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

Sponsor

Hospices Civils de Lyon (Other)

Overall Status

Not yet recruiting

CT.gov ID

NCT05366465

Collaborator

(none)

175

Enrollment

Location

Anticipated Duration (Months)

12.5

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Spinal Muscular Atrophy	Other: SMA adult patients

Study Design

Study Type:

Observational

Anticipated Enrollment :

175 participants

Observational Model:

Cohort

Time Perspective:

Cross-Sectional

Official Title:

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

Anticipated Study Start Date :

Jun 1, 2022

Anticipated Primary Completion Date :

Jun 1, 2023

Anticipated Study Completion Date :

Aug 1, 2023

Arms and Interventions

Arm	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.

Arm

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.

Outcome Measures

Primary Outcome Measures

Quality of life [At inclusion]
Quality of life of adult patients with spinal muscular atrophy in France, assessed using the QOLNMD score

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 85 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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)-

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Service L'Escale - Médecine Physique et de Réadaptation Pédiatrique Groupement Hospitalier Est des Hospices Civils de Lyon	Bron		France	69500

Sponsors and Collaborators

Hospices Civils de Lyon

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Hospices Civils de Lyon

ClinicalTrials.gov Identifier:

NCT05366465

Other Study ID Numbers:

69HCL22_0200

First Posted:

May 9, 2022

Last Update Posted:

May 9, 2022

Last Verified:

May 1, 2022

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Muscular Atrophy

Muscular Atrophy, Spinal

Atrophy

Study Results

No Results Posted as of May 9, 2022