MedTrace Logo

Personalized Medicine for SMA: a Translational Project

NCT05779956 · Status: UNKNOWN · Type: OBSERVATIONAL · Enrollment: 90

Last updated 2023-03-22

No results posted yet for this study

Summary

Major breakthroughs in the treatment for Spinal muscular atrophy (SMA) have been recently achieved with various therapeutic approaches that increase full-length SMN protein levels. The variability observed following the advent of commercial availability of Nusinersen for all types of SMA has highlighted the need to identify tools that may allow to predict possible therapeutic responses. The aim of this project is to establish whether an integrated approach using clinical, imaging (muscle MRI) and circulating biomarkers, can provide the possibility to develop a predictive model of therapeutic response to novel therapies for SMA patients. More specifically we wish to establish the correlation between clinical response, different biomarkers indicative of central nervous system efficacy (e.g. determination of neurofilaments levels), and markers that provide evidence of the skeletal muscle response (e.g. serum myostatin and muscle imaging) in different types of SMA

Conditions

Sponsors & Collaborators

  • University of Milan

    collaborator OTHER

  • Ospedale Pediatrico Bambino Gesu

    collaborator UNKNOWN

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    lead OTHER

Principal Investigators

  • Eugenio Mercuri, MD · F Policlinico Gemelli IRCCS

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-09-01
Primary Completion
2024-08-31
Completion
2024-08-31

Countries

  • Italy

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05779956 on ClinicalTrials.gov