At a glance
- What the study gets you
- Health checks and monitoring — no treatment given
- Type of study
- Observational (no treatment given)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Not specified by the sponsor
- How long the study runs
- Study runs about 38 months (dates as stated)
- About the drug or intervention
- Not specified by the sponsor
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Observing health over time
- Ages
- Up to 4 Years
- Who
- All
- Number of participants
- 90
- Started
- 2026-05-01
- Last checked
- 2025-08
Plain English Summary
What is this study?
- • Testing a new treatment for spinal muscular atrophy (sma)
- • Clinical study - 90 participants
- • Active-NBS is a study to evaluate the muscle development of patients with spinal muscular atrophy (SMA) who are diagnosed at birth
Who can take part?
- • Ages Up to 4 Years
- • Diagnosed with spinal muscular atrophy (sma)
Where?
- • Oxford - University of Oxford
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
Active-NBS is a study to evaluate the muscle development of patients with spinal muscular atrophy (SMA) who are diagnosed at birth. Medicines have become available in the last decade, and many patients are treated very early. Treatments are most effective if used before the patient develops symptoms. However, some patients may show symptoms by the time they receive treatment. This means that even with early diagnosis, they might still develop muscle weakness despite treatment. The investigators want to see when the movements of patients diagnosed at birth differ from normal development. This information will help identify the best time to give additional medicines currently being developed to support the muscle. The investigators will track the progress of up to 60 patients over a maximum of 30 months using wearable technologies which are worn at home. The investigators aim to validate their outcomes for use in this age group. The wearable devices are called Syde and Motor Assessment of an Infant in a Jumpsuit (MAIJU). They will be worn at regular intervals during the study and will not involve extra hospital visits for patients. The study will also recruit up to 30 healthy control participants and follow them for up to 30 months. This will help define normal development with use of the Syde device. Active-NBS will be conducted in the UK and internationally using a federated data model. Collaborative sites will collect harmonised data in accordance with the Active-NBS protocol, with data integration and oversight managed by the University of Oxford. International sites may contact the Oxford study team to establish collaboration.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: Up to 4 Years
- Who can join: All genders
What the study is looking for
- ✓Inclusion criteria (Test cohort):
- ✓Genetically confirmed SMA and number of SMN2 copies available
- ✓a. Patients identified by NBS and treated with disease modifying therapy (DMT)
- ✓(2)a,i 4 copies or more of SMN2 and not treated with DMT
- ✓(2)a,ii less than 4 copies of SMN2 and not treated with DMT
See the full criteria
Where Is This Study? (1 UK site)
University of Oxford
Oxford OX3 9DU, United Kingdom
How to Get in Touch
Charlotte Lilien
Sponsor contactCONTACT
Active-NBS Joint Mailbox
Sponsor contactCONTACT