Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102
This is a long-term follow-up study for people who previously received a treatment called RP-L102 for Fanconi Anaemia. Doctors want to keep checking on these patients for up to 15 years to understand the long-term safety and how well the treatment continues to work over time. If you took part in one of the past RP-L102 studies, you might be invited to join this follow-up. This helps researchers gather important information about this new treatment, which could help future patients with Fanconi Anaemia. The study involves regular check-ups, some of which can be done closer to home, but some visits to a special study centre will also be needed.
At a glance
What is this study about?
Fanconi Anaemia is a serious inherited condition that affects how the body creates new blood cells. People with Fanconi Anaemia can experience many health problems, including a higher risk of certain cancers, and often need special treatments like blood transfusions or bone marrow transplants. This can be a very challenging condition for patients and their families.
This study, called a long-term follow-up, is specifically for people who have already received an investigational treatment named RP-L102 in one of the earlier studies. RP-L102 is a type of gene therapy designed to help correct the underlying problem in Fanconi Anaemia by introducing a healthy gene into the patient's cells. Early studies looked at whether RP-L102 was safe in the short-term and if it started to help patients.
The main goal of this current study is to continue monitoring these patients for a much longer time – up to 15 years. This extended follow-up is very important because it helps doctors understand the long-term safety of the RP-L102 treatment and how effectively it works over many years. This sort of information is vital to know if the treatment has lasting benefits and if there are any new or delayed side effects. The information gathered will contribute significantly to scientific knowledge and could help improve future treatments for Fanconi Anaemia.
Key takeaways
- This is a long-term follow-up study for previous RP-L102 participants.
- It aims to track the safety and effectiveness of RP-L102 for up to 15 years.
- Participation involves regular check-ups and blood tests.
- Some visits require travel to the study centre, especially in the first two years.
- Joining helps advance understanding of Fanconi Anaemia treatments.
Who may be eligible?
To be able to join this follow-up study, you must have previously taken part in one of the main RP-L102 studies (these were called RP-L102-0418, RP-L102-0319, or RP-L102-0118). You must also have actually received the RP-L102 treatment as part of that earlier study.
It's important that you are also able and willing to attend all the required study appointments and follow any other instructions from the study team. You'll need to agree to take part by signing a consent form, which means you understand what the study involves and agree to participate. If you're a child, you'll also need to agree if you're old enough.
There are no reasons listed that would stop someone who meets these conditions from joining this particular follow-up study.
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 I take part in one of the earlier RP-L102 studies (RP-L102-0418, RP-L102-0319, or RP-L102-0118)?
- Did I actually receive the RP-L102 treatment in that earlier study?
- Am I able to attend regular appointments and follow study instructions?
- Am I willing to sign a consent form to participate?
What does participation involve?
If you decide to take part in this study, you will be monitored for up to 15 years after you received the RP-L102 treatment. This means you'll have regular check-ups with the study team. For the first two years after your treatment, you will need to travel to the main study centre for your appointments. After two years, some of your check-ups can be done closer to your home by your local healthcare team, though visiting the study centre is still encouraged if possible, especially between years 2 and 5.
During these visits, you will have blood samples taken. These samples will be stored and tested if there's a medical reason, for example, if you develop a new health problem. The study team will be in regular contact with you to arrange these appointments and make sure you receive the best care while helping gather important information.
Potential risks and benefits
Locations (3)
- Lucille Packard Children's Hospital, Stanford UniversityVerified postcodePalo Alto, United States
- Hospital Infantil Universitario Niño JesúsVerified postcodeMadrid, Spain
- University College London Great Ormond Street Institute of Child Health (GOSH)Verified postcodeLondon, United Kingdom
Common questions
What is RP-L102?
RP-L102 is an experimental gene therapy that was given in earlier studies to people with Fanconi Anaemia. It aims to help correct the underlying genetic problem of the condition.
Why is this study important?
This study is important because it will track how people who received RP-L102 are doing over a long period, up to 15 years. This helps doctors understand the long-term safety and effectiveness of the treatment.
Do I have to go to the study centre for every visit?
For the first two years after your RP-L102 treatment, you will need to visit the study centre. After that, some visits can be done closer to home, but central visits are still encouraged, especially in years 2 to 5.
What kind of tests will I have?
You will have regular check-ups, which will include blood tests. Your blood samples will be stored and checked if needed, for instance, if a new health concern arises.
Can I leave the study if I change my mind?
Yes, you can withdraw from the study at any time, for any reason, and this will not affect the medical care you receive for your Fanconi Anaemia.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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