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NCT05366465 | Recruiting | Spinal Muscular Atrophy


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

Civil Hospices of Lyon

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

Spinal Muscular Atrophy

Intervention/treatment

SMA adult patients

Study Type : Observational
Estimated Enrollment : 175 participants
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
Actual Study Start Date : October 19, 2022
Estimated Primary Completion Date : October 19, 2024
Estimated Study Completion Date : October 19, 2024

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 18 Years to 85 Years
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
Criteria
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)-

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

Location Details


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

How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations


Not yet recruiting

France,

L'Escale Service - Physical Medicine and Pediatric Rehabilitation Eastern Hospital Group of Hospices Civils de Lyon

Bron, France, 69500

Recruiting

France,

VUILLEROT Carole

Bron, France, 69500

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