Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
This study is checking on people who have Spinal Muscular Atrophy (SMA) and previously received the medicine called onasemnogene abeparvovec (OAV101) in another clinical trial. The main goal is to understand the long-term safety and how well this medicine works over time. Researchers will keep a close eye on participants for five years, checking their health and progress. This will help doctors learn more about the lasting effects of OAV101 and how it helps people with SMA. It's a way to make sure the treatment continues to be safe and effective in the long run.
At a glance
What is this study about?
This study is designed to follow up with people who have Spinal Muscular Atrophy (SMA) and have previously taken part in a clinical trial for a medicine called onasemnogene abeparvovec, also known as OAV101. SMA is a genetic condition that affects the nerves that control muscle movement, leading to muscle weakness and problems with everyday activities.
The main purpose of this follow-up study is to see how patients are doing in the long term after receiving OAV101. Doctors want to understand if the medicine continues to be safe and effective over many years. This is really important because it helps us learn more about the lasting impact of the treatment on people's health and quality of life.
By carefully watching participants for five years, the researchers hope to gather valuable information. This information will help them assess the long-term benefits and any potential safety concerns. The findings from this study will help healthcare professionals make the best decisions for those living with SMA and improve future care.
Key takeaways
- This study tracks the long-term safety and effects of OAV101 for people with SMA.
- It's for people who previously received OAV101 in another clinical trial.
- Participation involves routine health check-ups over five years.
- The goal is to understand how the medicine works over a long period.
- No new medication is given in this follow-up study.
- Your contribution helps improve future care for SMA patients.
Who may be eligible?
To be able to take part in this study, you must have already participated in an earlier clinical trial where you received the medicine OAV101. This study is specifically for people who have already had this treatment.
Before you start, you or your parent/guardian will need to give written permission. This is called 'informed consent' and it means you understand what the study involves and agree to take part. You also need to be willing and able to follow all the study instructions and attend appointments.
There are no reasons that would automatically stop you from joining this study if you meet the above requirements. This means if you were in a previous OAV101 trial and agree to the consent and follow-up, you can likely join.
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.
- Did you take part in an OAV101 clinical trial before?
- Are you, or your parent/guardian, willing to give written permission to join?
- Are you, or your parent/guardian, able to follow the study's instructions for visits and assessments?
- Do you want to help doctors learn more about the long-term effects of OAV101?
What does participation involve?
If you join this study, you will have a baseline visit at the beginning. After that, you'll have regular check-ups at the study clinic for five years. For the first two years, you can expect to have a visit every six months. Then, for the next three years (years 3 to 5), you will have one visit each year. During these visits, doctors will do tests and check-ups to see how you are doing, focusing on the safety and effects of the OAV101 medicine you received previously. There are no new medications given during this follow-up study; it's about checking your health after the original treatment.
Potential risks and benefits
Locations (32)
- Child Hosp Of The Kings DaughtersVerified postcodeNorfolk, United States· Recruiting
- Novartis Investigative SiteVerified postcodeSydney, Australia· Recruiting
- Novartis Investigative SiteVerified postcodeLeuven, Belgium· Recruiting
- Novartis Investigative SiteVerified postcodeCuritiba, Brazil· Recruiting
- Novartis Investigative SiteVerified postcodeSão Paulo, Brazil· Recruiting
- Novartis Investigative SiteVerified postcodeMontreal, Canada· Recruiting
- Novartis Investigative SiteVerified postcodeChongqing, China· Recruiting
- Novartis Investigative SiteVerified postcodeGuangzhou, China· Recruiting
- Novartis Investigative SiteVerified postcodeChengdu, China· Recruiting
- Novartis Investigative SiteVerified postcodeBeijing, China· Recruiting
- Novartis Investigative SiteVerified postcodeBeijing, China· Recruiting
- Novartis Investigative SiteVerified postcodeBeijing, China· Recruiting
Common questions
What is SMA?
SMA, or Spinal Muscular Atrophy, is a condition that affects muscles, making them weaker over time due to problems with the nerves that control movement.
What is OAV101?
OAV101 is a medicine that was given in previous clinical trials for people with Spinal Muscular Atrophy. This study is now following up on those participants.
Why is this study important?
This study is important because it helps doctors learn how OAV101 works in the long run and if it remains safe and effective for people with SMA over many years.
How long will I be in the study?
If you join this study, you will be followed for a total of five years, with regular check-ups during that time.
Will I receive new treatment in this study?
No, this study is a follow-up. You will not receive any new OAV101 medicine. It's about monitoring your health after previous treatment.
How to find out more
Novartis Pharmaceuticals
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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