Clinical trial NCT02097368: Bulbar Function in Neuromuscular Patients
Official title: Prospective Evaluation of Bulbar Function in Neuromuscular Patients
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Neuromuscular Diseases
Keywords: Tongue strength
neuromuscular
swallowing
Lead sponsor: Centre d'Investigation Clinique et Technologique 805
Sponsor class: OTHER
First posted: 2014-03-27
Enrollment: 100
Sex: ALL
Minimum age (years): 6
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: patients affected by neuromuscular pathology

Brief summary: The main objective is prospectively study the upper airways function in an adult population of neuromuscular patients. The main measurement will be the evolution of tongue strength recorded each year during annual clinical evaluation on a 5 years period.

Detailed description: The main objective is to study the function of the superior airways (tongue and pharynx) in an non invasive way in neuromuscular patients followed in the home ventilation unit of the Raymond POINCARE hospital (myopathy, neuropathy, disease of the neuromuscular junction or anterior horn) or of Necker Hospital and to detect prematurely the bulbar dysfunction.

The secondary objectives of this prospective monocentric observational physiopathological study are:

1. to study the correlation between the possible swallowing dysfunction and the anomalies of the respiratory parameters
2. to establish correlations between infraclinical or clinical and presence of morphological tongue anomalies detected by magnetic resonance imaging like in acid maltase deficiency.

The maximal tongue strength protrusion will be measured according to a method already used in the laboratory to explore sleep apnea syndromes.

Swallowing will be estimated by a non invasive method already used to highlight swallowing disorders in neurological and muscular pathologies This method will be associated with a method of recording of thoracic and abdominal movements.

Data wil be compared to a control database obtained for a previous study

Eligibility criteria: Inclusion Criteria:

* neuromuscular disorder
* age greater than or equal to 6
* Hospitalization in Home Ventilation Unit - Written informed consent

Exclusion Criteria:

* patient's refusal to participate
* major swallowing disfunction
* Acute respiratory failure
* no affiliation to social security

Primary outcomes:
measure: Change of tongue strength measurement
timeFrame: 5 years
Secondary outcomes:
measure: swallowing tests
timeFrame: 5 years
measure: Respiratory parameters
timeFrame: 5 years
measure: Vital capacity
timeFrame: 5 years
measure: Respiratory muscle strength
timeFrame: 5 years
Other outcomes:
Locations:
city: Garches
country: France
facility: Home Ventilation Unit, Raymond Poincare Hospital
city: Paris
country: France
facility: Non invasive ventilation Unit - Necker Hospital
Study officials:
affiliation: Centre d'Investigation Clinique et Technologique 805
name: David Orlikowski, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Centre d'Investigation Clinique et Technologique 805
name: Helene Prigent, MD,PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Centre d'Investigation Clinique et Technologique 805
name: Frederic Lofaso, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: