NCT04652401

Brief 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).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
119

participants targeted

Target at P50-P75 for all trials

Timeline
Completed

Started Dec 2020

Shorter than P25 for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 9, 2020

Completed
23 days until next milestone

Study Start

First participant enrolled

December 2, 2020

Completed
1 day until next milestone

First Posted

Study publicly available on registry

December 3, 2020

Completed
1 year until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 7, 2021

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 7, 2021

Completed
Last Updated

February 9, 2022

Status Verified

February 1, 2022

Enrollment Period

1 year

First QC Date

November 9, 2020

Last Update Submit

February 8, 2022

Conditions

Neuromuscular DiseasesRespiration, ArtificialTracheostomyNoninvasive VentilationQuality of Life

Outcome Measures

Primary Outcomes (2)

  • 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

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-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.

You may qualify if:

  • Patients with slowly progressive neuromuscular disorder (NMD), non walking
  • Aged ≥ 18 yo
  • Consent for participation
  • Affiliated to a social security regime

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

AFM Téléthon

Évry, France

Location

MeSH Terms

Conditions

Neuromuscular DiseasesRespiratory Aspiration

Condition Hierarchy (Ancestors)

Nervous System DiseasesRespiration DisordersRespiratory Tract DiseasesPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Officials

  • Mathieu Delorme, MSc

    Université Paris-Saclay, UVSQ, ERPHAN, 78000, Versailles, FRANCE

    PRINCIPAL INVESTIGATOR

  • Frederic Lofaso, MD, PhD

    Université Paris-Saclay, UVSQ, ERPHAN, 78000, Versailles, FRANCE

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
CROSS SECTIONAL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 9, 2020

First Posted

December 3, 2020

Study Start

December 2, 2020

Primary Completion

December 7, 2021

Study Completion

December 7, 2021

Last Updated

February 9, 2022

Record last verified: 2022-02

Data Sharing

IPD Sharing
Will not share

Locations

FR(1)