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A Study of Gene Therapy Using Modified Stem Cells for Patients with Transfusion-Dependent Beta-Thalassemia Who Need Regular Blood Transfusions

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What is this trial about?

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

This study is looking at a treatment for transfusion-dependent beta-thalassemia, which is a blood disorder where the body cannot make enough healthy red blood cells on its own, requiring patients to receive regular blood transfusions to survive. The main treatment being tested is FT007, which is made from the patient's own CD34+ cells that have been modified using a lentiviral vector that carries instructions for making a healthy form of beta-globin, a protein needed for red blood cells to work properly. This type of treatment is called gene therapy because it aims to correct the genetic problem causing the disease. In addition to the main treatment, patients will also receive other medications as part of the process, including Busulfan, which is a type of chemotherapy used to prepare the body for receiving the modified cells, Plerixafor, which helps collect stem cells from the blood, and Lenograstim, which supports the production of white blood cells during recovery.

The purpose of this study is to find out if the gene therapy treatment can help patients become independent from blood transfusions. Before the treatment, doctors will collect stem cells from the patient's own blood, modify these cells in a laboratory to add the working gene, and then give them back to the patient through an infusion into a vein. The study will follow patients for several years after they receive the treatment to check if they no longer need regular blood transfusions and to monitor their safety.

The study will measure success mainly by counting how many patients can go for at least twelve continuous months without needing any blood transfusions while maintaining a healthy level of hemoglobin in their blood. Doctors will also track the overall survival of patients and carefully watch for any side effects or safety concerns related to the treatment. The study includes both children and adults with this condition who have been receiving regular blood transfusions.

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

    Stem cell collection preparation

    Your body will be prepared for the collection of stem cells. Stem cells are special cells in your bone marrow that produce blood cells.

    You will receive lenograstim, a medication that helps move stem cells from your bone marrow into your bloodstream. This medication will be given as an injection under your skin, into your muscle, or through a vein.

    You may also receive plerixafor, another medication that helps release stem cells into your bloodstream. This medication will be given as an injection under your skin.

  2. Step 2

    Stem cell collection

    Your CD34+ stem cells will be collected from your blood through a process called apheresis. This involves removing blood from your body, separating out the stem cells, and returning the remaining blood to you.

    The collected stem cells belong to you and will be modified in a laboratory.

  3. Step 3

    Laboratory modification of stem cells

    Your collected stem cells will be sent to a specialized laboratory where they will be modified.

    The cells will be treated with a lentiviral vector that adds a working copy of the human beta-globin gene to your stem cells. This gene is important for producing healthy red blood cells.

    This process is called transduction and uses an improved laboratory method.

    During this time, you will not be in the hospital. The laboratory process takes several weeks.

  4. Step 4

    Preparation for stem cell transplant

    Before receiving your modified stem cells back, you will undergo a preparation treatment called conditioning.

    You will receive busulfan, a medication that removes your existing bone marrow cells to make room for the modified stem cells.

    Busulfan will be given through a vein as an infusion. The medication is provided as a concentrate that will be mixed into a solution before being given to you.

    This conditioning treatment is necessary to allow your modified stem cells to settle in your bone marrow and start producing new blood cells.

  5. Step 5

    Infusion of modified stem cells

    Your modified stem cells will be returned to your body through a single infusion into your vein.

    The modified cells are called FT007 or autologous CD34+ cells transduced with lentiviral vector encoding the human beta-globin gene.

    This is a one-time treatment. You will receive only one dose of the modified cells.

    The infusion is the main treatment of this gene therapy study.

  6. Step 6

    Recovery period

    After receiving your modified stem cells, you will need time for your body to recover and for the new cells to begin working.

    During the first 60 days after the infusion, you may still need red blood cell transfusions to support your body while the modified stem cells settle into your bone marrow and start producing new blood cells.

    Transfusions given during this 60-day period are considered support transfusions and are part of the recovery process.

  7. Step 7

    Monitoring for transfusion independence

    Starting from 60 days after your last support transfusion, your doctors will monitor whether you achieve transfusion independence.

    Transfusion independence means that your hemoglobin level (the protein in your blood that carries oxygen) stays at or above 9.0 grams per deciliter without needing any red blood cell transfusions.

    The goal is for you to remain free from transfusions for at least 12 continuous months.

    Your hemoglobin levels will be measured regularly to track your progress.

  8. Step 8

    Follow-up at 12 months

    At 12 months after receiving the modified stem cells, your overall health and the effectiveness of the treatment will be assessed.

    Your doctors will evaluate whether the gene therapy is working and monitor for any side effects.

    Blood tests and other examinations will be performed to check how well your body is producing healthy red blood cells.

  9. Step 9

    Follow-up at 24 months

    At 24 months after receiving the modified stem cells, another comprehensive assessment will be conducted.

    Your doctors will continue to monitor your overall health, the long-term effectiveness of the treatment, and any potential side effects.

    This follow-up is important to understand how well the gene therapy continues to work over time.

  10. Step 10

    Long-term follow-up

    You will continue to be monitored beyond 24 months to assess the long-term safety and effectiveness of the gene therapy.

    Regular visits and tests will be scheduled to ensure your health is maintained and to detect any late effects of the treatment.

    The study is expected to continue until October 2030.

Who can join the trial?

8 criteria

  • You must be diagnosed with transfusion-dependent beta-thalassemia, which is a blood disorder where your body does not make enough healthy red blood cells and you need regular blood transfusions. You must have a specific type called homozygous or compound heterozygous, meaning you inherited the condition from both parents.
  • Your medical records must show that you received at least 100 milliliters per kilogram of body weight per year or at least 10 units per year of packed red blood cells, which are concentrated blood cells given through transfusions, in each of the 2 years before joining the study.
  • For Group 1, you must be between 18 and 35 years old. For Group 2, you must be between 3 and 35 years old.
  • Your Karnofsky Index or Lansky score must be at least 80 percent. This is a measure of how well you can perform daily activities and take care of yourself.
  • Your heart, kidneys, liver, and lungs must be working well enough to safely undergo an autologous HSCT, which is a procedure where your own stem cells (special cells that can develop into blood cells) are collected and given back to you after treatment.
  • You must have a low risk for developing blood clots based on screening tests, and you must not have had any serious blood clotting problems in the past.
  • If your genetic testing was not done in a certified laboratory, the testing will need to be repeated during the screening period at the clinical site.
  • Both males and females can participate in this study.

Who cannot join the trial?

3 criteria

  • The study information does not list specific reasons why patients cannot participate in this clinical trial
  • If you have questions about whether you can join this study, you will need to discuss your individual medical situation with the research team during the screening process, which is when doctors check if you meet the requirements to participate
  • General factors that often prevent participation in clinical trials may include having certain other medical conditions, taking specific medications, or having had previous treatments, but these specific details are not provided in the available study information
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Investigated drugs

FT007 is a gene therapy treatment that uses the patient's own blood stem cells (called CD34+ hematopoietic stem cells). These cells are collected from the patient, then modified in a laboratory using a special tool called the GLOBE lentiviral vector. This vector helps insert working genetic material into the cells to help them produce healthy red blood cells. After the cells are modified using an improved process, they are given back to the patient through an infusion. The goal is to help patients with transfusion-dependent beta-thalassemia reduce or eliminate their need for regular blood transfusions.

What is already known about the treatment

FT007 – FT007 is an experimental gene therapy treatment currently being studied in clinical trials for patients with transfusion-dependent beta-thalassemia, a serious inherited blood disorder where the body cannot produce enough healthy red blood cells. The therapy works by collecting a patient's own blood stem cells (CD34+ cells), modifying them in a laboratory using a special virus called GLOBE lentiviral vector to insert correct genetic instructions, and then infusing these corrected cells back into the patient's body where they can produce healthy red blood cells. This one-time treatment aims to help patients become independent from regular blood transfusions, which are currently their main form of treatment. The therapy is administered as a single infusion directly into the bloodstream after the modified stem cells have been prepared.

Investigated diseases

Transfusion-Dependent Beta-Thalassemia – This is an inherited blood disorder where the body cannot produce enough functional hemoglobin, which is the protein in red blood cells that carries oxygen throughout the body. People with this condition have mutations in the genes responsible for making beta-globin, one of the building blocks of hemoglobin. As a result, their red blood cells are smaller and break down more quickly than normal. The bone marrow tries to compensate by producing more red blood cells, but they are still abnormal and insufficient. This leads to severe anemia, meaning the body does not have enough healthy red blood cells to supply adequate oxygen to tissues. Patients require regular blood transfusions throughout their lives to maintain hemoglobin levels and prevent complications from chronic anemia.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2025-522160-32-00Estimated enrolment9 patientsSponsorFondazione Telethon Ets

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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).