RECRUITINGOBSERVATIONAL
Adult SMA Research and Clinical Hub
Adult SMA REACH: Development and Implementation of a Standardised Data Set and Data Collection Research Study in the UK Adult SMA Population
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).
Who May Be Eligible (Plain English)
Who May Qualify:
- Age ≥16 years (when patients will begin the transition process to adulthood)
- Genetically confirmed diagnosis of 5q SMA
- Signed willing to sign a consent form to take part in the study
Who Should NOT Join This Trial:
- Non-5q SMA
- No genetic confirmation
- \<16 years of age
- No signed willing to sign a consent form or consent withdrawn
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
Inclusion Criteria:
* Age ≥16 years (when patients will begin the transition process to adulthood)
* Genetically confirmed diagnosis of 5q SMA
* Signed informed consent to take part in the study
Exclusion Criteria:
* Non-5q SMA
* No genetic confirmation
* \<16 years of age
* No signed informed consent or consent withdrawn
Treatments Being Tested
DRUG
Risdiplam
Risdiplam
DRUG
Nusinersen Injectable Product
Nusinersen
Locations (18)
University Hospitals Birmingham NHS Foundation Trust
Birmingham, United Kingdom
North Bristol NHS Trust
Bristol, United Kingdom
Cambridge University Hospitals NHS Foundation Trust
Cambridge, United Kingdom
Cardiff and Vale University Health Board
Cardiff, United Kingdom
NHS Greater Glasgow and Clyde
Glasgow, United Kingdom
Bradford Teaching Hospitals NHS Foundation Trust
Leeds, United Kingdom
The Walton Centre NHS Foundation Trust
Liverpool, United Kingdom
University College London Hospitals NHS Foundation Trust
London, United Kingdom
King's College Hospital NHS Foundation Trust
London, United Kingdom
St George's University NHS Foundation Trust
London, United Kingdom
Newcastle-upon-Tyne Hospitals NHS Trust
Newcastle upon Tyne, United Kingdom
Nottingham University Hospitals Trust
Nottingham, United Kingdom
The Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust
Oswestry, United Kingdom
Oxford University Hospitals Trust
Oxford, United Kingdom
The Northern Care Alliance NHS Foundation Trust
Salford, United Kingdom
Sheffield Teaching Hospitals NHS Foundation Trust
Sheffield, United Kingdom
University Hospital Southampton NHS Foundation Trust
Southampton, United Kingdom
South West Wales Neuromuscular Service in Swansea
Swansea, United Kingdom