Status:
RECRUITING
A Review of the Management and Outcomes of Children With SMA in the West Midlands During 2017-2022
Lead Sponsor:
Robert Jones and Agnes Hunt Orthopaedic and District NHS Trust
Collaborating Sponsors:
University Hospital Birmingham NHS Foundation Trust
Conditions:
Spinal Muscular Atrophy
Eligibility:
All Genders
Up to 16 years
Brief 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 ...
Detailed Description
Spinal muscular atrophy (SMA) is a rare neuromuscular disorder characterized by degeneration of alpha motor neurons in the spinal cord resulting in progressive muscle wasting, weakness, and paralysis,...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
December 6 2023
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
December 31 2024
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT05994950
Start Date
December 6 2023
End Date
December 31 2024
Last Update
April 12 2024
Active Locations (2)
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1
Birmingham Heartlands Hospital
Birmingham, Shropshire, United Kingdom, B9 5SS
2
The Robert Jones and Agnes Hunt Orthopaedic Hospital
Oswestry, Shropshire, United Kingdom, SY10 7AG