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Safety and efficacy of autologous CD34+ cells transduced with a vector encoding human NCF1 in children and adults with p47‑deficient chronic granulomatous disease

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What is this trial about?

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

The study focuses on Chronic granulomatous disease (CGD) caused by a defect called p47phox deficiency. This rare condition makes certain white blood cells unable to produce a chemical called NADPH oxidase activity that helps kill germs, leading to frequent infections and inflammation. The investigational treatment uses the patient’s own blood stem cells, known as CD34+ cells, which are taken out, altered with a lentiviral vector that carries a normal copy of the NCF1 gene, and then returned to the body through an infusion. The product is identified by the code name SGX-001. Before the cells are given back, participants receive a strong chemotherapy regimen, referred to as myeloablative conditioning, to prepare the bone marrow.

The purpose of the study is to evaluate the safety of this single administration. After the infusion, participants are followed for about a year, with regular visits that include physical exams, vital sign checks, blood tests, and heart rhythm recordings (ECG). Doctors will watch for any side effects and will also test the blood to see if at least 10 % of the white blood cells show restored granulocyte function, indicating that the gene therapy is working. The study does not involve a comparison group.

The research process

The trial runs in 10 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Enrollment and baseline assessment

    After joining the study, you will sign the informed consent form and undergo baseline evaluations including physical examination, laboratory tests, vital sign measurements, and an electrocardiogram.

    The purpose of these assessments is to document your health status before any study procedures begin.

  2. Step 2

    Collection of autologous cd34+ cells

    Your own stem cells, called cd34+ cells, will be collected from your blood using a process called apheresis.

    The collected cells are sent to a specialized laboratory where they will be modified for the study.

  3. Step 3

    Manufacturing of sgx-001

    In the laboratory, your cd34+ cells are transduced with a lentiviral vector that carries the human ncf1 gene.

    The resulting product, known as sgx-001, is prepared as a dispersion for infusion.

  4. Step 4

    Myeloablative conditioning

    Before receiving sgx-001, you will undergo myeloablative conditioning, which is an intensive chemotherapy regimen that clears space in the bone marrow for the new cells.

    Specific drugs, doses, and schedules are determined by the treating team and are part of the study protocol.

  5. Step 5

    Infusion of sgx-001

    A single infusion of sgx-001 is administered intravenously.

    The infusion is given as a single dose; the exact volume and concentration are defined by the manufacturing process.

  6. Step 6

    Immediate post‑infusion monitoring

    After the infusion, you will be closely monitored for vital signs, laboratory parameters, electrocardiograms, and any adverse events.

    This monitoring continues for several hours to ensure safety before discharge.

  7. Step 7

    Inpatient recovery and early safety assessments

    You will remain in the hospital until neutrophil recovery is observed, defined as a neutrophil count reaching the predefined threshold by day 42 after infusion.

    Platelet recovery is also tracked, with assessments continuing through month 12.

  8. Step 8

    Outpatient follow‑up visits

    After discharge, scheduled outpatient visits occur at month 1, month 2, month 3, month 6, month 9, and month 12.

    Each visit includes physical examination, vital sign measurement, laboratory tests, electrocardiograms, and collection of blood samples to measure transgene levels and nadhp oxidase activity.

  9. Step 9

    Assessment of treatment response

    At month 12, the primary efficacy endpoint is evaluated by measuring nadhp oxidase activity in peripheral blood granulocytes; a response is defined as activity in at least 10% of cells.

    Additional secondary assessments such as survival, infection rates, and need for antibacterial or antifungal prophylaxis are also recorded.

  10. Step 10

    Final study evaluation

    The study concludes with a comprehensive review of safety and efficacy data collected through month 12.

    Findings include overall survival, incidence of graft failure, and any long‑term adverse events.

Who can join the trial?

10 criteria

  • Have properly completed and signed informed consent (or assent if you are a minor) showing you understand the study.
  • Agree to stay in the study for up to 15 years for long‑term follow‑up requirements.
  • Have a confirmed diagnosis of Chronic Granulomatous Disease (CGD) caused by p47phox deficiency, proven by a genetic test that shows a mutation in the NCF1 gene.
  • Show absent or more than 95 % reduced activity of the enzyme NADPH oxidase in a special laboratory test called a dihydrorhodamine flow cytometric test.
  • Be at least 18 months old and weigh at least 10 kg at the time you sign the consent.
  • Have one or more ongoing or recurrent severe infections or inflammatory problems, as decided by the investigator.
  • Do not have an available fully matched (10/10) sibling donor for a stem cell transplant (HSCT).
  • Be able to return to the study site for follow‑up visits during the first year and to a local transplant center for later monitoring.
  • If you are a female who could become pregnant, you must have a negative serum pregnancy test performed within 3 days before each mobilization cycle and within 5 days before the infusion, and you must not be pregnant, lactating (breast‑feeding), or planning a pregnancy during the study period.
  • If you are a male with a female partner who could become pregnant, you must use highly effective birth control methods during the study and for the required time after treatment.

Who cannot join the trial?

19 criteria

  • Unable or unwilling to follow the study rules and visits required by the trial.
  • Cannot stop taking interferon‑gamma (a medication that helps the immune system) at least 30 days before the study treatment.
  • Has taken part in another interventional clinical study within the past 6 months.
  • Has any other health problem that the doctor believes could make the study unsafe, interfere with following the plan, or affect the study results.
  • Does not have a willing sibling who is a perfect HLA‑matched donor (a genetic match needed for a certain type of transplant) unless the risk of that transplant is too high.
  • Has had a previous allogeneic HSCT (a stem‑cell transplant using cells from another person).
  • Is pregnant or breastfeeding.
  • Has any condition that makes the following procedures unsafe:
    • Low haemoglobin (blood iron‑carrying protein) less than 8 g/dL, unstable heart or blood pressure (cardiovascular instability), or severe bleeding problems (coagulopathy) that would prevent moving the CD34⁺ cells (a type of stem cell) into the bloodstream.
    • Problems that would prevent the apheresis procedure (a process that collects blood cells).
    • Issues that would make the chemotherapy‑like preparation (conditioning regimen) unsafe.
    • Has a known allergy or other reason that prevents receiving the study drug or any part of it.
    • Has any of the following infections at the same time: HIV (human immunodeficiency virus), hepatitis B or hepatitis C viruses, adenovirus, parvovirus B19, human T‑lymphotropic virus, or toxoplasmosis (a parasite infection).
    • Has an active cancer that has spread (metastatic) or is locally advanced, including blood cancers (haematologic malignancy), and doctors expect survival to be less than 3 years.
    • Has severe organ problems such as:
      • Needing a breathing machine (mechanical ventilation).
      • Very weak heart function (heart shortening fraction on ultrasound less than 25%).
      • Kidney failure that requires dialysis (dialysis dependence).
      • Uncontrolled seizure disorder (seizures that cannot be managed).
      • A major birth defect (congenital anomaly).
      • Expected survival of less than 6 months.
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Investigated drugs

autologous CD34+ cells transduced with a lentiviral vector encoding the human NCF1 gene
This therapy uses the patient’s own blood stem cells (called CD34+ cells). The cells are taken out, then a harmless virus is used to add a working copy of the NCF1 gene, which is missing or faulty in people with a type of chronic granulomatous disease (CGD). After the gene is inserted, the modified cells are given back to the patient through an infusion. The goal is for these corrected cells to grow and produce healthy immune cells that can fight infections better, helping to treat CGD caused by p47phox deficiency. The treatment is given once after the patient receives strong chemotherapy (myeloablative conditioning) to prepare the body for the new cells.

What is already known about the treatment

autologous CD34+ cells transduced with a lentiviral vector encoding the human NCF1 gene - The product is a sterile liquid of the patient’s own CD34+ stem cells, which are given by intravenous infusion. It is an experimental gene‑therapy medicine still in early clinical trials and not yet approved for routine use. It is designed for people with chronic granulomatous disease caused by p47phox deficiency, a condition that makes them prone to infections. The stem cells are modified with a harmless virus that adds a normal NCF1 gene, allowing the cells to produce the missing protein and help the immune system kill bacteria; the product is classified as an autologous cell‑based gene therapy.

Investigated diseases

Chronic granulomatous disease (p47phox deficiency) - This is a genetic condition that affects the function of certain white blood cells. The defect reduces the cells’ ability to produce chemicals that kill bacteria and fungi. As a result, people may experience repeated infections, especially in the lungs, skin, and lymph nodes. Over time, the immune system can form small clumps of immune cells called granulomas, which may cause swelling or blockages. The disease often begins in early childhood and can become more noticeable as infections accumulate.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2025-524423-50-00Protocol codeSGX-001-CT-01Estimated enrolment5 patients

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