Spinal Muscular Atrophy Clinical Trial
— QOLSMAOfficial 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
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.
Status | Recruiting |
Enrollment | 175 |
Est. completion date | October 19, 2024 |
Est. primary completion date | October 19, 2024 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years to 85 Years |
Eligibility | 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)- |
Country | Name | City | State |
---|---|---|---|
France | Service L'Escale - Médecine Physique et de Réadaptation Pédiatrique Groupement Hospitalier Est des Hospices Civils de Lyon | Bron | |
France | VUILLEROT Carole | Bron |
Lead Sponsor | Collaborator |
---|---|
Hospices Civils de Lyon |
France,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Quality of life | Quality of life of adult patients with spinal muscular atrophy in France, assessed using the QOLNMD score | At inclusion |
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