Active not recruitingPHASE1, PHASE2INTERVENTIONAL

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.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

Genethon

Enrolment target

Start

01 Sep 2014

Estimated completion

01 Oct 2032

Wiskott-Aldrich Syndrome

What is this study about?

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.

Key takeaways

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.

Who may be eligible?

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.

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 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?

Answer every question to see your result.

What does participation involve?

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.

Potential risks and benefits

The main benefit of participating in this follow-up study is contributing valuable information that helps doctors and scientists better understand the long-term safety and effectiveness of the gene therapy for Wiskott-Aldrich Syndrome. This knowledge can help improve treatments for others in the future. As this is a follow-up and not a new treatment, there are no new treatment-related risks beyond those already discussed when you joined the original trial. However, there will be routine medical procedures like blood tests, which carry small risks such as bruising or minor discomfort. You are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (2)

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

Hopital Necker - Enfants Malades
Verified postcode
Paris, France
UCL Institute of Child Health
Verified postcode
London, United Kingdom

Common questions

What is the main goal of this study?

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.

Who can take part in this study?

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.

What will I have to do if I join?

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.

Can I stop participating in the study if I change my mind?

Yes, you can choose to stop participating in the study at any time, for any reason, and it will not affect your medical care.