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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Total Enrollment

149 participants

Study Classification

OBSERVATIONAL

Study Start Date

2022-10-19

Study Completion Date

2024-02-17

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

European Registry of Patients With Infantile-onset Spinal Muscular Atrophy

NCT03339830

Spinal Muscular Atrophy

COMPLETED

MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy

NCT05769465

Spinal Muscular Atrophy

RECRUITING

Palliative Care in Spinal Muscular Atrophy (SMA) 1

NCT01862042

Spinal Muscular Atrophy 1

COMPLETED NA

French Register of Patients With Spinal Muscular Atrophy

NCT04177134

Spinal Muscular Atrophy

RECRUITING

Identification of a Biomarker Associated With Cis-duplication of the SMN1 Gene

NCT02550691

Spinal Muscular Atrophy

TERMINATED NA

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Spinal Muscular Atrophy

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

CROSS_SECTIONAL

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

SMA adult patients

Intervention Type OTHER

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.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

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.

Intervention Type OTHER

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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)