Ongoing Clinical Trials for Alpha Thalassaemia
Currently, there are 2 ongoing clinical trials investigating new treatments for alpha thalassaemia. These studies are testing medications designed to help manage iron overload and improve red blood cell production in patients who are transfusion-dependent or have significant anemia. The trials are taking place in Italy, Greece, and Denmark.
Clinical trial locations
- Denmark
- Greece
- Italy
Study of SP-420 for Patients with Transfusion-Dependent Alpha or Beta Thalassemia
This trial is testing an oral medication called SP-420 to help manage iron overload in people with transfusion-dependent thalassaemia. Regular blood transfusions are necessary for many patients with this condition, but these transfusions can lead to a build-up of excess iron in the body, which can cause serious health problems if not managed properly.
Main inclusion criteria: Adults aged 18 years or older with transfusion-dependent α-thalassemia or β-thalassemia (including HbE/β-thalassemia) who need treatment to remove excess iron are eligible. Participants must weigh at least 35 kg and have been on a stable dose of iron chelation therapy for at least 4 weeks before the study. They must also have significant iron overload, measured as a liver iron concentration between 5 and 35 mg/g dry weight on MRI scan. Participants need to have been monitored at a specialized center for at least 6 months with detailed medical records.
Main exclusion criteria: People who are not transfusion-dependent, do not have α- or β-thalassemia, are outside the specified age range, or cannot follow study procedures are excluded. Pregnant or breastfeeding individuals, those with other interfering medical conditions, those taking medications that might affect results, recent participants in other clinical trials, and individuals with a history of allergic reactions to the study medication are also excluded.
Focus and goal: The trial aims to find the right dose of SP-420 and evaluate how well it removes excess iron from the body over 24 weeks. Participants will take SP-420 capsules orally, and the study will last up to 48 weeks. Iron levels will be measured at different times using MRI scans to track how much iron is removed from the liver. Researchers will also monitor any side effects to ensure the treatment is safe.
Investigational drug: SP-420 is an iron chelator being tested to see if it can effectively reduce iron levels in people with transfusion-dependent thalassaemia. It works by modulating iron metabolism to help balance iron levels in the body.
Study on Luspatercept for Treating Anemia in Adults and Adolescents with Alpha-Thalassemia
This trial is studying a medication called luspatercept (also known as BMS-986346/ACE-536) for treating anemia in people with alpha-thalassaemia. The medication is designed to help the body produce more red blood cells, potentially reducing the need for blood transfusions and improving quality of life.
Main inclusion criteria: Adults aged 18 years or older, or adolescents aged 12 to less than 18 years, with a documented diagnosis of alpha-thalassemia HbH disease are eligible. For transfusion-dependent adults, at least 6 red blood cell units must have been received in the 24 weeks before joining, with no transfusion-free period longer than 56 days. Non-transfusion-dependent adults must have received less than 6 units during that time, been transfusion-free for at least 8 weeks, and have a mean hemoglobin level of 10 g/dL or less. Similar criteria apply to adolescents, with at least 4 transfusion events required for transfusion-dependent participants. All participants must have a performance status score of 50 or higher.
Main exclusion criteria: Individuals with any other serious health condition that could interfere with the study, recent major surgery, current participation in another clinical trial, or history of allergic reactions to the study medication are excluded. People with uncontrolled high blood pressure, severe liver or kidney disease, pregnancy or breastfeeding status, history of drug or alcohol abuse, recent blood transfusions, or any condition the study doctors believe would make participation unsafe are also excluded.
Focus and goal: The trial evaluates how well luspatercept works in treating anemia in adults with alpha-thalassaemia HbH disease. It also assesses the safety and appropriate dosage in adolescents with the same condition. Participants will receive regular injections of luspatercept under the skin and be monitored closely. The study looks at the need for blood transfusions and hemoglobin levels over several months. The goal is to find a safe and effective treatment that reduces transfusion needs and improves overall quality of life.
Investigational drug: Luspatercept is an erythroid maturation agent that helps the body produce more red blood cells. It works by binding to certain proteins that regulate red blood cell production, enhancing the body’s ability to produce these cells and potentially improving anemia symptoms.
Summary
Both ongoing trials for alpha thalassaemia are concentrated in Southern Europe, with Italy and Greece hosting both studies, while Denmark participates in one. The two trials take different approaches to managing this condition: one focuses on iron chelation with SP-420 to address iron overload from transfusions, while the other tests luspatercept to improve red blood cell production and potentially reduce transfusion needs. Together, these studies address two major challenges faced by people with transfusion-dependent thalassaemia—managing excess iron and reducing dependence on blood transfusions. Both trials include careful monitoring and are designed to ensure participant safety while evaluating potential new treatment options.