Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
600 participants
OBSERVATIONAL
2022-07-29
2027-04-30
Brief Summary
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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).
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Detailed Description
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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.
Conditions
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Study Design
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COHORT
OTHER
Study Groups
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Participants with Spinal Muscular Atrophy
Participants over 16 years old with a genetically confirmed diagnosis of 5q SMA.
Risdiplam
Risdiplam
Nusinersen Injectable Product
Nusinersen
Interventions
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Risdiplam
Risdiplam
Nusinersen Injectable Product
Nusinersen
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Genetically confirmed diagnosis of 5q SMA
* Signed informed consent to take part in the study
Exclusion Criteria
* No genetic confirmation
* \<16 years of age
* No signed informed consent or consent withdrawn
16 Years
ALL
No
Sponsors
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Newcastle University
OTHER
John Walton Muscular Dystrophy Research Centre
UNKNOWN
Biogen
INDUSTRY
Roche Products Limited
UNKNOWN
Newcastle-upon-Tyne Hospitals NHS Trust
OTHER
Responsible Party
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Chiara Marini Bettolo
Consultant Neurologist and Clinical Lead
Principal Investigators
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Prof Chiara Marini-Bettolo, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
Newcastle-upon-Tyne Hospitals NHS Trust
Locations
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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
Countries
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Central Contacts
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Facility Contacts
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Role: primary
Role: primary
Role: primary
Role: primary
Role: primary
Role: primary
Role: primary
Role: primary
Role: primary
Role: primary
Role: primary
Role: primary
Role: primary
Role: primary
Related Links
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Related Info
Other Identifiers
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9832
Identifier Type: -
Identifier Source: org_study_id
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