Quality of Life and Participation of the Adult With Spinal Muscular Atrophy in France (QOLSMA)

October 24, 2023 updated by: Hospices Civils de Lyon

Specificities of Quality of Life and Influence of Participation on the Quality of Life of the Adult With Spinal Muscular Atrophy in France: a Cross-sectional Study

Spinal muscular atrophy is a hereditary motorneuron disease caused by a mutation of the SMN1 gene, which is at the origin of a progressive limb and axial motor deficiency. It concerns 1200 individuals in France, including 700 adults in 2018. The main objective of this study is to assess the quality of life of SMA patients in France.

The secondary objectives are, in one hand, to compare the quality of life of SMA patients to a population of neuromuscular diseases patients. And on the other hand to evaluate the determinants of participation and the impact of participation on quality of life in adult SMA patients.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Estimated)

175

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • France
      • Bron, France, 69500
        • Not yet recruiting
        • Service L'Escale - Médecine Physique et de Réadaptation Pédiatrique Groupement Hospitalier Est des Hospices Civils de Lyon
        • Contact:
          • VUILLEROT Carole, Pr
      • Bron, France, 69500
        • Recruiting
        • VUILLEROT Carole
        • Contact:
          • VUILLEROT Carole, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 85 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Probability Sample

Study Population

Adult patients with spinal muscular atrophy in France. And adult NMD patients from the study of Dany et al "Construction of a Quality of Life Questionnaire for slowly progressive neuromuscular disease" (2015)

Description

Inclusion Criteria:

  • Patient with SMA type 1, 2, 3 or 4
  • ≥ 18 years old
  • giving informed consent to participate to the study
  • patients from the study of Dany et al "Construction of a Quality of Life Questionnaire for slowly progressive neuromuscular disease" (2015)

Exclusion Criteria:

  • patients who do not complete ≥ 80% of the questionnaire)-

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
SMA adult patients
Other: SMA adult patients
An online questionnaire aimed at collecting demographic and social data, and data concerning activity limitations, participation and quality of life of SMA patients from validated scales : QOLNMD, Rosenberg.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Quality of life
Time Frame: At inclusion
Quality of life of adult patients with spinal muscular atrophy in France, assessed using the QOLNMD score
At inclusion

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Hospices Civils de Lyon

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 19, 2022

Primary Completion (Estimated)

October 19, 2024

Study Completion (Estimated)

October 19, 2024

Study Registration Dates

First Submitted

May 4, 2022

First Submitted That Met QC Criteria

May 4, 2022

First Posted (Actual)

May 9, 2022

Study Record Updates

Last Update Posted (Actual)

October 25, 2023

Last Update Submitted That Met QC Criteria

October 24, 2023

Last Verified

October 1, 2022

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 69HCL22_0200

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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