Respiratory Muscles and Work of Breathing in Children

Plain English Summary

Respiratory Muscles and Work of Breathing in Children is a Not Applicable clinical trial sponsored by Assistance Publique - Hôpitaux de Paris studying Neuromuscular Diseases, Congenital Diaphragmatic Hernia, Lung Diseases, Cardiac Diseases, Scoliosis, Diaphragmatic Impairment, Respiratory Muscle Impairment. This trial tests how well a child's breathing muscles work and how hard they have to breathe. It is for children under 18 who have problems with their breathing muscles, often due to other health conditions. Participation involves a breathing test using a special catheter, and may include follow-up tests. Alternatives may include standard breathing tests or therapies, depending on the child's specific condition. The trial aims to enroll 550 participants.

Official 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. 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.

Who Can Participate

Here is what you need to know about eligibility for this trial. Children under 18 years old with breathing muscle problems who are treated at Necker Hospital can join. Children who are breathing on their own, or using breathing support (non-invasive or invasive), can participate. Children cannot join if they have no social insurance, significant developmental delays, are unable to cooperate, are very agitated, are hemodynamically unstable, or have a temporary condition that could affect test results. This trial is studying Neuromuscular Diseases, Congenital Diaphragmatic Hernia, Lung Diseases, Cardiac Diseases, Scoliosis, Diaphragmatic Impairment, Respiratory Muscle Impairment, so participants generally need a confirmed diagnosis. The trial is currently accepting new participants.

What They're Measuring

The primary outcome measures will show how much effort a child's breathing muscles are exerting, helping doctors understand the severity of their breathing impairment. The specific primary outcome measures are: Esophageal pressure-time product (PTPoes) (Day 0); Diaphragmatic pressure-time product (PTPdi) (Day 0); Work of breathing (WOB) (Day 0). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This study does not have a traditional clinical phase designation. It may be an observational study that follows patients without intervening in their care, an expanded access or compassionate use program, or other non-interventional research. These studies contribute valuable data about disease progression, treatment patterns, and patient outcomes.

Why This Trial Matters

This trial aims to better understand and manage breathing difficulties in children by precisely measuring their respiratory muscle function and effort, filling a gap in detailed diagnostic tools. This research targets Neuromuscular Diseases, Congenital Diaphragmatic Hernia, Lung Diseases, Cardiac Diseases, Scoliosis, Diaphragmatic Impairment, Respiratory Muscle Impairment, where improved treatment options are needed.

Investor Insight

This trial focuses on a niche but critical area of pediatric respiratory care, potentially leading to improved diagnostics and treatments for rare conditions, signaling a need for specialized medical The large enrollment target of 550 participants suggests significant investment in this program.

Is This Trial Right for Me?

Ask your doctor about what the breathing test involves and if it's suitable for your child. Understand that the test uses a catheter inserted through the nose or mouth to measure pressures inside the body. Participation may involve one test or multiple tests over time to see how treatments are working. This trial is currently recruiting participants. The trial is being conducted at 1 site. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

• Study Type: INTERVENTIONAL
• Allocation: NA
• Model: SINGLE_GROUP
• Masking: NONE
• Enrollment: 550 participants

Interventions

• OTHER: Esogastric pressure measurement — 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.

Primary Outcomes

• Esophageal pressure-time product (PTPoes) (Day 0)
• Diaphragmatic pressure-time product (PTPdi) (Day 0)
• Work of breathing (WOB) (Day 0)

Secondary Outcomes

• Vital capacity (Day 0)
• Maximal respiratory static pressures (Day 0)
• Maximal sniff pressure (Day 0)
• Maximal whistle pressure (Day 0)
• Peak expiratory flow (Day 0)

Full Eligibility Criteria

Inclusion Criteria:

* 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

Exclusion Criteria:

* 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

• Hôpital Necker-Enfants Malades, Paris, France

Frequently Asked Questions

Related Conditions

• Neuromuscular Diseases
• Congenital Diaphragmatic Hernia
• Lung Diseases
• Cardiac Diseases
• Scoliosis
• Diaphragmatic Impairment
• Respiratory Muscle Impairment
• Pediatric Respiratory Failure
• Ventilator Weaning
• Childhood Asthma

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