Clinical Trials Logo
  1. Home
  2. Spinal Muscular Atrophy
  3. NCT05366465
  4. Details
NCT05366465 Clinical Trial

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

Spinal Muscular Atrophy Clinical Trial

— QOLSMA  

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

Clinical Trial Details — Status: Recruiting

Administrative data
NCT number NCT05366465
Other study ID # 69HCL22_0200
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date October 19, 2022
Est. completion date October 19, 2024

Study information
Verified date October 2022
Source Hospices Civils de Lyon
Contact VUILLEROT Carole, Pr
Phone +334 72 12 95 04
Email carole.vuillerot@chu-lyon.fr
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial 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.

Recruitment information / eligibility
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)-

Study Design

Related Conditions & MeSH terms

Intervention

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.

Locations
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

Sponsors (1)
Lead Sponsor Collaborator
Hospices Civils de Lyon

Country where clinical trial is conducted

France, 

Outcome
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
See also
  Status Clinical Trial Phase
Completed NCT04851873 - Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) Phase 3
Completed NCT03223051 - Development of a Space Exploration Assessment for Children With Spinal Muscular Atrophy N/A
Completed NCT04335942 - Characterization of the Postural Habits of Wheelchair Users Analysis of the Acceptability of International Recommendations in the Prevention of Pressure Sores Risk by Using a Connected Textile Sensor N/A
Recruiting NCT05794139 - Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy Phase 2
Completed NCT02003937 - Aerobic Training in Patients With Spinal Muscular Atrophy Type III N/A
Not yet recruiting NCT00961103 - Motor Development and Orthoses in Spinal Muscular Atrophy (SMA) N/A
Completed NCT00227266 - Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy Phase 2
Completed NCT00374075 - Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy Phase 1
Enrolling by invitation NCT05539456 - Reliability and Validity of the Turkish Version of the PedsQL 3.0 Neuromuscular Module for 2-to 4- Year-old
Recruiting NCT05779956 - Personalized Medicine for SMA: a Translational Project
Recruiting NCT03217578 - Neonatal Spinal Muscular Atrophy (SMA) Screening
Recruiting NCT03300869 - Natural History of Types 2 and 3 SMA in Taiwan
Completed NCT01703988 - An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Phase 1/Phase 2
Withdrawn NCT02235090 - Study of Feasibility to Reliably Measure Functional Abilities' Changes in Nonambulant Neuromuscular Patients Without Trial Site Visiting N/A
Completed NCT02123186 - Newborn Screening for Spinal Muscular Atrophy N/A
Completed NCT00756821 - A Pilot Study of Biomarkers for Spinal Muscular Atrophy N/A
Completed NCT00004771 - Phase II Study of Leuprolide and Testosterone for Men With Kennedy's Disease or Other Motor Neuron Disease Phase 2
Recruiting NCT06310421 - Spinal Muscular Atrophy Neonatal Screening Program
Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Recruiting NCT05219487 - Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration