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Status unknownNCT03452618PREDAPPUpdated Sep 1, 2021

Predictive Factors for the Diagnosis of Early Noninvasive Ventilation Equipment

An interventional study of diagnosis variables in Amyotrophic Lateral Sclerosis, sponsored by University Hospital, Toulouse. Status unknown at 1 site in France. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2021-09-01.

Sponsored by University Hospital, Toulouse · Not applicable, Interventional, and Diagnostic

The sponsor has not verified this record recently (last verified Aug 2021), so the status shown — last known as Recruiting — may be out of date.
Phase
Not applicable
Study type
Interventional
Enrollment
100
Allocation
Non-randomized
Ages
18 Years and older
Sex
All
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Study summary

To compensate for insufficiency of diagnostic tools, the present study propose to look for the predictive factors of an early fitting by noninvasive ventilation.

Read the detailed description

Amyotrophic lateral sclerosis (ALS) is the most common motor neuron disease. The diagnosis of this diaphragmatic dysfunction in the ALS subject is done all too often on the occasion of a hospitalization for acute respiratory insufficiency, which aggravates then the prognosis of the patients.

The presence of these factors at diagnosis will allow more surveillance to detect early diaphragmatic insufficiency and establish a NIV (Non Invasive Ventilation), the only therapeutic measure definitely improving the quality of life and survival of patients. A clinical score will be established to determine the risk of early Non Invasive Ventilation (NIV) equipment.

02

Conditions studied

  • Amyotrophic Lateral Sclerosis

Keywords

  • diagnostic criteria
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Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • diagnosis of amyotrophic lateral sclerosis just performed,
  • Patient who do not have the criteria for fitting by NIV and who have a Vital capacity ≥ 70% of theoretical values

Exclusion criteria

Exclusion Criteria:

  • Patient under court bail/ guardianship
  • Lack of consent for participation in the study
  • Presence of criteria for setting up the NIV for diagnosis according to the HAS (HealthCare Analytics Summit) 2006 recommendations
  • Vital capacity \<70% of the theoretical values
  • Patient under Continuous Positive Pressure (CPP) or NIV for a respiratory pathology other
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Study design

Phase
Not applicable
Primary purpose
Diagnostic
Allocation
Non-randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
100 participants (estimated)

Study arms

  • Experimental
    patients with a NIV equipment

    Determination of diagnosis variables in a group of patient who benefit of the Non Invasive ventilation equipment one year after the diagnostic

    Diagnostic Test: diagnosis variables

  • Active comparator
    patients without a NIV equipment

    Determination of diagnosis variables in a group of patient who not benefit of the Non Invasive ventilation equipment one year after the diagnostic

    Diagnostic Test: diagnosis variables

Interventions

  • Diagnostic testdiagnosis variables

    The study compare the diagnostic variables in patients who received Non invasive ventilation equipment one year after diagnosis with the patient who don't need it. For the independent variables, the present study determine a threshold at the diagnosis for which an early fitting risk appears The diagnostic variable are : * Age of the patient * Sex * Presence of bulbar involvement or not * Time from onset of first symptoms to diagnosis of ALS * Score ALSFRS-R * Presence of another underlying respiratory pathology (COPD, asthma ...) * FVC measurement (Forced vital capacity) * PaCO2 measurement * Measure SNIP (Sniff nasal inspiratory pressure) * Measure of Pimax (Pressure inspiratory maximal) * Measurement of the nocturnal desaturation index (number of desaturation less than 90% per hour of sleep)

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What researchers measure

Primary outcomes

  1. Early Surgical Clinical Score

    An Early Surgical Clinical Score will be established taking into account the independent variables correlated to diagnosis at early fitting. Clinical Variables : * Presence of bulbar involvement or not * Time from onset of first symptoms to diagnosis * Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) score : The Amyotrophic Lateral Sclerosis Functional Ra ting Scale (ALSFRS) is an instrument for evaluating the functional status of patients with Amyotrophic Lateral Sclerosis. It can be used to monitor functional change in a patient over time. * Presence of another underlying respiratory disease Functional Variables : * Time from onset of first symptoms to diagnosis of ALS * Presence of another underlying respiratory pathology * Forced Vital Capacity (FVC) measurement * PaCO2 measurement * Measure Sniff Nasal Inspiratory Pressure (SNIP) * Measurement of diaphragmatic activity on ultrasound * Parameters of the Polysomnography (PSG)

    Time frame: One year after diagnostic

Secondary outcomes

  1. Sensitivity, specificity, negative and positive predictive values

    Sensitivity, specificity, negative and positive predictive values of the diaphragmatic ultrasound (fraction of shortening and diaphragmatic stroke) compared to the SNIP test and for the values of PSG (Polysomnography) compared to SNIP test

    Time frame: One year after the diagnostic

  2. Percentage of decrease

    % decrease in shortening fraction and diaphragmatic stroke between two quarterly reevaluations correlated with early fitting and % decrease in FVC, SNIP test et Pimax

    Time frame: One year after the diagnostic

  3. Slope of FVC decline

    Slope of FVC decline before and after NIV implementation and slope of decline of shortening fraction and diaphragmatic stroke before and after NIV

    Time frame: One year after the diagnostic

  4. Quality of life score

    Quality of life score by the Severe Respiratory Insufficiency (SRI) questionnaire :before and after implementation of the NIV The Severe Respiratory Insufficiency (SRI) Questionnaire: a specific measure of health-related quality of life in patients receiving home mechanical ventilation.

    Time frame: One year after the diagnostic

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Study locations

1 of 1 sites recruiting
  • University Hospital Toulouse
    Toulouse, 31052, France
    Recruiting
07

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT03452618
Lead sponsor
University Hospital, Toulouse
Responsible party
Sponsor
First posted
Mar 2, 2018
Start date
Dec 14, 2017
Primary completion
Dec 14, 2021 (estimated)
Completion
Dec 14, 2022 (estimated)
Last update
Sep 1, 2021

Study contacts

Marion Dupuis, MD
Contact
dupuis.m@chu-toulouse.fr
5 67 77 16 91 ext. 33
Isabelle Olivier, PhD
Contact
olivier.i@chu-toulouse.fr
5 61 77 70 51 ext. 33
Marion Dupuis, MD
principal investigator · University Hospital, Toulouse

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
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