Active not recruitingPHASE3INTERVENTIONAL

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

This study is for individuals who have previously received a gene therapy treatment called onasemnogene abeparvovec-xioi for Spinal Muscular Atrophy (SMA) in an earlier clinical trial. The main goal is to keep a long-term eye on their health and safety. Researchers also want to understand how well the gene therapy continues to work over many years and if its positive effects last. Participants in this study will have already taken part in a 'parent' study, and this new study will allow for continuous monitoring to make sure the treatment remains safe and effective for them. It's an important step to understand the lasting impact of this special treatment.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Novartis Gene Therapies

Enrolment target

Start

10 Feb 2020

Estimated completion

31 Dec 2035

Spinal Muscular Atrophy Type I Spinal Muscular Atrophy Type II Spinal Muscular Atrophy Type III SMA

What is this study about?

This study is a long-term check-up for people who have received a special treatment for Spinal Muscular Atrophy (SMA) called onasemnogene abeparvovec-xioi. SMA is a condition that affects nerves and muscles, making them weaker over time. The treatment is a type of gene therapy, which aims to fix the root cause of SMA.

Since this treatment is quite new, it’s really important to keep checking on people who have had it. This study wants to see how well the treatment continues to work over many years and if its good effects last. Researchers also want to make sure the treatment remains safe in the long term. Participants will be joining this study after being part of an earlier trial where they first received the gene therapy.

By carefully watching these individuals over time, doctors and scientists can learn a lot about the lasting benefits and any potential side effects of onasemnogene abeparvovec-xioi. This helps them understand the treatment better and ensures that people with SMA can continue to benefit from it safely in the future.

Key takeaways

This study is a long-term check to see how well SMA gene therapy works and if it's safe over many years.
It's only for people with SMA who have already had the onasemnogene abeparvovec-xioi treatment in a past trial.
No new medication is given; the focus is on monitoring the existing treatment's effects.
Participation helps scientists learn more about long-term care for SMA.
You will have regular check-ups to monitor your health and the treatment's ongoing effectiveness.

Who may be eligible?

You might be able to join this study if you have Spinal Muscular Atrophy (SMA) and have already received the onasemnogene abeparvovec-xioi gene therapy as part of an earlier study from Novartis Gene Therapies.

It's important that you, or your parent or legal guardian if you are a child, are happy to take part and can understand and sign the consent forms. You also need to be able to come to all the study appointments as scheduled.

Basically, if you've already had this specific gene treatment in a previous research trial, and you're willing to follow the study plan, you could be eligible.

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.

Do you have Spinal Muscular Atrophy (SMA)?
Have you already received the onasemnogene abeparvovec-xioi gene therapy in a previous study from Novartis Gene Therapies?
Are you, or your parent/guardian, willing to understand and sign consent forms?
Can you attend regular appointments for health check-ups?
Are you able to participate in a study that lasts for many years?

Answer every question to see your result.

What does participation involve?

If you decide to join this study, it means you'll be regularly seen by the study team. They will continue to monitor your health and overall well-being over a long period. This will involve regular visits to a clinic, typically for assessments and check-ups. These assessments might include physical examinations, questions about your health, and possibly some tests to see how the treatment is still working and if there are any changes in your condition. You won't receive new medication as part of this follow-up study; it's about seeing how the original treatment continues to work. The total duration of your participation will be long-term, lasting for several years, ensuring that the effects of the therapy are tracked over a significant period. The exact schedule of visits will be explained in detail.

Potential risks and benefits

The main benefit of taking part is contributing valuable information that helps doctors understand the long-term safety and effectiveness of onasemnogene abeparvovec-xioi, which can improve care for others with SMA. While no new medication is given, there are still potential risks associated with study visits, such as the time commitment and any procedures involved in monitoring your health. You will be fully informed of any potential risks before you consent. Remember, choosing to participate is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (31)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Stanford University Medical Center
Verified postcode
Palo Alto, United States
Children's Hospital Colorado
Verified postcode
Aurora, United States
Ann Robert H. Lurie Children's Hospital of Chicago
Verified postcode
Chicago, United States
John Hopkins Hospital - David M. Rubenstein Child Health Building
Verified postcode
Baltimore, United States
Massachusetts General Hospital
Verified postcode
Boston, United States
Boston Children's Hospital
Verified postcode
Boston, United States
Spectrum Health Hospitals Helen DeVos Children's Hospital
Verified postcode
Grand Rapids, United States
Washington Unviersity School of Medicine in Saint Louis
Verified postcode
St Louis, United States
Columbia University Medical Center
Verified postcode
New York, United States
Duke University
Verified postcode
Durham, United States
Nationwide Children's Hospital
Verified postcode
Columbus, United States
Children's Hospital of Philadelphia
Verified postcode
Philadelphia, United States

Common questions

What is the main purpose of this study?

The study aims to check on the long-term safety and how well the onasemnogene abeparvovec-xioi gene therapy continues to work for people with SMA who have already received it.

Do I need to have SMA to join?

Yes, you must have Spinal Muscular Atrophy (SMA) and have previously received the gene therapy in a specific earlier study.

Will I get new treatment in this study?

No, this study is about checking on the long-term effects of the gene therapy you've already received, not giving new treatment.

How long will I need to be in the study?

This is a long-term study, meaning you would be monitored for several years to understand the lasting impact of the treatment.

Can I leave the study if I change my mind?

Yes, you can choose to leave the study at any time, and it won't affect your medical care.