NCT07543003Recruiting
An International Federated Model for Wearable-derived Remote Longitudinal Motor Monitoring in Young Children With Spinal Muscular Atrophy Compared With Healthy Controls: Active-NBS Study (UK)
A Prospective, Longitudinal and Decentralised Study Investigating the Motor Development of Patients With Spinal Muscular Atrophy Identified by Newborn Screening Age 4 Years and Below: Active-NBS UK.
Source: ClinicalTrials.gov · Last updated 2 Nov 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 90 (estimated)
- First submitted
- 20 Aug 2025
- Start date
- 1 May 2026
- Primary completion date
- 31 Jul 2029
- Lead sponsor
- University of Oxford
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT07543003
What you commit to
- Study type: Observational
- Planned participants: 90
- From 1 May 2026 to the primary completion date, 31 Jul 2029
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- (1) Any acute or chronic condition which, according to the investigator, significantly interferes with the assessments and/or the motor evolution
- (1)Any acute or chronic condition which, according to the investigator, significantly interferes with the assessments and/or the motor evolution
Who the study is looking for
- 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
- or
- (2)b. Patients diagnosed due to a sibling or alternative means
- (2)b,i 4 copies or more of SMN2 and not treated with DMT
- (2)b,ii less than 4 copies of SMN2 and not treated with DMT
- (3)Patients between 4 months and below 4 years at baseline. Inclusion of patients can be before 4 months of age
- (4)Parent(s)/legal guardian(s) able to provide written informed consent prior to the patient's participation in the study
- Typically developing child
- Participant between 6 months and 4 years at baseline
- Parent(s)/legal guardian(s) able to provide written informed consent prior to the participation in the study
Age in the registry: - to 4 years · Sex: All · Accepts healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| University of Oxford Coordinator of this site? Confirm its status | Oxford | United Kingdom | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.