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Predictive Factors for the Diagnosis of Early Noninvasive Ventilation Equipment (PREDAPP)

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ClinicalTrials.gov Identifier: NCT03452618
Recruitment Status : Recruiting
First Posted : March 2, 2018
Last Update Posted : September 1, 2021
Sponsor:
Information provided by (Responsible Party):
University Hospital, Toulouse

Study Description
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Brief 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.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Diagnostic Test: diagnosis variables Not Applicable

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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 100 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: The study compare the diagnostic's variables in patients who received NIV one year after diagnosis and those who did not.
Masking: None (Open Label)
Primary Purpose: Diagnostic
Official Title: Predictive Factors for the Diagnosis of Early Noninvasive Ventilation Equipment in Patients With Amyotrophic Lateral Sclerosis
Actual Study Start Date : December 14, 2017
Estimated Primary Completion Date : December 14, 2021
Estimated Study Completion Date : December 14, 2022

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
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

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)
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

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)


Outcome Measures
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Primary Outcome Measures :
  1. Early Surgical Clinical Score [ Time Frame: One year after diagnostic ]

    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)


Secondary Outcome Measures :
  1. Sensitivity, specificity, negative and positive predictive values [ Time Frame: One year after the diagnostic ]
    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

  2. Percentage of decrease [ Time Frame: One year after the diagnostic ]
    % decrease in shortening fraction and diaphragmatic stroke between two quarterly reevaluations correlated with early fitting and % decrease in FVC, SNIP test et Pimax

  3. Slope of FVC decline [ Time Frame: One year after the diagnostic ]
    Slope of FVC decline before and after NIV implementation and slope of decline of shortening fraction and diaphragmatic stroke before and after NIV

  4. Quality of life score [ Time Frame: One year after the diagnostic ]
    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.


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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:

  • 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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03452618


Contacts
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Contact: Marion Dupuis, MD 5 67 77 16 91 ext 33 dupuis.m@chu-toulouse.fr
Contact: Isabelle Olivier, PhD 5 61 77 70 51 ext 33 olivier.i@chu-toulouse.fr

Locations
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France
University Hospital Toulouse Recruiting
Toulouse, France, 31052
Contact: Marion Dupuis, MD    5 67 77 16 91 ext 33    dupuis.m@chu-toulouse.fr   
Sponsors and Collaborators
University Hospital, Toulouse
Investigators
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Principal Investigator: Marion Dupuis, MD University Hospital, Toulouse
More Information
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Responsible Party: University Hospital, Toulouse
ClinicalTrials.gov Identifier: NCT03452618    
Other Study ID Numbers: RC31/17/0201
2017-A02202-51 ( Other Identifier: ID-RCB )
First Posted: March 2, 2018    Key Record Dates
Last Update Posted: September 1, 2021
Last Verified: August 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by University Hospital, Toulouse:
diagnostic criteria
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
 Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases