An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID
This study is a long-term check-up for individuals who have already received a gene therapy treatment for a very rare and serious immune system condition called ADA-SCID. This gene therapy helped their bodies make a missing enzyme. The main goal is to carefully watch these patients over many years to see how safe the gene therapy has been and how well it continues to work. Doctors will be collecting information about their health from their regular check-ups. It's important for understanding the long-term effects of this new type of medicine. No new treatment will be given during this study; it's purely about observing those already treated.
At a glance
What is this study about?
This study is like a long-term health check for people who have already had a special type of treatment called "gene therapy" for a very rare and serious condition. This condition is called Adenosine Deaminase Deficiency, or ADA-SCID for short. People with ADA-SCID have a very weak immune system, making them very vulnerable to infections.
The gene therapy treatment used in the past aimed to correct the problem at its root by giving the body instructions to make a missing enzyme. This particular study isn't about giving new treatment; instead, it's about carefully watching these patients over many years to understand how safe the gene therapy has been in the long run and how well it is continuing to help them.
By collecting health information from their regular doctor's visits, researchers can learn important details about the effects of this advanced treatment over time. This helps to make sure that these new therapies are safe and effective for patients both now and in the future, and meets important guidelines set by health authorities.
Key takeaways
- This study tracks the long-term health of people who had gene therapy for ADA-SCID.
- It helps us understand how safe and well gene therapy works over many years.
- No new treatments are given in this study; it's purely for observation.
- Your health information is collected from your normal, yearly doctor's visits.
- The goal is to follow patients for up to 15 years after their treatment.
- Participation helps future patients and improves our understanding of gene therapy.
Who may be eligible?
This study is for anyone who has previously received a specific gene therapy treatment for ADA-SCID. This treatment was part of a clinical program called OTL-101.
To join, your doctor must also confirm that the gene therapy is still working within your body, meaning the modified cells are still present.
Most importantly, you (or your parent/guardian if you're a child) must be willing to give your permission to take part and understand what the study involves. There are no reasons that would prevent you from joining if you meet these basic requirements.
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.
- Have I already received the specific gene therapy for ADA-SCID (part of the OTL-101 program)?
- Has my doctor confirmed that the gene therapy is still working in my body?
- Am I (or my legal guardian) willing to provide consent and understand the study?
- Can I attend my regular annual health check-ups?
- Do I live in the UK and receive care from a local healthcare professional?
What does participation involve?
If you join this study, you won't be given any new medications or treatments. Instead, the study will collect information about your health from your regular annual check-ups with your doctor or local healthcare professional. This means you will simply continue with your usual medical care.
Before you start, you or your legal guardian will need to give your informed consent. This is a conversation where you'll learn all about the study, how long it's expected to last for you, and what health information will be collected. It also explains the schedule for collecting this information, which is usually once a year during your standard doctor's visits.
The study aims to follow patients for up to 15 years after their gene therapy treatment. For some patients, doctors might need to look back at past health records from before the study started. For everyone else, information will be gathered going forward.
Potential risks and benefits
Locations (2)
- Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical CenterVerified postcodeLos Angeles, United States
- UCL Great Ormond Street Institute of Child HealthVerified postcodeLondon, United Kingdom
Common questions
What is ADA-SCID?
ADA-SCID is a very rare and serious disease where the body can't make an important enzyme, leading to a very weak immune system and a high risk of severe infections.
What is gene therapy?
Gene therapy is a new type of treatment that aims to fix the root cause of certain diseases by correcting or replacing faulty genes in the body's cells.
Will I receive any new treatment in this study?
No, this study is only about observing and collecting information on patients who have already received the gene therapy treatment. No new medicines or treatments are given.
How long will I be in the study?
The study aims to follow patients for up to 15 years after their gene therapy treatment to understand its very long-term effects.
Will my regular doctor look after me?
Yes, your usual doctor or local healthcare professional will continue to provide your standard medical care, and the study will gather information from these visits.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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