Neuromuscular DiseasesEqualVENTNCT04652401
Quality of Life in Ventilator-dependant Neuromuscular Patients
An observational study of Neuromuscular Diseases and Respiration, Artificial, run by Centre d'Investigation Clinique et Technologique 805 at 1 site in France.
Trial summary
This study aims at evaluating the quality of life of patients with slowly progressive neuromuscular disorders who are dependant on mechanical ventilation (daily usage ≥ 16h). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Neuromuscular Diseases, Respiration, Artificial, Tracheostomy
- How many people
- 119 participants
- Where
- 1 siteFrance
- Study dates
- 2 Dec 2020to 7 Dec 2021
- Phase
- Observational
- 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
Study population: Patients with slowly progressive neuromuscular disease using home mechanical ventilation for at least 16 hours a day since at least one year. Patients may be ventilated via invasive or noninvasive interface.
Who may be able to join
- Patients with slowly progressive neuromuscular disorder (NMD), non walking
- Patients dependant on mechanical ventilation (daily usage ≥ 16h) for at least 1 year before inclusion
- Aged ≥ 18 yo
- Consent for participation
- Affiliated to a social security regime
Who cannot join
- Patients with medically non-stable NMD: fever, acute respiratory infection, organ decompensation
- Patients with significant cognitive impairment (inability to read and/or incomprehension of questions)
- Patients with fast-evolving neuromuscular disease
- Patients declining to participate in the study
- Patients under guardianship and/or trusteeship
- Pregnant women
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Assessment of Quality of Life and Its Determinants in Ventilator-dependent Patients With Slowly Progressive Neuromuscular Disease
This study aims at evaluating the quality of life of patients with slowly progressive neuromuscular disorders who are dependant on mechanical ventilation (daily usage ≥ 16h).
- Conditions
- Neuromuscular Diseases, Respiration, Artificial, Tracheostomy, Noninvasive Ventilation, Quality of Life
- Medical subject headings
- Neuromuscular Diseases, Respiratory Aspiration
What is being measured
The primary outcome is the main question the study is designed to answer.
Score of the Severe Respiratory Insufficiency questionnaire (SRI)
Higher score represents better quality of life
Measured: 1 Day
Score of the Quality of Life Measure for people with slowly progressive and genetic neuromuscular disease questionnaire (QoL-gNMD)
Higher score represents better quality of life
Measured: 1 Day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Dec 2020
- Study start2 Dec 2020
- Primary completion7 Dec 2021
- Study completion7 Dec 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromuscular DiseasesStudies of the same condition, open and under way.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 119 participants (actual)
Sponsor and investigators
- Lead sponsor
- Centre d'Investigation Clinique et Technologique 805
- Sponsor type
- University, hospital or other
- Collaborators
- Fondation Garches, AFM Téléthon, FilNeMus
- Responsible party
- Sponsor
- Investigators
- Mathieu Delorme, MSc, Principal investigator, Université Paris-Saclay, UVSQ, ERPHAN, 78000, Versailles, FRANCEFrederic Lofaso, MD, PhD, Study director, Université Paris-Saclay, UVSQ, ERPHAN, 78000, Versailles, FRANCE
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04652401
- Sponsor's protocol ID
- 2020-A00997-32
- Acronym
- EqualVENT
- Status last verified
- Feb 2022
- First submitted
- 9 Nov 2020
- Last updated
- 9 Feb 2022
- Results posted
- No
- Data monitoring committee
- Yes
- 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 9 Feb 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment of Quality of Life and Its Determinants in Ventilator-dependent Patients With Slowly Progressive Neuromuscular Disease
- ClinicalTrials.gov ID
- NCT04652401
- Acronym / sponsor ID
- EqualVENT / 2020-A00997-32
- Conditions
- Neuromuscular Diseases, Respiration, Artificial, Tracheostomy, Noninvasive Ventilation, Quality of Life
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 119 participants (actual)
- Lead sponsor
- Centre d'Investigation Clinique et Technologique 805University, hospital or other
- Collaborators
- Fondation Garches, AFM Téléthon, FilNeMus
- Study start
- 2 Dec 2020 (actual)
- Primary completion
- 7 Dec 2021 (actual)
- Study completion
- 7 Dec 2021 (actual)
- First posted
- 3 Dec 2020 (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.
This study aims at evaluating the quality of life of patients with slowly progressive neuromuscular disorders who are dependant on mechanical ventilation (daily usage ≥ 16h).
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with slowly progressive neuromuscular disease using home mechanical ventilation for at least 16 hours a day since at least one year. Patients may be ventilated via invasive or noninvasive interface.
Key inclusion criteria (5 listed)
- Patients with slowly progressive neuromuscular disorder (NMD), non walking
- Patients dependant on mechanical ventilation (daily usage ≥ 16h) for at least 1 year before inclusion
- Aged ≥ 18 yo
- Consent for participation
- Affiliated to a social security regime
Key exclusion criteria (6 listed)
- Patients with medically non-stable NMD: fever, acute respiratory infection, organ decompensation
- Patients with significant cognitive impairment (inability to read and/or incomprehension of questions)
- Patients with fast-evolving neuromuscular disease
- Patients declining to participate in the study
- Patients under guardianship and/or trusteeship
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Score of the Severe Respiratory Insufficiency questionnaire (SRI)
Time frame: 1 Day
Higher score represents better quality of life
Score of the Quality of Life Measure for people with slowly progressive and genetic neuromuscular disease questionnaire (QoL-gNMD)
Time frame: 1 Day
Higher score represents better quality of life
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Feb 2022.
- 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.
Mathieu Delorme, MSc principal investigator
Université Paris-Saclay, UVSQ, ERPHAN, 78000, Versailles, FRANCE
Frederic Lofaso, MD, PhD study director
Université Paris-Saclay, UVSQ, ERPHAN, 78000, Versailles, FRANCE
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Neuromuscular Diseases and Respiration, Artificial, run by Centre d'Investigation Clinique et Technologique 805 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 5 inclusion and 6 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: finished, as of the sponsor's last update on 9 Feb 2022. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Score of the Severe Respiratory Insufficiency questionnaire (SRI) (time frame: 1 Day). The study lists 2 primary outcomes in all. 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 9 Feb 2022.