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
This trial does not accept healthy volunteersQualifiers
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No signed informed consent or consent withdrawn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 population
Participants with SMA will volunteer to participate in this study. The study will be advertised through neuromuscular disease clinics, the registry website, patient organisations and conferences throughout the UK and Ireland.
Trial design
Design model
Cohort
Time perspective
Other
Treatments tested in this trial
Risdiplam
DrugRisdiplam
Nusinersen Injectable Product
DrugNusinersen
Treatment groups
600 Participants
are divided into 1 treatment groupGroup A: Participants with Spinal Muscular Atrophy2 interventions
Trial outcomes
Primary outcomes
1
World Health Organization (WHO) Motor Milestones
The WHO motor milestone assessment is a six-item checklist that dichotomously assesses whether a child can sit independently, crawl, stand with/without support, and walk with/without support. It is used in the Adult SMA population and in this study to categorise individuals as 'non-sitters', 'sitters' or 'walkers'. Changes in this score (loss or gain in function) and maintenance of score from baseline throughout treatment duration is assessed.
Time frame
Baseline (at treatment initiation) and 6 monthly through study completion2
Revised Upper Limb Module (RULM)
The RULM is designed to capture upper limb function and consists of 19 items, and is scored out of 37 points (higher scores indicating better function).
Time frame
Baseline (at treatment initiation) and 6 monthly through study completion3
6 Minute Walk Test (6MWT)
The 6MWT, which measures the distance a patient is able to walk within 6 min is used for only ambulant adult SMA patients in this study.
Time frame
Baseline (at treatment initiation) and 6 monthly through study completion4
The Hammersmith Functional Motor Scale Expanded (HFMSE)
The HFMSE consists of 33 items, with a maximum of 66 points (higher scores indicating better function).
Time frame
Baseline (at treatment initiation) and 6 monthly through study completionSponsors and contacts
Click on the lead sponsor to view all of their trials.
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