Observational long-term follow- up study for patients previously treated with ex vivo gene therapy
Great Ormond Street Hospital (GOSH) is following up with patients who had gene therapy for inherited immune system conditions like Chronic Granulomatous Disease and Severe Combined Immunodeficiency. These conditions make it hard for the body to fight infections. The gene therapy helps by fixing the faulty gene in a patient's own blood stem cells. After gene therapy, patients are usually monitored for a few years. This study extends that monitoring for up to 15 years to make sure the treatment is safe for a long time and keeps working well. It mainly involves reviewing medical records and routine check-ups at GOSH. This helps doctors learn more about gene therapy and improve it for future patients.
At a glance
What is this study about?
You might be familiar with gene therapy if you or someone you know has had it for certain inherited immune system conditions. These conditions, like Chronic Granulomatous Disease and Severe Combined Immunodeficiency (SCID), mean the body struggles to fight off infections. Gene therapy works by taking a patient’s own blood stem cells, correcting the 'faulty' gene in them, and then putting these corrected cells back into the body to help rebuild a healthy immune system.
While patients are usually monitored for 2 to 3 years after receiving this treatment, it's really important to understand how gene therapy works in the long run. That's why Great Ormond Street Hospital (GOSH) is carrying out this special follow-up study, which will monitor patients for up to 15 years. This long-term monitoring helps doctors keep a close eye on the treatment's safety and effectiveness over many years.
The main goal of this study is to gather information from existing medical records and your regular check-ups. This helps doctors learn more about gene therapy and make sure it continues to be a safe and effective option. Your involvement can help improve these life-changing treatments for other children and families in the future.
Key takeaways
- It's a long-term follow-up study for gene therapy patients at GOSH.
- It checks the safety and effectiveness of gene therapy for immune conditions over many years.
- Mainly involves reviewing your existing medical records and routine check-ups.
- No direct health benefits, but helps improve gene therapy for others.
- Very low risk; you can withdraw at any time.
- Runs until 2042 to gather important long-term information.
Who may be eligible?
This study is for patients who have already received gene therapy at Great Ormond Street Hospital (GOSH). This includes those who were part of a gene therapy clinical trial at GOSH, as well as patients who received the treatment because it was considered the best option for their condition, even if it wasn't part of a formal trial.
To join, your gene therapy must have shown that it made a lasting change to your cells. Also, you shouldn't already be part of another long-term follow-up study for the same condition. You, or your parent/guardian if you're under 16, will need to agree to take part by signing a consent form.
If you meet these requirements, you may be able to join the study. If you prefer not to take part, that's perfectly fine and won't affect your medical care at GOSH.
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 you already had gene therapy at Great Ormond Street Hospital (GOSH)?
- Did you receive gene therapy as part of a GOSH trial or through 'compassionate use'?
- Is your gene therapy still showing effects in your cells?
- Are you currently not in another long-term follow-up study for the same condition?
- Are you, or your parent/guardian, able to give permission to join?
What does participation involve?
If you take part in this study, you will continue with your usual medical care and regular follow-up appointments at Great Ormond Street Hospital (GOSH). The study itself mainly involves doctors reviewing your existing medical records and information gathered during your routine check-ups. You won't need extra hospital visits just for the study.
In some cases, you might be asked to provide an additional blood sample during a routine visit. Very occasionally, a bone marrow sample might be needed, but this would be fully discussed with you or your parents/guardians beforehand. The total duration of this study aims to follow patients until June 2042, meaning some patients could be followed for up to 15 years.
Potential risks and benefits
Locations (1)
- -Approximate-, England
Common questions
What kind of conditions does gene therapy treat in this study?
This study looks at gene therapy treatments for inherited immune system conditions like Chronic Granulomatous Disease and Severe Combined Immunodeficiency (SCID), which make it hard for the body to fight infections.
Will I have to do anything differently if I join the study?
Not usually. You'll continue with your normal medical care and routine follow-up appointments at GOSH. The study mainly involves reviewing your medical records.
Are there any extra tests for this study?
Sometimes, an extra blood sample might be taken during your routine appointments. Very rarely, a bone marrow sample might be needed, but this would be discussed carefully with you first.
How long will this study last?
The study is expected to run until June 2042, meaning some patients could be followed for up to 15 years to track the long-term effects of gene therapy.
Who is paying for this study?
The study is being funded by Great Ormond Street Hospital (GOSH) in the UK.
How to find out more
Fahmida Hoque
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.