous breathing or during mechanical ventilation.
Read the detailed description
Respiratory muscle testing allows a quantitative assessment of inspiratory and expiratory muscles in children of any age with primary or secondary respiratory muscle impairment, in order to better understand the pathophysiology of respiratory impairment and guide therapeutic management. The use of an invasive technique (esogastric probe) makes it possible to specifically explore the diaphragm, the accessory inspiratory muscles and the expiratory muscles in order to detect dysfunction or paralysis of these muscles, and to estimate the work of breathing in order to better guide the respiratory management.
Respiratory muscle testing by means of esogastric measurements may allow assessing the effect of pharmacological treatment by comparing respiratory muscle strength before and after a few months with treatment. Moreover, esogastric measurements can be used to better adapt mechanical ventilation or to determine the possibility of weaning from the respiratory support.
The primary objective of the study is to evaluate the respiratory effort in children with primary or secondary impairment of the respiratory muscles during spontaneous breathing or during mechanical ventilation.
- Conditions
- Neuromuscular Diseases, Congenital Diaphragmatic Hernia, Lung Diseases, Cardiac Diseases, Scoliosis, Diaphragmatic Impairment
- Being tested
- Esogastric pressure measurement
- Keywords
- Respiratory muscle testing, Respiratory muscle strength, Work of breathing, Esogastric pressures, Diaphragmatic dysfunction, Diaphragmatic paralysis
- Medical subject headings
- Neuromuscular Diseases, Hernias, Diaphragmatic, Congenital, Lung Diseases, Heart Diseases, Scoliosis, Respiratory Paralysis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Esogastric pressure measurementOther
Measurement of work of breathing and respiratory muscles strength using an esogastric catheter.
One measurement or before and after (6 months and 1 year) the initiation of a pharmacological treatment in order to assess the effect of the treatment on respiratory muscle function.
| Study group | Role | What they receive |
|---|---|---|
| Respiratory muscle impairment | Experimental | Minor patients with primary or secondary impairment of respiratory muscles and followed at Necker Hospital Receives: Other: Esogastric pressure measurement |
What is being measured
The primary outcome is the main question the study is designed to answer.
Esophageal pressure-time product (PTPoes)
The integral of the esophageal pressure signal over inspiratory time
Measured: Day 0
Diaphragmatic pressure-time product (PTPdi)
The integral of the transdiaphragmatic pressure signal over inspiratory time
Measured: Day 0
Work of breathing (WOB)
Total work of breathing (WOBt) is calculated (in Joules/L) as the area under the pressure-volume curve, using the Campbell diagram. Elastic (WOBe) and resistive (WOBr) are estimated as the 2/3 and 1/3 of WOBt value, respectively.
Measured: Day 0
Secondary outcomes (29)
Vital capacity
Vital capacity is the maximum amount of air a person can expel from the lungs after a maximum inhalation, using spirometry
Measured: Day 0
Maximal respiratory static pressures
Maximal static inspiratory and expiratory airway pressures
Measured: Day 0
Maximal sniff pressure
Airway pressure during a sniff test
Measured: Day 0
Maximal whistle pressure
Airway pressure during a whistle test
Measured: Day 0
Peak expiratory flow
Maximal flow during expiratory maneuver
Measured: Day 0
Peak cough flow
Maximal flow during cough
Measured: Day 0
Esophageal pressure during sniff
Esophageal pressure measurement during a sniff test
Measured: Day 0
Gastric pressure during sniff
Gastric pressure measurement during a sniff test
Measured: Day 0
Transdiaphragmatic pressure during sniff
Transdiaphragmatic pressure measurement during a sniff test
Measured: Day 0
Crying transdiaphragmatic pressure
Transdiaphragmatic pressure measurement during crying in young children
Measured: Day 0
Gastric pressure during cough
Gastric pressure measurement during a maximal cough maneuver
Measured: Day 0
Twitch transdiaphragmatic pressure
Transdiaphragmatic pressure measurement during cervical magnetic stimulation
Measured: Day 0
Inspiratory muscles tension-time index
Measurement of the tension-time index of the inspiratory muscles
Measured: Day 0
Diaphragmatic tension-time index
Measurement of the tension-time index of the diaphragm
Measured: Day 0
Correlation between the pressure-time product, the work of breathing and the respiratory muscle strength with the disease severity
The pressure-time product of the respiratory muscles, the work of breathing and the respiratory muscle strength will be correlated with disease severity
Measured: Day 0
Correlation between the pressure-time product, the work of breathing and the respiratory muscle strength with polysomnography and gas exchange results
The pressure-time product of the respiratory muscles, the work of breathing and the respiratory muscle strength will be correlated with the polysomnography and gas exchange results
Measured: Day 0
Esophageal pressure-time products in patients with any treatment and in patients with no treatment
The esophageal pressure-time product will be compared between patients with any treatment and patients receiving no treatment
Measured: Day 0
The total work of breathing in patients with any treatment and in patients with no treatment
The total work of breathing (WOBt), which is calculated (in Joules/L) as the area under the pressure-volume curve using the Campbell diagram, be compared between patients with any treatment and patients receiving no traeatment, using the Student t test
Measured: Day 0
Measurement of esophageal pressure during a sniff in patients with any treatment and patients with no treatment
The esophageal pressure during a sniff will be compared between patients with any treatment and patients receiving no treatment
Measured: Day 0
Measurement of the esophageal pressure-time product before and after pharmacological treatment
The esophageal pressure-time product will be compared before treatment, and 6 months and 1 year after pharmacological treatment initiation
Measured: 6 months and 1 year after treatment initiation
The total work of breathing before and after pharmacological treatment
The total work of breathing (WOBt), which is calculated (in Joules/L) as the area under the pressure-volume curve using the Campbell diagram, will be compared before treatment, and 6 months and 1 year after pharmacological treatment initiation, using the ANOVA on repeated measures
Measured: 6 months and 1 year after treatment initiation
Measurement of the esophageal pressure during a sniff before and after pharmacological treatment
The esophageal pressure during a sniff will be compared before treatment, and 6 months and 1 year after pharmacological treatment initiation
Measured: 6 months and 1 year after treatment initiation
Measurement of transdiaphragmatic pressure during a sniff before and after pharmacological treatment
The transdiaphragmatic pressure measurement during a sniff will be compared before treatment, and 6 months and 1 year after pharmacological treatment initiation
Measured: 6 months and 1 year after treatment initiation
Adaptation of the respiratory support according to the esophageal pressure-time product values
The esophageal pressure-time product will be measured to guide the respiratory support adaptation
Measured: Day 0
Adaptation of the respiratory support according to the total work of breathing values
The total work of breathing (WOBt), which is calculated (in Joules/L) as the area under the pressure-volume curve using the Campbell diagram, will be measured to guide the respiratory support adaptation
Measured: Day 0
Measurement of the esophageal pressure-time product in patients with weaning success and in patients with weaning failure
The esophageal pressure-time product will be compared between patients with weaning success and weaning failure
Measured: Day 0
Measurement of the total work of breathing in patients with weaning success and in patients with weaning failure
The total work of breathing (WOBt), which is calculated (in Joules/L) as the area under the pressure-volume curve using the Campbell diagram, will be compared between patients with weaning success and weaning failure, using the Student t test
Measured: Day 0
Measurement of the esophageal pressure during a sniff in patients with weaning success and in patients with weaning failure
The esophageal pressure during a sniff will be compared between patients with weaning success and weaning failure
Measured: Day 0
Correlation between the pressure-time product, the work of breathing and the respiratory muscle strength with the gas exchange during weaning trial
The pressure-time product, the work of breathing and the respiratory muscle strength will be correlated with the gas exchange during a weaning trial
Measured: Day 0
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Sep 2021
- Study start19 Jan 2022
- Primary completionJan 2028 (estimated)
- Study completionJan 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromuscular DiseasesStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 550 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- URC-CIC Paris Descartes Necker Cochin
- Responsible party
- Sponsor
- Investigators
- Brigitte FAUROUX, MD, PhD, Principal investigator, Assistance Publique - Hôpitaux de ParisSonia KHIRANI, PhD, Study director, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05051254
- Sponsor's protocol ID
- APHP210261
- Other IDs
- ID-RCB 2020-A03393-36
- Acronym
- WOB&MR_Ped
- Status last verified
- Mar 2026
- First submitted
- 14 Jun 2021
- Last updated
- 3 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Respiratory Muscles and Work of Breathing in Children
- ClinicalTrials.gov ID
- NCT05051254
- Acronym / sponsor ID
- WOB&MR_Ped / APHP210261
- Conditions
- Neuromuscular Diseases, Congenital Diaphragmatic Hernia, Lung Diseases, Cardiac Diseases, Scoliosis, Diaphragmatic Impairment
- Keywords
- Respiratory muscle testing, Respiratory muscle strength, Work of breathing, Esogastric pressures, Diaphragmatic dysfunction, Diaphragmatic paralysis
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 550 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- URC-CIC Paris Descartes Necker Cochin
- Study start
- 19 Jan 2022 (actual)
- Primary completion
- Jan 2028 (estimated)
- Study completion
- Jan 2028 (estimated)
- First posted
- 21 Sep 2021 (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.
Respiratory muscle testing allows a quantitative assessment of inspiratory and expiratory muscles in children of any age with primary or secondary respiratory muscle impairment, in order to better understand the pathophysiology of respiratory impairment and guide therapeutic management. The use of an invasive technique (esogastric probe) makes it possible to specifically explore the diaphragm, the accessory inspiratory muscles and the expiratory muscles in order to detect dysfunction or paralysis of these muscles, and to estimate the work of breathing in order to better guide the respiratory management.
The primary objective of the study is to evaluate the respiratory effort in children with primary or secondary impairment of the respiratory muscles during spontaneous breathing or during mechanical ventilation.
Detailed description
Respiratory muscle testing allows a quantitative assessment of inspiratory and expiratory muscles in children of any age with primary or secondary respiratory muscle impairment, in order to better understand the pathophysiology of respiratory impairment and guide therapeutic management. The use of an invasive technique (esogastric probe) makes it possible to specifically explore the diaphragm, the accessory inspiratory muscles and the expiratory muscles in order to detect dysfunction or paralysis of these muscles, and to estimate the work of breathing in order to better guide the respiratory management.
Respiratory muscle testing by means of esogastric measurements may allow assessing the effect of pharmacological treatment by comparing respiratory muscle strength before and after a few months with treatment. Moreover, esogastric measurements can be used to better adapt mechanical ventilation or to determine the possibility of weaning from the respiratory support.
The primary objective of the study is to evaluate the respiratory effort in children with primary or secondary impairment of the respiratory muscles during spontaneous breathing or during mechanical ventilation.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Respiratory muscle impairment | Experimental | Other: Esogastric pressure measurementMinor patients with primary or secondary impairment of respiratory muscles and followed at Necker Hospital |
Who may qualify
- Ages
- 0 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Patients aged less than 18 years old with primary or secondary impairment of respiratory muscles and followed at Necker Hospital
- Patients under spontaneous breathing or noninvasive or invasive mechanical ventilation
- Written informed consent
Key exclusion criteria (6 listed)
- No social insurance
- Significant psychomotor retardation
- Absence of cooperation
- Significant agitation
- Hemodynamic instability
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Esogastric pressure measurementMeasurement of work of breathing and respiratory muscles strength using an esogastric catheter. One measurement or before and after (6 months and 1 year) the initiation of a pharmacological treatment in order to assess the effect of the treatment on respiratory muscle function. | Other | Respiratory muscle impairment |
Primary outcome
Esophageal pressure-time product (PTPoes)
Time frame: Day 0
The integral of the esophageal pressure signal over inspiratory time
Diaphragmatic pressure-time product (PTPdi)
Time frame: Day 0
The integral of the transdiaphragmatic pressure signal over inspiratory time
Work of breathing (WOB)
Time frame: Day 0
Total work of breathing (WOBt) is calculated (in Joules/L) as the area under the pressure-volume curve, using the Campbell diagram. Elastic (WOBe) and resistive (WOBr) are estimated as the 2/3 and 1/3 of WOBt value, respectively.
29 secondary outcomes
Vital capacity
Time frame: Day 0
Maximal respiratory static pressures
Time frame: Day 0
Maximal sniff pressure
Time frame: Day 0
Maximal whistle pressure
Time frame: Day 0
Peak expiratory flow
Time frame: Day 0
Peak cough flow
Time frame: Day 0
Esophageal pressure during sniff
Time frame: Day 0
Gastric pressure during sniff
Time frame: Day 0
Transdiaphragmatic pressure during sniff
Time frame: Day 0
Crying transdiaphragmatic pressure
Time frame: Day 0
Gastric pressure during cough
Time frame: Day 0
Twitch transdiaphragmatic pressure
Time frame: Day 0
Inspiratory muscles tension-time index
Time frame: Day 0
Diaphragmatic tension-time index
Time frame: Day 0
Correlation between the pressure-time product, the work of breathing and the respiratory muscle strength with the disease severity
Time frame: Day 0
Correlation between the pressure-time product, the work of breathing and the respiratory muscle strength with polysomnography and gas exchange results
Time frame: Day 0
Esophageal pressure-time products in patients with any treatment and in patients with no treatment
Time frame: Day 0
The total work of breathing in patients with any treatment and in patients with no treatment
Time frame: Day 0
Measurement of esophageal pressure during a sniff in patients with any treatment and patients with no treatment
Time frame: Day 0
Measurement of the esophageal pressure-time product before and after pharmacological treatment
Time frame: 6 months and 1 year after treatment initiation
The total work of breathing before and after pharmacological treatment
Time frame: 6 months and 1 year after treatment initiation
Measurement of the esophageal pressure during a sniff before and after pharmacological treatment
Time frame: 6 months and 1 year after treatment initiation
Measurement of transdiaphragmatic pressure during a sniff before and after pharmacological treatment
Time frame: 6 months and 1 year after treatment initiation
Adaptation of the respiratory support according to the esophageal pressure-time product values
Time frame: Day 0
Adaptation of the respiratory support according to the total work of breathing values
Time frame: Day 0
Measurement of the esophageal pressure-time product in patients with weaning success and in patients with weaning failure
Time frame: Day 0
Measurement of the total work of breathing in patients with weaning success and in patients with weaning failure
Time frame: Day 0
Measurement of the esophageal pressure during a sniff in patients with weaning success and in patients with weaning failure
Time frame: Day 0
Correlation between the pressure-time product, the work of breathing and the respiratory muscle strength with the gas exchange during weaning trial
Time frame: Day 0
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Apr 2026.
- Individual participant dataSharing planned: No
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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.
Brigitte FAUROUX, MD, PhD principal investigator
Assistance Publique - Hôpitaux de Paris
Sonia KHIRANI, PhD study director
Assistance Publique - Hôpitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Esogastric pressure measurement in people with Neuromuscular Diseases and Congenital Diaphragmatic Hernia, run by Assistance Publique - Hôpitaux de Paris 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 3 inclusion and 6 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: looking for participants now, as of the sponsor's last update on 3 Apr 2026. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Brigitte FAUROUX, MD, PhD (1 71 19 60 92 ext. +33, brigitte.fauroux@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Esophageal pressure-time product (PTPoes) (time frame: Day 0). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 3 Apr 2026.