Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome
This project is a long-term follow-up study for individuals who previously took part in an early-stage clinical trial for Wiskott-Aldrich Syndrome. These people received a gene therapy treatment that used their own blood stem cells, modified in a lab, to help correct the underlying problem with the syndrome. The main goal of this current study is to carefully monitor their health over many years to see how safe and effective this special gene therapy is in the long run. Researchers want to understand if the benefits continue and if there are any new or delayed side effects. It helps scientists learn more about this innovative treatment.
Överblick
Vad handlar denna studie om?
Wiskott-Aldrich Syndrome is a rare genetic condition that mainly affects boys. It can cause problems with a child's immune system, making them more prone to infections and bleeding, and can also lead to eczema. In the past, some children with this condition took part in a special kind of treatment called gene therapy. This treatment involved taking some of their own blood stem cells, changing them in a laboratory to fix the genetic problem, and then putting them back into the child.
This current study is not a new treatment trial. Instead, it's a very important follow-up for those who received that gene therapy in earlier studies, which took place in France and the United Kingdom. Think of it like a long-term health check to see how everyone is doing years after their treatment. The doctors want to make sure the gene therapy continues to be safe and effective, and to understand any long-term effects, good or bad.
By carefully watching the health of these individuals over many years, doctors and scientists can gather crucial information. This long-term monitoring helps them understand if the improved immune system and other benefits last, or if there are any new health issues that might appear much later. This information is key to making sure future gene therapies are as safe and successful as possible for others with Wiskott-Aldrich Syndrome.
Viktiga punkter
- This study is long-term monitoring for gene therapy previously received.
- It aims to understand the long-term safety and benefits of the treatment.
- Only individuals from specific past trials are eligible to participate.
- Participation involves regular health check-ups over an extended period.
- Your contribution helps improve future treatments for Wiskott-Aldrich Syndrome.
Vem kan vara berättigad?
This study is specifically for certain people who have already been involved in earlier gene therapy trials for Wiskott-Aldrich Syndrome. To be part of this long-term check-up, you must have previously participated in one of the initial Phase 1/2 studies (called GTG002.07 or GTG003.08) in either France or the United Kingdom.
If you, or the person you are a guardian for, are happy to come back for these follow-up appointments and agree to take part, then you would be eligible. It's important that you understand what's involved and agree to continue with the study's requirements.
However, if a patient, or their parents or guardians, decide they don't want to come for the regular follow-up visits or cannot commit to the study period, then they would not be able to participate in this particular long-term safety study.
Kan denna studie passa dig?
Svara på dessa snabba frågor för att se om du kan vara berättigad. Detta är endast en vägledning — forskarteamet fattar det slutgiltiga beslutet.
- Have I participated in one of the original gene therapy trials (GTG002.07 or GTG003.08) for Wiskott-Aldrich Syndrome?
- Am I, or my guardian, willing and able to attend regular follow-up appointments for many years?
- Do I understand that this study is about monitoring my health long-term, not a new treatment?
- Am I comfortable with regular health checks and blood tests as part of this study?
Vad innebär deltagande?
If you decide to take part in this follow-up study, it means you'll have regular check-ups over a long period. The exact number and frequency of these visits will be discussed with you, but they are designed to monitor your health and the effects of the gene therapy. During these visits, doctors will likely carry out various assessments, such as blood tests to check your immune system and overall health, and perhaps other examinations relevant to Wiskott-Aldrich Syndrome.
There won't be any new medication given as part of this follow-up study; the purpose is to observe the long-term impact of the treatment you received previously. The total duration of your participation will be many years, as the goal is to track the long-term safety and effectiveness of the gene therapy. This ongoing monitoring is crucial for understanding how the treatment works over an extended time.
Potentiella risker och fördelar
Platser (2)
- Hopital Necker - Enfants MaladesVerifierat postnummerParis, Frankrike
- UCL Institute of Child HealthVerifierat postnummerLondon, Storbritannien
Vanliga frågor
Vad är huvudmålet med denna studie?
The main goal is to carefully check on the health of people who received gene therapy for Wiskott-Aldrich Syndrome in earlier trials, to see how they are doing many years later and to monitor the long-term safety and effectiveness of the treatment.
Is this a new treatment for Wiskott-Aldrich Syndrome?
No, this is not a new treatment. It's a follow-up study for people who already had a particular gene therapy in previous trials.
Vem kan delta i denna studie?
Only people who were part of specific earlier gene therapy trials for Wiskott-Aldrich Syndrome in France or the UK and are willing to continue with follow-up appointments can join.
Vad måste jag göra om jag går med?
You will have regular health check-ups and medical tests, like blood tests, to help doctors see how you are doing over a long period.
Kan jag sluta delta i studien om jag ändrar mig?
Yes, you can choose to stop participating in the study at any time, for any reason, and it will not affect your medical care.
Hur du får reda på mer
Prata alltid med din läkare eller specialist innan du bestämmer dig för att delta i en studie.
Diskussion
Gemenskapsdiskussion
Drivs av vårt forum på community.patient.info. Var vänlig och respektfull — detta är inte medicinsk rådgivning.