NCT05366465Completed
Quality of Life and Participation of the Adult with Spinal Muscular Atrophy in France
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
Source: ClinicalTrials.gov · Last updated 7 Mar 2025
Brief summary (registry, in English)
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 (registry)
- Completed
- Study type
- Observational
- Enrollment
- 149 (actual)
- First submitted
- 4 May 2022
- Start date
- 19 Oct 2022
- Primary completion date
- 17 Feb 2024
- Lead sponsor
- Hospices Civils de Lyon
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT05366465
What you commit to
- Study type: Observational
- Participants enrolled: 149
- From 19 Oct 2022 to the primary completion date, 17 Feb 2024
- 2 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other
- patients who do not complete ≥ 80% of the questionnaire)-
Who the study is looking for
- 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)
Age in the registry: 18 years to 85 years · Sex: All · Does not accept healthy volunteers
Sites (2)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| VUILLEROT Carole Coordinator of this site? Confirm its status | Bron | France | Unknown status |
| Service L'Escale - Médecine Physique et de Réadaptation Pédiatrique Groupement Hospitalier Est des Hospices Civils de Lyon Coordinator of this site? Confirm its status | Bron | France | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.