RecruitingRare disease

Long-Term Safety Study of Gene Therapy with Etuvetidigene Autotemcel for Patients with Wiskott-Aldrich Syndrome

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What is this trial about?

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

This clinical trial focuses on the long-term safety of a gene therapy treatment for patients with Wiskott-Aldrich Syndrome, a rare genetic disorder that affects the immune system and blood clotting. The treatment involves using the patient's own blood stem cells, which are modified outside the body with a special virus to carry a healthy version of the WASP gene. This modified cell product is known as etuvetidigene autotemcel and is given to patients through an infusion.

The purpose of the study is to monitor patients who have received this gene therapy to ensure its safety over a long period. Participants will have regular check-ups to assess their overall health, including their immune system function and blood cell counts. The study will also look for any delayed side effects, such as new health issues that might develop years after the treatment.

Throughout the study, researchers will evaluate the need for additional treatments, such as antibiotics or blood transfusions, and will perform optional tests to check the health of the bone marrow. The study aims to provide valuable information on the long-term effects of this gene therapy for Wiskott-Aldrich Syndrome and help improve future treatments for this condition.

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
Initial treatment
Receive a single infusion of autologous CD34+ cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector. This is administered through intravenous administration as a solution for infusion.
Step 2
Yearly follow-up visits
Attend yearly visits to evaluate the safety of the gene therapy. This includes checking for any serious adverse events (SAEs) and monitoring for delayed events such as malignancies, blood-related issues, autoimmune events, and mortality.
Assess the gene therapy procedure's safety by analyzing gene transfer through lentiviral integration sites in different cell subpopulations and quantifying vector copy numbers (VCN) using real-time q-PCR.
Check for replication competent lentivirus (RCL) during these visits.
Step 3
Clinical status evaluation
At 3, 4, 5, 6, 7, 8, 9, and 10 years post-gene therapy, evaluate the clinical status, including weight and a complete clinical exam.
Monitor the evolution of key medical events related to Wiskott-Aldrich Syndrome (WAS), such as eczema status, infections, bleeding symptoms, and autoimmune manifestations.
Step 4
Hematological and immune system evaluation
Evaluate blood reconstitution at 3, 4, 5, 6, 7, 8, 9, and 10 years post-gene therapy, including complete blood count (CBC) with platelet count and size.
Assess the reconstitution of cell-mediated and humoral immunity, including lymphocyte subsets, antibody production, and response to antigens.
Step 5
Additional evaluations
Evaluate the need for associated treatments such as immunoglobulins, antibacterial, antifungal, and antiviral drugs, and transfusions during yearly visits.
Assess the representation of TCR families and bone marrow integrity at 3, 4, and 5 years post-gene therapy. Bone marrow aspiration is optional.

Who can join the trial?

6 criteria

The study is for patients who have Wiskott-Aldrich Syndrome, a rare genetic disorder.
Participants must have been part of earlier phase I/II studies and received a specific treatment. This treatment involved a single infusion of their own blood cells, called **CD34+ cells**, which were modified using a special method known as the **lentiviral vector**.
The study was conducted in France and the United Kingdom.
Participants or their parents/guardians must have signed a document called an **informed consent**, which means they agree to take part in the study after understanding what it involves.
The study is open to male participants only.
The study includes participants who are considered part of a **vulnerable population**, meaning they may need extra protection or care.

Who cannot join the trial?

3 criteria

Only male patients can participate, so females are excluded.
Patients who have not received a specific type of cell treatment called autologous transplantation with CD34+ cells are excluded. This means the patient must have had their own cells used in a previous treatment.
Patients who have not been treated with a special type of virus called a lentiviral vector are excluded. This is a tool used to deliver genetic material into cells.

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Hopital Necker Enfants Malades
Paris, France

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Investigated drugs

CD34+ cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector: This therapy involves using a patient's own stem cells, which are collected and then modified in the laboratory. The modification is done using a special type of virus called a lentiviral vector. This vector carries a corrected version of a gene that is not working properly in patients with Wiskott-Aldrich Syndrome. After the modification, the cells are returned to the patient to help improve their immune system and blood cell function.

Etuvetidigene Autotemcel

What is already known about the treatment

CD34+ Cells Transduced with w1.6_hWASP_WPRE (VSVg) Lentiviral Vector – This treatment is administered through an autologous transplantation, where a patient's own stem cells are modified and reintroduced into their body. It is currently being studied in clinical trials for its long-term safety and effectiveness in treating Wiskott-Aldrich Syndrome, a rare genetic disorder. The therapy works by using a lentiviral vector to deliver a corrected gene into the patient's stem cells, aiming to restore normal immune function. It falls under the category of gene therapy, which involves altering genetic material to treat or prevent disease.

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, recurrent infections, and a tendency to bleed easily due to low platelet counts. The disease is caused by mutations in the WAS gene, which leads to abnormal functioning of the immune cells. As the condition progresses, individuals may experience autoimmune disorders and an increased risk of developing certain cancers. The severity of symptoms can vary widely among affected individuals. The syndrome primarily affects males, as it is linked to the X chromosome.