At a glance
- What the study gets you
- Health checks and monitoring — no treatment given
- Type of study
- Observational (no treatment given)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Risdiplam (drug), Nusinersen Injectable Product (drug)
- How long the study runs
- Study runs about 57 months (dates as stated)
- About the drug or intervention
- Risdiplam — drug: Risdiplam · Nusinersen Injectable Product — drug: Nusinersen
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Observing health over time
- Ages
- 16 Years and over
- Who
- All
- Number of participants
- 600
- Started
- 2022-07-29
- Last checked
- 2025-05
Plain English Summary
What is this study?
- • Testing a new treatment for spinal muscular atrophy (sma)
- • Clinical study - 600 participants
- • Adult SMA REACH is a data collection study aiming to gain a better understanding of the impact of standards of care and new treatments on the natural history of Spinal Muscular Atrophy (SMA)
Who can take part?
- • Ages 16 Years and over
- • Diagnosed with spinal muscular atrophy (sma)
Where?
- • Birmingham - University Hospitals Birmingham NHS Foundation Trust
- • Bristol - North Bristol NHS Trust
- • Cambridge - Cambridge University Hospitals NHS Foundation Trust
- • Cardiff - Cardiff and Vale University Health Board
- • +14 more UK sites
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
Adult SMA REACH is a data collection study aiming to gain a better understanding of the impact of standards of care and new treatments on the natural history of Spinal Muscular Atrophy (SMA). This study is sponsored by The Newcastle upon Tyne Hospitals NHS Foundation Trust. Adult SMA REACH is funded by Biogen and Roche. Currently, there are three drug treatments available for SMA in the UK: Zolgensma, Nusinersen and Risdiplam. Zolgensma is the only approved drug - Nusinersen and Risdiplam are currently available as part of Managed Access Agreements (MAA).
More detail
Spinal muscular atrophy (SMA) is a genetic motor neuron disease with a broad spectrum of severity, affecting both infants and adults. Advances in treatment, including Nusinersen (Spinraza), onasemnogene abeparvovec (Zolgensma), and Risdiplam (Evrysdi), have significantly improved patient outcomes, highlighting the need for stronger clinical networks to monitor the long-term effects of these therapies. The Adult SMA REACH Study builds upon the success of SMA REACH UK, which has been instrumental in collecting natural history and treatment data for paediatric SMA patients. The study benefits from collaboration with TREAT-NMD, the UK SMA Patient Registry, and iSMAC, aligning with international efforts to harmonise SMA data collection. By leveraging Newcastle University's experience in global SMA initiatives, Adult SMA REACH aims to enhance patient care, inform clinical decision-making, and contribute to future SMA research.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 16 Years and over
- Who can join: All genders
What the study is looking for
- ✓Age ≥16 years (when patients will begin the transition process to adulthood)
- ✓Genetically confirmed diagnosis of 5q SMA
- ✓Signed agreement to take part to take part in the study
Who cannot take part
- ✗Non-5q SMA
- ✗No genetic confirmation
- ✗\<16 years of age
- ✗No signed agreement to take part or consent withdrawn
See the full criteria
Where Is This Study? (18 UK sites)
University Hospitals Birmingham NHS Foundation Trust
Birmingham B15 2GW, United Kingdom
North Bristol NHS Trust
Bristol BS10 5NB, United Kingdom
Cambridge University Hospitals NHS Foundation Trust
Cambridge CB2 0QQ, United Kingdom
Cardiff and Vale University Health Board
Cardiff CF14 4XW, United Kingdom
NHS Greater Glasgow and Clyde
Glasgow G12 0XH, United Kingdom
Bradford Teaching Hospitals NHS Foundation Trust
Leeds BD9 6RJ, United Kingdom
The Walton Centre NHS Foundation Trust
Liverpool L9 7LJ, United Kingdom
University College London Hospitals NHS Foundation Trust
London NW1 2BU, United Kingdom
King's College Hospital NHS Foundation Trust
London SE5 9RS, United Kingdom
St George's University NHS Foundation Trust
London SW17 0QT, United Kingdom
Newcastle-upon-Tyne Hospitals NHS Trust
Newcastle upon Tyne NE1 3BZ, United Kingdom
Nottingham University Hospitals Trust
Nottingham NG5 1PB, United Kingdom
The Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust
Oswestry SY10 7AG, United Kingdom
Oxford University Hospitals Trust
Oxford OX3 9DU, United Kingdom
The Northern Care Alliance NHS Foundation Trust
Salford M6 8HD, United Kingdom
Sheffield Teaching Hospitals NHS Foundation Trust
Sheffield S10 2JF, United Kingdom
University Hospital Southampton NHS Foundation Trust
Southampton SO16 6YD, United Kingdom
South West Wales Neuromuscular Service in Swansea
Swansea SA6 6NL, United Kingdom
How to Get in Touch
Adult SMA REACH Team
Sponsor contactCONTACT