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

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Brief Title

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

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

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.
    



Study Type

Observational


Primary Outcome

Quality of life


Condition

Spinal Muscular Atrophy

Intervention

SMA adult patients

Study Arms / Comparison Groups

 SMA adult patients
Description:  

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Other

Estimated Enrollment

175

Start Date

June 2022

Completion Date

August 2023

Primary Completion Date

June 2023

Eligibility 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)-
      

Gender

All

Ages

18 Years - 85 Years

Accepts Healthy Volunteers

No

Contacts

, +334 72 12 95 04, [email protected]

Location Countries

France

Location Countries

France

Administrative Informations


NCT ID

NCT05366465

Organization ID

69HCL22_0200


Responsible Party

Sponsor

Study Sponsor

Hospices Civils de Lyon


Study Sponsor

, , 


Verification Date

May 2022