Review of outcomes for children with spinal muscular atrophy in the West Midlands from 2017 to 2022
A Review of the Management and Outcomes of Children With SMA in the West Midlands During 2017-2022
This study looks at how children with spinal muscular atrophy in the West Midlands have been treated and what their health outcomes have been like over the past five years, especially after new therapies were introduced.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 30 (estimated) |
| Ages | 0 Years to 16 Years |
| Sex | All |
| Sponsor | Robert Jones and Agnes Hunt Orthopaedic and District NHS Trust Government |
| Locations | 2 sites (Birmingham, Shropshire and 1 other locations) |
| Trial ID | NCT05994950 on ClinicalTrials.gov |
What this trial studies
This observational study reviews the management and outcomes of children diagnosed with spinal muscular atrophy (SMA) in the West Midlands over a five-year period. It focuses on children aged 0-16 years who were under the care of specific neuromuscular services and had at least two clinical reviews during the study timeframe. The research aims to assess how the introduction of new therapies has impacted patient outcomes, particularly in the context of changing treatment paradigms and disease phenotypes. By analyzing retrospective data, the study seeks to provide insights into the evolving clinical landscape for SMA management.
Who should consider this trial
Good fit: Ideal candidates for this study are children aged 0-16 years with genetically confirmed 5q spinal muscular atrophy who were treated at designated healthcare facilities during the study period.
Not a fit: Patients aged 16 years or older as of July 1, 2017, or those with non-5q SMA may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could enhance understanding of treatment outcomes and inform future management strategies for children with SMA.
How similar studies have performed: While this study is observational and retrospective, similar studies have shown success in understanding treatment impacts in SMA, making this approach relevant and potentially beneficial.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Patient aged \<16 years old as of 1st July 2017. (16 years old is the age in which patients typically start their transition process to adulthood - retrospective data collection will stop at the date in which the patient turned 16 years old, if this is before 30th June 2022). * OR patient was born between 1st July 2017 - 30th June 2022. * Genetically confirmed 5q SMA. * Patients must have been under the care of the named Key Collaborative Site and Neuromuscular Service for their SMA anytime during 1st July 2017- 30th June 2022 and must have had at least two clinical reviews during this time. * Deceased patients can be reviewed, as long as they met the eligibility criteria before their date of death. Exclusion Criteria: * Aged ≥16 years as of 1st July 2017. * Genetically confirmed as having non-5q SMA or have no genetic confirmation of their diagnosis. * Patient was not under the care of the named Key Collaborative Site and Neuromuscular Service for their SMA specialist care anytime during 1st July 2017 - 30th June 2022.
Where this trial is running
Birmingham, Shropshire and 1 other locations
- Birmingham Heartlands Hospital — Birmingham, Shropshire, United Kingdom (Recruiting)
- The Robert Jones and Agnes Hunt Orthopaedic Hospital — Oswestry, Shropshire, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Professor Tracey Willis
- Email: tracey.willis1@nhs.net
- Phone: 01691404047
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.