Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms)

Plain-language summary

This study examines the functional effects of Nusinersen treatment on motor abilities in adults with 5q-spinal muscular atrophy Type 2 or Type 3. It enrolled adults over 18 who had an indication for Nusinersen and agreed to the treatment. The study was conducted as a non-phase study and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Not Applicable

Status

Completed

Sponsor

CHU de Reims

Intervention

Monthly assessments of functional motor abilities by a trained therapist, Nusinersen

Start date

1/24/2022

Estimated completion

9/8/2023

Countries

France

Related genes

Locations

• Reims, France

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

inclusion criteria :

* Adults (over 18 years of age)
* 5q-SMA type 2 or 3
* with indication for nusinersen treatment by the physician of the center of reference and competence for neuromuscular diseases
* accepting treatment by nusinersen
* Agreeing to participate in the study (signature of the informed consent form).
* living within a radius of 40 km of the investigation center (for logistical reasons related to the conduct of assessments in the patient's home).
* affiliated to a social security system.

exclusion criteria :

* minors (less than 18 years of age)
* with a contra-indication to the nusinersen: pregnancy, breast feeding, hypersensitivity to the nusinersen
* with a contraindication to lumbar puncture: hemostasis disorder, intracerebral mass
* benefiting from another gene therapy drug to treat spinal muscular atrophy.