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Study of G1XCGD Lentiviral Vector in Patients with X-Linked Chronic Granulomatous Disease

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What is this trial about?

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

This clinical trial is focused on studying a rare genetic disorder called X-linked Chronic Granulomatous Disease (X-CGD). This condition affects the immune system, making it difficult for the body to fight off certain infections. The study is testing a new treatment involving a special type of cell therapy. The treatment uses the patient's own blood stem cells, known as CD34+ cells, which are modified in the laboratory with a lentiviral vector called G1XCGD. This vector carries a healthy version of the human CYBB gene, which is important for normal immune function. The modified cells are then injected back into the patient to help restore their immune system.

The purpose of the study is to evaluate the safety and effectiveness of this treatment in patients with X-CGD. Participants will receive the treatment through an intravenous injection, which means it is delivered directly into the bloodstream. The study will monitor the patients over a period of time to see how well the treatment works and to check for any side effects. The main goal is to see if the treatment can help the immune system function better and remain stable over time.

Throughout the study, researchers will look at various aspects of the patients' health, including their nutritional status, growth, and development. They will also assess how well the immune system is working by checking for improvements in the body's ability to fight infections. The study aims to provide valuable information on whether this new cell therapy can be a safe and effective option for people with X-CGD.

The research process

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

  1. Step 1

    Enrollment

    Eligibility is determined based on specific criteria, including age, confirmed diagnosis, and absence of certain infections.

    Consent is required from the patient or their guardian.

  2. Step 2

    Preparation

    Patients may undergo mobilization of peripheral blood stem cells or bone marrow harvests, depending on age and physician's decision.

  3. Step 3

    Treatment administration

    The treatment involves the use of G1XCGD, a solution for injection containing autologous CD34+ cells transduced with a lentiviral vector.

    The solution is administered intravenously.

  4. Step 4

    Monitoring and follow-up

    Safety and efficacy are evaluated through biochemical and functional tests over a period of 12 months.

    Regular assessments include monitoring for adverse events and checking the functionality of specific blood cells.

  5. Step 5

    Evaluation of outcomes

    Primary outcomes focus on safety and the restoration of cell function.

    Secondary outcomes include improvements in nutritional status, growth, and immune function.

Who can join the trial?

8 criteria

  • The study is for **male** patients with a condition called **X-linked Chronic Granulomatous Disease (X-CGD)**.
  • Patients should be older than **23 months**. Younger patients, older than **1 month** and up to **23 months**, may join if the doctor agrees. For these younger patients, a different procedure might be used.
  • A **molecular diagnosis** must be confirmed by a test called **DNA sequencing**. This should show a lack or more than **70% reduction** in the activity of a specific enzyme called **NAHPD-oxidase**.
  • Patients must have at least one **severe infection** or **inflammatory complication** that is ongoing, resistant, or at high risk of coming back, and requires hospital treatment despite regular therapy.
  • No suitable **HLA-matched donor** should be available after a **3-month search**, unless waiting for a match or doing a different type of transplant is too risky.
  • Patients should not have **co-infections** with viruses like **HIV**, **hepatitis B**, or **hepatitis C**.
  • Adult patients must provide **written informed consent** to participate.
  • For children, a **parent or guardian** must give consent, and if appropriate, the child should also agree.

Who cannot join the trial?

4 criteria

  • Individuals who are not male cannot participate in the study.
  • Individuals who are not within the specified age range cannot participate. The age range is typically defined in the study details.
  • Individuals who do not have the specific medical condition being studied, which in this case is **X-linked Chronic Granulomatous Disease**, cannot participate. This is a genetic disorder affecting the immune system.
  • Individuals who are considered part of a vulnerable population may not be eligible. A vulnerable population includes groups who may have limited ability to give informed consent or are at higher risk of harm.
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Investigated drugs

Autologous CD34+ Cells Transduced with the G1XCGD Lentiviral Vector This therapy involves using a patient's own stem cells, known as CD34+ cells, which are collected and then modified in the laboratory. The modification is done using a special tool called the G1XCGD lentiviral vector. This tool helps to insert a new gene into the cells. The goal is to correct a genetic problem in patients with X-linked chronic granulomatous disease, a condition that affects the immune system. After the cells are modified, they are returned to the patient's body to help improve their immune function.

What is already known about the treatment

G1XCGD Lentiviral Vector – This medication is administered through a process involving the collection and modification of a patient's own stem cells, specifically CD34+ cells, which are then reintroduced into the patient. It is currently being studied in clinical trials for its potential to treat X-linked Chronic Granulomatous Disease, a rare genetic disorder. The main therapeutic indication is to restore normal immune function by correcting the genetic defect in the patient's cells. At the molecular level, the lentiviral vector works by inserting a functional copy of the gene into the patient's stem cells, allowing them to produce the necessary proteins to fight infections. This medication falls under the pharmacological classification of gene therapy.

Investigated diseases

X-linked Chronic Granulomatous Disease – This is a rare genetic disorder that affects the immune system, specifically the ability of certain white blood cells to kill certain bacteria and fungi. It is caused by mutations in genes that are responsible for producing a protein complex called NADPH oxidase, which is crucial for the respiratory burst in phagocytes. Individuals with this disease often experience recurrent bacterial and fungal infections, as their immune cells cannot effectively destroy these pathogens. The disease is inherited in an X-linked pattern, meaning it primarily affects males, while females may be carriers. Over time, patients may develop chronic inflammation and granulomas, which are clusters of immune cells that form in response to persistent infection or inflammation. The condition requires careful management to prevent and treat infections and inflammation.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2024-512790-27-00Protocol codeG1XCGD.02Estimated enrolment5 patientsSponsorGenethon

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).