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
Newcastle-upon-Tyne Hospitals NHS Trust
600 participants
Jul 29, 2022
OBSERVATIONAL
Conditions
Summary
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).
Eligibility
Inclusion Criteria3
- 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 Criteria4
- Non-5q SMA
- No genetic confirmation
- \<16 years of age
- No signed informed consent or consent withdrawn
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Interventions
Risdiplam
Nusinersen
Locations(18)
View Full Details on ClinicalTrials.gov
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NCT06978985