NCT07543003 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)
| NCT ID | NCT07543003 |
| Status | Recruiting |
| Phase | — |
| Sponsor | University of Oxford |
| Condition | Spinal Muscular Atrophy (SMA) |
| Study Type | OBSERVATIONAL |
| Enrollment | 90 participants |
| Start Date | 2026-05-01 |
| Primary Completion | 2029-07 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 90 participants in total. It began in 2026-05-01 with a primary completion date of 2029-07.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
Inclusion criteria (Test cohort): 1. Genetically confirmed SMA and number of SMN2 copies available 2. 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 (5)Male or female Exclusion Criteria (Test cohort): 1. Any acute or chronic condition which, according to the investigator, significantly interferes with the assessments and/or the motor evolution 2. Currently enrolled in an experimental treatment study Inclusion criteria (Control): 1. Typically developing child 2. Participant between 6 months and 4 years at inclusion 3. Parent(s)/legal guardian(s) able to provide written informed consent prior to the participation in the study 4. Male or female Exclusion criteria (Control): (1)Any acute or chronic condition which, according to the investigator, significantly interferes with the assessments and/or the motor evolution
Contact & Investigator
Frequently Asked Questions
Who can join the NCT07543003 clinical trial?
This trial is open to participants of all sexes, up to 4 Years, studying Spinal Muscular Atrophy (SMA). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT07543003 currently recruiting?
Yes, NCT07543003 is actively recruiting participants. Contact the research team at charlotte.lilien@paediatrics.ox.ac.uk for enrollment information.
Where is the NCT07543003 trial being conducted?
This trial is being conducted at Oxford, United Kingdom.
Who is sponsoring the NCT07543003 clinical trial?
NCT07543003 is sponsored by University of Oxford. The trial plans to enroll 90 participants.