Thalassaemia – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Wed, 17 Jun 2026 04:02:38 +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 – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 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.

]]> Study on the Safety and Effectiveness of CTX001 for Children with Transfusion-Dependent Beta-Thalassemia Using Exagamglogene Autotemcel and a Drug Combination https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-ctx001-for-children-with-transfusion-dependent-beta-thalassemia-using-exagamglogene-autotemcel-and-a-drug-combination/ Wed, 29 Apr 2026 15:01:54 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-ctx001-for-children-with-transfusion-dependent-beta-thalassemia-using-exagamglogene-autotemcel-and-a-drug-combination/ This clinical trial is focused on studying a condition known as Transfusion-Dependent β-Thalassemia, a blood disorder that requires regular blood transfusions. The study is evaluating a new treatment called CTX001, which involves using a technique known as CRISPR-Cas9 gene editing. This technique modifies the patient’s own blood stem cells to help them produce a type of hemoglobin that can reduce or eliminate the need for blood transfusions.

The purpose of the study is to assess the safety and effectiveness of a single dose of CTX001 in children with this condition. Participants will receive a dose of their own modified stem cells, which have been altered to improve their ability to produce hemoglobin. The study will monitor the participants over time to see if they can maintain healthy hemoglobin levels without needing transfusions.

In addition to CTX001, the study involves other medications such as Filgrastim, Busulfan, and Plerixafor, which are used to prepare the body for the treatment and support the process. The study will take place over several years, with regular check-ups to ensure the safety and effectiveness of the treatment. Participants will be closely monitored for any side effects or changes in their condition.

]]> Study on the Effectiveness and Safety of Mitapivat for Patients with Non-Transfusion-Dependent Alpha or Beta Thalassemia https://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-mitapivat-for-patients-with-non-transfusion-dependent-alpha-or-beta-thalassemia/ Wed, 29 Apr 2026 15:01:32 +0000 https://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-mitapivat-for-patients-with-non-transfusion-dependent-alpha-or-beta-thalassemia/ This clinical trial is focused on studying a condition known as non-transfusion-dependent alpha- or beta-thalassemia. Thalassemia is a blood disorder that affects the body’s ability to produce hemoglobin, which is essential for carrying oxygen in the blood. People with this condition do not require regular blood transfusions. The study is testing a treatment called Mitapivat, which is taken in tablet form. Mitapivat is being compared to a placebo to see how effective it is in treating anemia, a condition where there is a lack of healthy red blood cells, in individuals with this type of thalassemia.

The purpose of the study is to evaluate the effectiveness and safety of Mitapivat in improving anemia in patients with non-transfusion-dependent thalassemia. Participants in the study will be randomly assigned to receive either Mitapivat or a placebo. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual medication and who is receiving the placebo. This helps ensure that the results are unbiased. The study will take place over several weeks, with regular check-ups to monitor the participants’ health and response to the treatment.

Throughout the study, the main goal is to observe if there is an increase in hemoglobin levels, which would indicate an improvement in anemia. Participants will be closely monitored to ensure their safety and to gather data on how well Mitapivat works compared to the placebo. This research aims to provide valuable information that could lead to better treatment options for individuals with non-transfusion-dependent thalassemia.

]]> Study on the Effects of Mitapivat for Patients with Transfusion-Dependent Alpha or Beta Thalassemia https://clinicaltrials.eu/trial/study-on-the-effects-of-mitapivat-for-patients-with-transfusion-dependent-alpha-or-beta-thalassemia/ Wed, 29 Apr 2026 15:01:17 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-of-mitapivat-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 alpha-thalassemia and beta-thalassemia. These are blood disorders that affect the body’s ability to produce normal hemoglobin, leading to a need for regular blood transfusions. The study is testing a treatment called Mitapivat, also known by its code name AG-348, which is taken in tablet form. Mitapivat is being compared to a placebo to see how it affects the need for blood transfusions in people with this condition.

The purpose of the study is to evaluate how effective and safe Mitapivat is for individuals with transfusion-dependent thalassemia. Participants in the study will be randomly assigned to receive either Mitapivat or a placebo. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual medication and who is receiving the placebo. This helps ensure that the results are unbiased. The study will take place over a period of time, during which participants will be monitored to see if there is a reduction in the number of blood transfusions they need.

Throughout the study, participants will have regular check-ups to monitor their health and the effects of the treatment. The main goal is to see if Mitapivat can reduce the number of blood transfusions needed by at least 50% over a 12-week period compared to the start of the study. This could potentially improve the quality of life for those living with transfusion-dependent thalassemia by reducing their reliance on blood transfusions.

]]> Study on Long-Term Safety of Ruxolitinib, Panobinostat, and Siremadlin for Patients Continuing Treatment from Previous Studies https://clinicaltrials.eu/trial/study-on-long-term-safety-of-ruxolitinib-panobinostat-and-siremadlin-for-patients-continuing-treatment-from-previous-studies/ Wed, 29 Apr 2026 14:59:37 +0000 https://clinicaltrials.eu/trial/study-on-long-term-safety-of-ruxolitinib-panobinostat-and-siremadlin-for-patients-continuing-treatment-from-previous-studies/ This clinical trial is focused on evaluating the long-term safety of treatments for patients who have previously participated in studies involving the medication ruxolitinib, either alone or in combination with other drugs. Ruxolitinib is a medication used to treat certain types of blood disorders. In this study, it may be combined with other medications such as panobinostat, siremadlin (also known as HDM201), or rineterkib. The purpose of the study is to gather information on the safety of these treatments over a longer period.

Participants in this study will continue their treatment with ruxolitinib or its combinations as they have been doing in their previous studies. The study will monitor the frequency and severity of any side effects or adverse events that occur during the treatment. This information will help researchers understand the long-term safety of these medications and their combinations.

The study is open-label, meaning that both the participants and the researchers know which treatment is being administered. It is conducted at multiple centers, allowing for a diverse group of participants. The study aims to provide valuable data on the safety of these treatments, which can benefit future patients who may receive these medications. Participants will be assessed regularly to determine any clinical benefits they may experience from the continued treatment.

]]> Long-term safety study of oral etavopivat tablets in children, adolescents and adults with sickle cell disease or thalassaemia who completed previous etavopivat treatment https://clinicaltrials.eu/trial/long-term-safety-study-of-etavopivat-for-adults-adolescents-and-children-with-sickle-cell-disease-or-thalassemia/ Wed, 29 Apr 2026 14:57:44 +0000 https://clinicaltrials.eu/trial/long-term-safety-study-of-etavopivat-for-adults-adolescents-and-children-with-sickle-cell-disease-or-thalassemia/ This clinical trial focuses on studying long-term treatment of sickle cell disease and thalassemia, which are inherited blood disorders that affect how red blood cells function in the body. The study uses etavopivat (also known as FT-4202), which is given as a 200 mg tablet taken by mouth. These conditions can cause severe pain, fatigue, and other serious health problems due to abnormal red blood cells.

The purpose of this research is to understand how safe and effective etavopivat is when used for an extended period in adults, teenagers, and children who have previously completed treatment with this medication in other studies. The medication will be given to participants who have shown improvement during their previous treatment with etavopivat.

During the study, patients may continue taking other medications they were already using for their condition, such as hydroxyurea, crizanlizumab, or L-glutamine, as long as their doses have remained stable. The study will monitor various aspects of the participants’ health, including any side effects, hospital stays, and changes in their blood cell measurements over time.

]]> Study on Long-Term Safety of Luspatercept for Patients with Myelodysplastic Syndrome, Beta-Thalassemia, or Myelofibrosis Who Previously Participated in Luspatercept Trials https://clinicaltrials.eu/trial/study-on-long-term-safety-of-luspatercept-for-patients-with-myelodysplastic-syndrome-beta-thalassemia-or-myelofibrosis-who-previously-participated-in-luspatercept-trials/ Wed, 29 Apr 2026 14:56:51 +0000 https://clinicaltrials.eu/trial/study-on-long-term-safety-of-luspatercept-for-patients-with-myelodysplastic-syndrome-beta-thalassemia-or-myelofibrosis-who-previously-participated-in-luspatercept-trials/ This clinical trial is focused on studying the long-term safety of a treatment called luspatercept, also known by its code name ACE-536 or BMS-986346. The study involves participants who have previously taken part in other trials using this treatment. The diseases being studied include myelodysplastic syndrome (MDS), beta-thalassemia, and myelofibrosis. These are conditions that affect the blood and bone marrow, leading to issues like anemia and other blood-related problems.

The purpose of this study is to evaluate the long-term safety of luspatercept, including monitoring for any progression to more serious conditions such as acute myeloid leukemia (AML) or the development of other types of cancer. Participants in this study will receive luspatercept as a solution for injection, which is administered under the skin. The study is designed as an open-label, single-arm, rollover study, meaning all participants will receive the treatment without a comparison group receiving a placebo.

Throughout the study, participants will be monitored for any adverse events or changes in their condition. The study will also track overall survival and any new growths that may develop. The maximum treatment period for participants is up to 360 days, during which they will continue to receive the treatment and be regularly assessed by healthcare professionals. This study aims to provide valuable information on the long-term effects and safety of luspatercept for individuals with these blood disorders.

]]> Long-term Safety Study of Exagamglogene Autotemcel for Patients with Sickle Cell Disease or Transfusion-Dependent Thalassemia https://clinicaltrials.eu/trial/long-term-safety-study-of-exagamglogene-autotemcel-for-patients-with-sickle-cell-disease-or-transfusion-dependent-thalassemia/ Wed, 29 Apr 2026 14:28:48 +0000 https://clinicaltrials.eu/trial/long-term-safety-study-of-exagamglogene-autotemcel-for-patients-with-sickle-cell-disease-or-transfusion-dependent-thalassemia/ This clinical trial is focused on studying two blood disorders: Sickle Cell Disease and Transfusion-dependent Thalassemia. These conditions affect the blood’s ability to carry oxygen effectively, leading to various health issues. The treatment being tested is called CTX001, which is a type of cell therapy. It involves using a technique known as CRISPR-Cas9 gene editing to modify a specific part of the patient’s own blood-forming stem cells. This modification aims to restore the natural production of a type of hemoglobin called fetal hemoglobin, which can help improve the symptoms of these diseases.

The purpose of this study is to evaluate the long-term safety of CTX001 in patients who have already received this treatment for either Sickle Cell Disease or Transfusion-dependent Thalassemia. Participants in the study will be monitored over an extended period to observe any new health issues, such as new cancers or blood disorders, and to track their overall health and well-being. The study will also look at changes in hemoglobin levels and the presence of the intended genetic changes in the blood and bone marrow over time.

Throughout the study, participants will undergo regular check-ups to ensure their safety and to gather information on how the treatment affects their health. This includes monitoring for any serious side effects and assessing the overall impact on their quality of life. The study is designed to provide valuable insights into the long-term effects of CTX001 and its potential benefits for individuals with these challenging blood disorders.

]]> 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.

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