MedTrace Logo

A Review of the Management and Outcomes of Children With SMA in the West Midlands During 2017-2022

NCT05994950 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 30

Last updated 2024-04-12

No results posted yet for this study

Summary

Spinal Muscular Atrophy (SMA) is a rare neuromuscular condition, characterised by loss of motor neurons as a result of a mutation in the survival motor neuron gene. This results in muscle wasting and in the most common and severe type, death before 24 months. Over the recent years there has been a dynamic shift in the therapeutic options for these patients involving both approved therapies, including gene therapy, and access to clinical trials in genetic modifying.

As a result of this mortality and morbidity have changed particularly for the SMA type 1 population and therefore there is now a changing phenotype with many children needing interventions at different time points compared to the natural history. This review process is a retrospective review from 1st July 2017 - 30th June 2022, when most of the new drug therapies were being introduced, of all the children aged from 0-16 years in the West Midlands region and their outcomes.

Conditions

Sponsors & Collaborators

  • University Hospital Birmingham NHS Foundation Trust

    collaborator OTHER

  • Robert Jones and Agnes Hunt Orthopaedic and District NHS Trust

    lead OTHER_GOV

Eligibility

Min Age
0 Years
Max Age
16 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-12-06
Primary Completion
2024-12-31
Completion
2024-12-31

Countries

  • United Kingdom

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 NCT05994950 on ClinicalTrials.gov