Wearable Technology Monitoring for Spinal Muscular Atrophy
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)
Recruiting
Observational Study
Spinal Muscular Atrophy (SMA)
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Healthy Volunteers Welcome
You do not need to have the condition being studied to take part.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
Ready to participate?
Review the details below, then apply to join this clinical trial.
At a Glance
Age
Up to 4
Sex
Any
Study type
Observational
Participants needed
90 (estimated)
Sponsor
University of Oxford · Other
Who this trial is looking for
This trial is looking for young children diagnosed with spinal muscular atrophy (SMA) and healthy children to track their muscle development. Participants will wear devices at home to help the researchers gather information over time.
Are You a Good Fit for This Trial?
You may be able to join if
I have been diagnosed with spinal muscular atrophy.
I am between 4 months and 4 years old.
My parent or guardian can give written consent.
I have a sibling with SMA or was diagnosed through newborn screening.
You may not be able to join if
I have a serious health condition that affects my movements.
I am currently in another treatment study.
Summarized in plain language from this trial's official eligibility criteria.
The full criteria are further down this page — only the research team can
confirm whether you qualify.
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
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…
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.
Trial Locations
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Status
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…
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
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