Spinal muscular atrophy trials in United Kingdom

In United Kingdom 12 Also abroad 68

12 trials · From ClinicalTrials.gov, checked 12 November 2026. Ask the study team whether a trial fits you.

No phaseTaking people
Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
Where
Sheffield
Age
3 years and over
People
90 worldwide
Run by
Alcyone Therapeutics, Inc
See details and how to joinNCT05866419
No phaseTaking people
An International Federated Model for Wearable-derived Remote Longitudinal Motor Monitoring in Young Children With Spinal Muscular Atrophy Compared With Healthy Controls: Active-NBS Study (UK)
Where
Oxford
Age
Up to 4 years
People
90
Run by
University of Oxford
See details and how to joinNCT07543003
No phaseTaking people
Service Evaluation for Newborn Screening for Spinal Muscular Atrophy
Where
London, Oxford, Warwick
Age
5 days to 5 years
People
750,000
Run by
University of Oxford
See details and how to joinNCT07888868
Phase 2Taking people
A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy
Where
Birmingham, Cambridge, Glasgow, Leeds, London, Oxford, Sheffield
Age
5 years to 17 years
People
60 worldwide
Run by
argenx
See details and how to joinNCT07287982 · EU 2025-523496-32-00
Phase 2Taking people
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
Where
Leeds
Age
Up to 2 years
People
52 worldwide
Run by
Scholar Rock, Inc.
See details and how to joinNCT07047144
Phase 1Taking people
A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)
Where
Sheffield
Age
3 years and over
People
78 worldwide
Run by
Biogen
See details and how to joinNCT06555419 · EU 2024-514239-21-00
No phaseTaking people
UK SMA Patient Registry
Where
Newcastle upon Tyne
People
800
Run by
Newcastle University
See details and how to joinNCT04292574
No phaseNot yet taking people in United Kingdom
Spinal Muscular Atrophy and School Transitions
Where
Oswestry
Age
Up to 25 years
People
20
Run by
Robert Jones and Agnes Hunt Orthopaedic and District NHS Trust
See details and how to joinNCT07720518
No phaseNot yet taking people in United Kingdom
Exploring Lived Experiences of Families of Children With Spinal Muscular Atrophy(SMA) Type 1 Regarding Feeding and Communication
Where
London
Age
16 years and over
People
15
Run by
Guy's and St Thomas' NHS Foundation Trust
See details and how to joinNCT07596277
No phaseNot yet taking people in United Kingdom
Adrenal& Bone Complications in Paediatric Patients Living With Spinal Muscular Atrophy (SMA)
Where
Sheffield
Age
0 years and over
People
550
Run by
Sheffield Children's NHS Foundation Trust
See details and how to joinNCT07530926
No phaseTaking people
A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies
Where
Birmingham, Bristol, London, Newcastle upon Tyne, Nottingham, Oswestry, Salford, Sheffield, Southampton
Age
18 years to 54 years
People
20 worldwide
Run by
Biogen
See details and how to joinNCT05789758
No phaseTaking people
Adult SMA Research and Clinical Hub
Where
Birmingham, Bristol, Cambridge, Cardiff, Glasgow, Leeds, Liverpool, London, Newcastle upon Tyne, Nottingham, Oswestry, Oxford, Salford, Sheffield, Southampton, Swansea
Age
16 years and over
People
600
Run by
Newcastle-upon-Tyne Hospitals NHS Trust
See details and how to joinNCT06978985