RecruitingRare disease

Long-term Safety and Efficacy Study of Etuvetidigene Autotemcel for Patients with Wiskott-Aldrich Syndrome

Verified siteInvestigational

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial focuses on a rare genetic disorder called Wiskott-Aldrich Syndrome (WAS). This condition affects the immune system and can lead to issues such as bleeding, infections, and eczema. The study is examining a treatment known as Telethon003, which is a type of gene therapy. Gene therapy involves using a patient's own cells, which are modified outside the body to correct the genetic defect, and then infused back into the patient. The specific treatment being studied is called etuvetidigene autotemcel, also known as OTL-103.

The purpose of this study is to understand the long-term safety and effectiveness of the Telethon003 treatment for individuals who have already received it. Participants in the study will be monitored over a long period to see how well they are doing after the treatment. This includes checking for any side effects or health issues that might arise, as well as assessing their overall health and quality of life. The study will also look at how the treatment affects survival rates and the frequency of infections and bleeding episodes.

Throughout the study, various health aspects will be evaluated, such as the occurrence of eczema, autoimmune conditions, and blood disorders. The study will also track changes in growth for children, and assess quality of life using specific questionnaires. The goal is to gather comprehensive information on how the treatment impacts the health and well-being of those with Wiskott-Aldrich Syndrome over time.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, it is confirmed that you have been previously treated with Telethon003 for Wiskott-Aldrich syndrome (WAS). You or your legal guardian must provide informed consent to participate in the study.
You must be willing to attend all scheduled study visits and comply with the study protocol.
Step 2
Treatment administration
The study involves a long-term follow-up of the treatment you have already received. No new medication is administered during this phase.
The focus is on monitoring the long-term safety and effectiveness of the Telethon003 treatment.
Step 3
Monitoring and assessments
Regular assessments will be conducted to monitor your health and the effects of the previous treatment. This includes recording any adverse events or serious adverse events related to the gene therapy.
Specific assessments include checking for any new or worsening health conditions, such as infections, bleeding episodes, eczema, autoimmune disorders, and any signs of cancer.
Step 4
Quality of life evaluations
Your quality of life will be evaluated using various questionnaires, such as the PedsQL (Pediatric Quality of Life Inventory) and other health-related quality of life scales.
These evaluations help understand the impact of the treatment on your daily life and overall well-being.
Step 5
Long-term follow-up
The study aims to follow your health and treatment outcomes for up to 15 years after the initial treatment with Telethon003.
This long-term follow-up is crucial to ensure the continued safety and effectiveness of the treatment.

Who can join the trial?

3 criteria

The patient has been treated with Telethon003.
The patient or, if applicable, the patient’s parent(s) or legal guardian(s), are able and willing to provide informed consent. Informed consent means they understand the study and agree to participate.
Willingness to attend the study visit schedule and be compliant with the protocol. This means the patient agrees to follow the study rules and attend all required appointments.

Who cannot join the trial?

8 criteria

Patients with any other serious health condition that might interfere with the study.
Patients who have had a recent infection that could affect their participation.
Patients who are currently taking medications that might interfere with the study treatment.
Patients who have a history of allergic reactions to similar treatments.
Patients who are pregnant or breastfeeding.
Patients who have participated in another clinical trial recently.
Patients who have a history of substance abuse.
Patients who are unable to comply with the study procedures.

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Ospedale San Raffaele S.r.l.
Milan, Italy

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Recruiting

Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

Telethon003 is a type of gene therapy used to treat Wiskott-Aldrich Syndrome (WAS), a rare genetic disorder that affects the immune system and blood cells. This therapy involves taking some of the patient's own blood stem cells and modifying them outside the body using a special virus called a lentivirus. The virus helps to insert a healthy copy of the gene that is faulty in WAS into the patient's stem cells. These modified cells are then returned to the patient's body, where they can produce healthy blood cells. The goal of this therapy is to improve the patient's immune system and reduce the symptoms of WAS over the long term. This clinical trial is focused on understanding how safe and effective this treatment is over an extended period.

Etuvetidigene Autotemcel

What is already known about the treatment

Telethon003 – This medication is administered through a process called autologous ex vivo gene therapy, where a patient's own stem cells are modified outside the body and then reintroduced. Currently, Telethon003 is being studied for its long-term safety and effectiveness in treating Wiskott-Aldrich syndrome (WAS), a rare genetic disorder. The main therapeutic indication for Telethon003 is to address the symptoms and complications associated with WAS. At the molecular level, the therapy works by using a lentiviral vector to insert a correct copy of the gene responsible for WAS into the patient's stem cells, aiming to restore normal immune function. Telethon003 falls under the pharmacological classification of gene therapy products.

Investigated diseases

Wiskott-Aldrich syndrome – Wiskott-Aldrich syndrome is a rare genetic disorder that affects the immune system and blood clotting. It is characterized by a combination of eczema, low platelet count, and recurrent infections. The disease is caused by mutations in the WAS gene, which leads to abnormal functioning of white blood cells and platelets. As the condition progresses, individuals may experience increased susceptibility to infections, bleeding episodes, and autoimmune disorders. Over time, there may also be a risk of developing certain types of cancers. The severity of symptoms can vary widely among affected individuals.