Thalassaemia beta – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 11:23:32 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Thalassaemia beta – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Evaluation of the Efficacy, Safety, and Tolerability of Subcutaneous REGN7999 in Adults with Iron Overload Due to Non-Transfusion Dependent Beta-Thalassemia https://clinicaltrials.eu/trial/evaluation-of-the-efficacy-safety-and-tolerability-of-subcutaneous-regn7999-in-adults-with-iron-overload-due-to-non-transfusion-dependent-beta-thalassemia/ Wed, 29 Apr 2026 15:08:57 +0000 https://clinicaltrials.eu/trial/evaluation-of-the-efficacy-safety-and-tolerability-of-subcutaneous-regn7999-in-adults-with-iron-overload-due-to-non-transfusion-dependent-beta-thalassemia/ This clinical trial is focused on studying a condition known as non-transfusion dependent beta-thalassemia, which can lead to an excess of iron in the body, referred to as iron overload. The trial will test a new treatment called REGN7999, which is a type of protein designed to help manage iron levels in the body. REGN7999 is administered as an injection under the skin.

The purpose of the study is to evaluate how effective and safe REGN7999 is for people with iron overload due to non-transfusion dependent beta-thalassemia. Participants in the study will receive either REGN7999 or a matching placebo. The study will use MRI scans to measure the amount of iron in the liver, which is a key indicator of iron overload. The study will also monitor any side effects and how well participants tolerate the treatment.

Throughout the study, participants will receive regular injections and undergo MRI scans to track changes in their liver iron levels. The study aims to determine if REGN7999 can reduce iron levels and improve overall health outcomes for those with this condition. The trial will also assess changes in hemoglobin levels, which is an important component of red blood cells, and monitor the need for any additional blood transfusions.

]]> A Study of Gene Therapy Using Modified Stem Cells for Patients with Transfusion-Dependent Beta-Thalassemia Who Need Regular Blood Transfusions https://clinicaltrials.eu/trial/a-study-of-gene-therapy-using-modified-stem-cells-for-patients-with-transfusion-dependent-beta-thalassemia-who-need-regular-blood-transfusions/ Wed, 29 Apr 2026 15:08:51 +0000 https://clinicaltrials.eu/trial/a-study-of-gene-therapy-using-modified-stem-cells-for-patients-with-transfusion-dependent-beta-thalassemia-who-need-regular-blood-transfusions/ 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.

]]> Long-Term Safety and Efficacy Study of Betibeglogene Autotemcel Gene Therapy for Patients with Transfusion-Dependent Beta-Thalassemia https://clinicaltrials.eu/trial/long-term-safety-and-efficacy-study-of-betibeglogene-autotemcel-gene-therapy-for-patients-with-transfusion-dependent-beta-thalassemia/ Wed, 29 Apr 2026 14:59:34 +0000 https://clinicaltrials.eu/trial/long-term-safety-and-efficacy-study-of-betibeglogene-autotemcel-gene-therapy-for-patients-with-transfusion-dependent-beta-thalassemia/ This clinical trial focuses on the long-term follow-up of individuals with transfusion-dependent beta-thalassemia, a blood disorder that reduces the production of hemoglobin, the protein in red blood cells that carries oxygen. The study involves a treatment called LentiGlobin BB305 Drug Product, which is a type of gene therapy. This treatment uses a patient’s own blood stem cells, which are modified outside the body to include a new gene that helps produce a functional protein. These modified cells are then returned to the patient through an infusion.

The purpose of the study is to monitor the long-term safety and effectiveness of this gene therapy in individuals who have already received the treatment in previous studies. Participants will be observed over an extended period to assess any potential long-term health effects, such as the development of new or worsening health conditions, including immune-related issues or blood disorders. The study will also evaluate how well the treatment works over time, including its impact on reducing the need for blood transfusions and improving hemoglobin levels.

Throughout the study, participants will undergo regular check-ups to track their health and any changes in their condition. This includes monitoring the expression of the therapeutic gene in their blood and assessing their overall quality of life. The study aims to provide valuable information on the long-term benefits and risks of using LentiGlobin BB305 Drug Product for treating transfusion-dependent beta-thalassemia.

]]> Study on the Safety and Effectiveness of CRISPR-Cas9 Modified Stem Cells for Patients with Transfusion-Dependent Beta-Thalassemia Using Exagamglogene Autotemcel https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-crispr-cas9-modified-stem-cells-for-patients-with-transfusion-dependent-beta-thalassemia-using-exagamglogene-autotemcel/ Wed, 29 Apr 2026 14:59:32 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-crispr-cas9-modified-stem-cells-for-patients-with-transfusion-dependent-beta-thalassemia-using-exagamglogene-autotemcel/ This clinical trial is focused on studying a condition known as Transfusion-Dependent Beta-Thalassemia (TDT). TDT is a blood disorder that requires regular blood transfusions. The study is testing a new treatment called exagamglogene autotemcel, also known by its code name CTX001. This treatment involves using a technique called CRISPR-Cas9 gene editing to modify certain cells in the body. The goal is to help the body produce more of its own healthy blood cells, reducing the need for transfusions.

Participants in the study will receive a single dose of the modified cells. Before receiving the treatment, participants will undergo a process to prepare their body, which includes the use of a medication called busulfan. This medication helps create space in the bone marrow for the new cells to grow. The study will also involve the use of other medications, such as filgrastim and plerixafor, which help in collecting the necessary cells from the body. These medications are given through injections.

The purpose of the study is to evaluate the safety and effectiveness of this new treatment. Participants will be monitored closely for any side effects and to see how well the treatment works in reducing the need for blood transfusions. The study will track various health indicators over time, including how quickly the body starts producing its own blood cells and the overall health of the participants. The study aims to provide a new treatment option for people living with TDT, potentially improving their quality of life by reducing their dependence on regular blood transfusions.

]]> Study of SP-420 for Patients with Transfusion-Dependent Alpha or Beta Thalassemia https://clinicaltrials.eu/trial/study-of-sp-420-for-patients-with-transfusion-dependent-alpha-or-beta-thalassemia/ Wed, 29 Apr 2026 14:28:26 +0000 https://clinicaltrials.eu/trial/study-of-sp-420-for-patients-with-transfusion-dependent-alpha-or-beta-thalassemia/ This clinical trial is focused on studying a condition known as transfusion-dependent thalassemia, which includes both α-thalassemia and β-thalassemia. These are blood disorders that require regular blood transfusions. The trial will test a new treatment called SP-420, which is taken as a capsule. The purpose of the study is to find the right dose of SP-420 and to see how well it works in removing excess iron from the body over a period of 24 weeks.

Participants in the study will take SP-420 orally, which means they will swallow the capsules. The study will last for up to 48 weeks, during which the amount of iron in the body will be measured at different times. This will help researchers understand how much iron is removed by SP-420. The study will also monitor changes in the amount of iron in the liver using a method called magnetic resonance imaging (MRI), which is a type of scan that provides detailed images of the inside of the body.

Throughout the study, researchers will keep track of any side effects that participants might experience. The main goal is to determine how effective SP-420 is at reducing iron levels in people with transfusion-dependent thalassemia, while also ensuring the treatment is safe. Participants will be closely monitored by healthcare professionals to ensure their well-being during the trial.

]]> Study on the Effects of Exagamglogene Autotemcel for Patients with Transfusion-Dependent Beta-Thalassemia or Severe Sickle Cell Disease https://clinicaltrials.eu/trial/study-on-the-effects-of-exagamglogene-autotemcel-for-patients-with-transfusion-dependent-beta-thalassemia-or-severe-sickle-cell-disease/ Wed, 29 Apr 2026 14:27:23 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-of-exagamglogene-autotemcel-for-patients-with-transfusion-dependent-beta-thalassemia-or-severe-sickle-cell-disease/ This clinical trial is focused on studying two serious blood disorders: Transfusion-dependent β-thalassemia and Severe Sickle Cell Disease. These conditions often require regular blood transfusions and can lead to significant health challenges. The study is testing a new treatment called CTX001, which involves using a patient’s own blood stem cells that have been modified using a technology called CRISPR-Cas9. This technology aims to help the body produce more fetal hemoglobin, a type of hemoglobin that can reduce the symptoms of these diseases.

The purpose of the study is to evaluate the effectiveness and safety of a single dose of CTX001 in patients with these conditions. Participants will receive the treatment and be monitored over time to see how their levels of fetal hemoglobin change. The study will also look at the safety of the treatment by checking for any side effects and monitoring the health of the participants. Other medications used in the study include Filgrastim, Busulfan, and Plerixafor, which are used to prepare the body for the main treatment.

During the study, participants will receive the treatment through an infusion, which is a way of delivering medication directly into the bloodstream. They will be closely monitored by healthcare professionals to ensure their safety and to track the effectiveness of the treatment. The study aims to provide valuable information on whether this new approach can help reduce the need for blood transfusions and improve the quality of life for people with these challenging conditions.

]]> Study on Luspatercept for Improving Brain Oxygenation in Patients with Non-Transfusion Dependent Thalassemia https://clinicaltrials.eu/trial/study-on-luspatercept-for-improving-brain-oxygenation-in-patients-with-non-transfusion-dependent-thalassemia/ Wed, 29 Apr 2026 14:26:06 +0000 https://clinicaltrials.eu/trial/study-on-luspatercept-for-improving-brain-oxygenation-in-patients-with-non-transfusion-dependent-thalassemia/ This clinical trial is focused on studying a condition known as non-transfusion dependent beta-thalassemia, a type of blood disorder that affects the body’s ability to produce hemoglobin, the protein in red blood cells that carries oxygen. The treatment being tested in this study is called luspatercept, which is administered as a powder for solution for injection. Luspatercept is designed to help improve anemia, a condition where the body doesn’t have enough healthy red blood cells, in patients with this type of thalassemia.

The purpose of the study is to see if luspatercept can improve the way the brain uses oxygen in patients with non-transfusion dependent thalassemia. This will be assessed by measuring the cerebral metabolic rate of oxygen (CMRO2) using a special imaging technique called MRI. The study will also look at other factors, such as blood flow in the brain and heart function, over a period of 27 weeks. Participants will receive regular assessments, including blood tests and heart scans, to monitor their health and the effects of the treatment.

During the study, some participants will receive luspatercept, while others may receive a placebo. The study aims to determine if luspatercept can lead to an increase in hemoglobin levels by at least 1 gram per deciliter, which could potentially improve the symptoms of anemia in these patients. The trial is expected to run until early 2025, with recruitment starting in October 2023.

]]> Study on the Safety and Effects of Luspatercept for Children with Beta Thalassemia https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-luspatercept-for-children-with-beta-thalassemia/ Wed, 29 Apr 2026 14:23:58 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-luspatercept-for-children-with-beta-thalassemia/ This clinical trial is focused on studying a condition called Beta Thalassemia, which is a blood disorder that reduces the production of hemoglobin. Hemoglobin is an important part of red blood cells that carries oxygen throughout the body. The study is testing a treatment called Luspatercept, also known by its code name ACE-536. Luspatercept is a medication that is given as an injection under the skin and is designed to help increase the production of healthy red blood cells in people with Beta Thalassemia.

The purpose of this study is to find a safe and tolerable dose of Luspatercept for children with Beta Thalassemia who either need regular blood transfusions or do not. The study will also look at how the body processes the medication. Participants in the study will receive either Luspatercept or a placebo. The study will last for a period of time, during which participants will receive regular injections and attend follow-up visits to monitor their health and the effects of the treatment.

Throughout the study, researchers will collect information on the safety of Luspatercept, including any side effects that may occur. They will also measure changes in the need for blood transfusions and levels of hemoglobin in the participants. This information will help determine if Luspatercept is a beneficial treatment option for children with Beta Thalassemia. The study aims to provide valuable insights into managing this condition and improving the quality of life for those affected.

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