Adult SMA Research and Clinical Hub
ConditionSpinal Muscular Atrophy (SMA)
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age16+
SponsorNewcastle-upon-Tyne Hospitals NHS Trust
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).
Eligibility criteria
Qualifiers
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
Disqualifiers
Non-5q SMA
No genetic confirmation
<16 years of age
No signed informed consent or consent withdrawn
Trial design
Treatments tested in this trial
- Risdiplam
- Nusinersen Injectable Product
Treatment groups
600 Participants
are divided into 1 treatment groupSponsors and collaborators
Newcastle-upon-Tyne Hospitals NHS Trust
Lead sponsor
Newcastle University
Collaborator
John Walton Muscular Dystrophy Research Centre
Collaborator
Biogen
Collaborator
Roche Products Limited
Collaborator
Source ClinicalTrials.gov