Spinal muscular atrophy trials in United Kingdom
12 trials · From ClinicalTrials.gov, checked 10 Nov 2026. Ask the study team whether a trial fits you.
No phaseRecruitingResearch participation
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 joinNCT05866419Registry updated 2026-11-09
No phaseRecruitingResearch participation
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 joinNCT07543003Registry updated 2026-11-02
No phaseRecruitingResearch participation
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 joinNCT07888868Registry updated 2026-10-22
Phase 2RecruitingTrial-stage medicine
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 joinNCT07287982EU 2025-523496-32-00Registry updated 2026-10-08
Phase 2RecruitingTrial-stage medicine
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 joinNCT07047144Registry updated 2026-10-02
Phase 1RecruitingTrial-stage medicine
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 joinNCT06555419EU 2024-514239-21-00Registry updated 2026-09-03
No phaseRecruitingResearch participation
UK SMA Patient Registry
- Where
- Newcastle upon Tyne
- People
- 800
- Run by
- Newcastle University
See details and how to joinNCT04292574Registry updated 2026-08-20
No phaseNot yet recruitingResearch participation
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 joinNCT07720518Registry updated 2026-07-22
No phaseNot yet recruitingResearch participation
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 joinNCT07596277Registry updated 2026-06-02
No phaseNot yet recruitingResearch participation
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 joinNCT07530926Registry updated 2026-04-24
No phaseRecruitingResearch participation
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 joinNCT05789758Registry updated 2026-03-09
No phaseRecruitingResearch participation
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 joinNCT06978985Registry updated 2025-05-18
All Spinal muscular atrophy trials and the map
This page is not medical advice. Talk to your doctor before contacting a trial.