Quality of Life in Ventilator-dependant Neuromuscular Patients (EqualVENT)

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).

Study Overview

• Status: Completed
• Conditions: Quality of Life; Neuromuscular Diseases; Tracheostomy; Respiration, Artificial; Noninvasive Ventilation

• Study Type: Observational
• Enrollment (Actual): 119

Contacts and Locations

Study Locations

• France
  • Évry, France
    • AFM Téléthon

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

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.

Description

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Score of the Severe Respiratory Insufficiency questionnaire (SRI)	Higher score represents better quality of life	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	1 Day

Collaborators and Investigators

• Sponsor: Centre d'Investigation Clinique et Technologique 805
• Collaborators: Fondation Garches; AFM Telethon; FilNeMus
• Investigators: Principal Investigator: Mathieu Delorme, MSc, Université Paris-Saclay, UVSQ, ERPHAN, 78000, Versailles, FRANCE; Study Director: Frederic Lofaso, MD, PhD, Université Paris-Saclay, UVSQ, ERPHAN, 78000, Versailles, FRANCE

Study record dates

Study Major Dates

• Study Start (ACTUAL): December 2, 2020
• Primary Completion (ACTUAL): December 7, 2021
• Study Completion (ACTUAL): December 7, 2021

Study Registration Dates

• First Submitted: November 9, 2020
• First Submitted That Met QC Criteria: December 2, 2020
• First Posted (ACTUAL): December 3, 2020

Study Record Updates

• Last Update Posted (ACTUAL): February 9, 2022
• Last Update Submitted That Met QC Criteria: February 8, 2022
• Last Verified: February 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neuromuscular Diseases
• Other Study ID Numbers: 2020-A00997-32

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No