Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative Therapies
This study is looking for a new and more accurate way to measure how well babies with Spinal Muscular Atrophy (SMA) are moving, especially those who are having innovative new treatments. SMA is a serious condition that affects movement. While new therapies are helping these babies survive, doctors need better ways to understand how much these treatments are improving their movement and day-to-day abilities. Current methods for checking movement can be general. This study will explore using small, wearable sensors to detect and track movement. The goal is to see if these sensors can provide a clearer and more sensitive picture of a baby's motor development, helping doctors better understand the effects of these important new treatments.
At a glance
What is this study about?
Spinal Muscular Atrophy (SMA) is a serious illness that affects children, making it hard for them to move their muscles. It's actually one of the most common genetic illnesses that can be life-threatening for babies. In the most severe cases, it can even cause breathing problems.
Recently, exciting new treatments like gene therapy or special medications have become available. These treatments have been very successful in helping babies with SMA live longer. However, doctors want to understand more precisely how much these treatments are improving the children's ability to move and develop. Currently, the ways doctors measure movement are a bit general and might not show all the small improvements.
This is where our study comes in. We're looking at using small, wearable sensors, similar to fitness trackers, which can be placed on a baby. These sensors are safe and easy to use. They can help us measure a baby's movements in a much more detailed way than traditional methods. By doing this, we hope to create a new, clearer method to see how much these innovative therapies are helping babies with SMA move better and grow stronger.
Key takeaways
- Aims to improve how doctors measure movement in babies with SMA.
- Uses safe, wearable sensors to track movement precisely.
- Helps understand how well new SMA treatments are working.
- For babies with SMA up to 2 years old, receiving innovative therapies.
- Does not involve new treatments or medications.
- Participation helps future children with SMA.
Who may be eligible?
This study is for babies up to 2 years old, both boys and girls, who have been diagnosed with Spinal Muscular Atrophy (SMA). They must have been diagnosed through a genetic test confirming a problem with the SMN1 gene. The babies should also be receiving their care at the Necker Neuromuscular Reference Center (GNMH) and have been approved for one of the new, innovative treatments for SMA (like gene therapy or certain medications) by a national team of experts.
To be eligible for these new treatments (and therefore this study), their SMA symptoms must have started before they were 1 year old. They also shouldn't have severe breathing problems that require a breathing machine for most of the day, or other serious health issues that affect their ability to swallow.
There are also some reasons why a baby might not be able to join the study. For example, if either parent doesn't agree, if the baby has unstable breathing or heart problems, if they can't have the innovative therapy for any medical reason, or if they have another health condition that affects their movement (like difficulties during birth).
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Is my baby diagnosed with SMA by a genetic test?
- Is my baby receiving care at the Necker Neuromuscular Reference Center?
- Has my baby been approved for an innovative SMA therapy?
- Did my baby's SMA symptoms start before they were 1 year old?
- Does my baby have severe breathing problems needing a machine for most of the day?
- Do both parents agree to participate in the study?
What does participation involve?
This study focuses on observation and measurement, not on giving new treatments. Your baby would continue to receive their innovative therapy for SMA as decided by their doctors. The main part of taking part would involve having small, wearable sensors placed on your baby. These sensors would then record their movements. The study aims to develop a new method, so it doesn't involve changes to your baby's current treatment plan, additional medications, or extra hospital visits beyond their usual care. The total duration of participation isn't specified in detail but would likely align with the period needed to gather sufficient movement data from the sensors.
Potential risks and benefits
Locations (1)
- Hôpital Necker-Enfants MaladesVerified postcodeParis, France
Common questions
What is Spinal Muscular Atrophy (SMA)?
SMA is a genetic condition that makes muscles weak and causes problems with movement. It can affect how babies crawl, walk, breathe, and swallow.
What are 'innovative therapies'?
These are the new, advanced treatments for SMA, like gene therapy or certain medications, that have been shown to help babies with the condition.
What are 'inertial sensors'?
These are small, wearable devices, like tiny motion detectors, that can track and measure a baby's movements very precisely.
Will my baby get extra treatment or medicine in this study?
No, this study is about measuring movement. Your baby will continue to receive their regular SMA treatment as planned by their doctors.
Is using the sensors safe?
Yes, the sensors are designed to be safe, non-invasive (nothing goes into the body), and easy to use on babies.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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