Amyotrophic Lateral SclerosisPREDAPPNCT03452618
Predictive Factors for the Diagnosis of Early Noninvasive Ventilation Equipment
A study testing diagnosis variables in people with Amyotrophic Lateral Sclerosis, run by University Hospital, Toulouse at 1 site in France.
Trial 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 more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- diagnosis variables
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 1 siteFrance
- Study dates
- 14 Dec 2017to 14 Dec 2022
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
France1 site, 1 recruiting
- Recruiting
University Hospital Toulouse
Toulouse, 31052
Marion Dupuis, MD, 5 67 77 16 91 ext. 33, dupuis.m@chu-toulouse.fr
About this study
Official title: Predictive Factors for the Diagnosis of Early Noninvasive Ventilation Equipment in Patients With Amyotrophic Lateral Sclerosis
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.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- diagnosis variables
- Keywords
- diagnostic criteria
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
diagnosis variablesDiagnostic test
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)
| Study group | Role | What they receive |
|---|---|---|
| patients with a NIV equipment | Experimental | Determination of diagnosis variables in a group of patient who benefit of the Non Invasive ventilation equipment one year after the diagnostic Receives: Diagnostic Test: diagnosis variables |
| patients without a NIV equipment | Active comparator | Determination of diagnosis variables in a group of patient who not benefit of the Non Invasive ventilation equipment one year after the diagnostic Receives: Diagnostic Test: diagnosis variables |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: One year after diagnostic
Secondary outcomes (4)
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
Measured: One year after the diagnostic
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
Measured: One year after the diagnostic
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
Measured: One year after the diagnostic
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.
Measured: One year after the diagnostic
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Mar 2018
- Study start14 Dec 2017
- Primary completion14 Dec 2021 (estimated)
- Study completion14 Dec 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- More trials run by University Hospital, ToulouseThe 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
- Non-randomised
- Intervention model
- Parallel groups
- 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)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Toulouse
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Marion Dupuis, MD, Principal investigator, University Hospital, Toulouse
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03452618
- Sponsor's protocol ID
- RC31/17/0201
- Other IDs
- 2017-A02202-51
- Acronym
- PREDAPP
- Status last verified
- Aug 2021
- First submitted
- 26 Feb 2018
- Last updated
- 1 Sep 2021
- 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 1 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Predictive Factors for the Diagnosis of Early Noninvasive Ventilation Equipment in Patients With Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT03452618
- Acronym / sponsor ID
- PREDAPP / RC31/17/0201
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- diagnostic criteria
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- University Hospital, ToulouseUniversity, hospital or other
- Study start
- 14 Dec 2017 (actual)
- Primary completion
- 14 Dec 2021 (estimated)
- Study completion
- 14 Dec 2022 (estimated)
- First posted
- 2 Mar 2018 (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.
To compensate for insufficiency of diagnostic tools, the present study propose to look for the predictive factors of an early fitting by noninvasive ventilation.
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
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- The study compare the diagnostic's variables in patients who received NIV one year after diagnosis and those who did not.
| Arm | Type | Receives |
|---|---|---|
| patients with a NIV equipment | Experimental | Diagnostic Test: diagnosis variablesDetermination of diagnosis variables in a group of patient who benefit of the Non Invasive ventilation equipment one year after the diagnostic |
| patients without a NIV equipment | Active comparator | Diagnostic Test: diagnosis variablesDetermination of diagnosis variables in a group of patient who not benefit of the Non Invasive ventilation equipment one year after the diagnostic |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (5 listed)
- 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
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 |
|---|---|---|
| diagnosis variablesThe 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) | Diagnostic test | patients with a NIV equipment, patients without a NIV equipment |
Primary outcome
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)
4 secondary outcomes
Sensitivity, specificity, negative and positive predictive values
Time frame: One year after the diagnostic
Percentage of decrease
Time frame: One year after the diagnostic
Slope of FVC decline
Time frame: One year after the diagnostic
Quality of life score
Time frame: One year after the diagnostic
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Sep 2021.
- 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. This study is not recruiting at the moment.
Marion Dupuis, MD principal investigator
University Hospital, Toulouse
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing diagnosis variables in people with Amyotrophic Lateral Sclerosis, run by University Hospital, Toulouse 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 2 inclusion and 5 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: status not confirmed, as of the sponsor's last update on 1 Sep 2021. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Marion Dupuis, MD (5 67 77 16 91 ext. 33, dupuis.m@chu-toulouse.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Early Surgical Clinical Score (time frame: One year after diagnostic). 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 1 Sep 2021.