Luspatercept with or without low-dose thalidomide for adults with transfusion-dependent β-thalassemia
Compares luspatercept plus thalidomide with luspatercept alone in adults with transfusion-dependent β-thalassemia.
Plain-language title generated from the registry record
Registry title: Evaluating the Clinical Efficacy and Safety of Luspatercept Combined With Thalidomide in the Treatment of β-TDT Patients
Key facts
- Where
- Baise City, Kunming, Liuzhou, Naning, Shenzhen, and Yulin, ChinaSee the 8 sites
- Phase
- Phase 2
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 75 years old?
Source: NCT07338344, who can take part
Where in China
- Affiliated Hospital of Youjiang Medical College for NationalitiesBaise City, ChinaTaking people
- Baise People's HospitalBaise City, ChinaTaking people
- Yunnan Provincial First People's HospitalKunming, ChinaTaking people
- Liuzhou People's HospitalLiuzhou, ChinaTaking people
- Liuzhou Workers HospitalLiuzhou, ChinaTaking people
- The First Affiliated Hospital of Guangxi Medical UniversityNaning, ChinaTaking people
- Southern Medical University Shenzhen HospitalShenzhen, ChinaTaking people
- Yulin First People's HospitalYulin, ChinaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Rongrong Liu
- Trial code
- NCT07338344
- Record updated
- 13 January 2026
- Original description
β-thalassemia is one of the most common inherited hemoglobinopathies worldwide and a major public health issue that severely impacts birth quality, human health, and social progress. Currently, there are limited clinical drugs specifically designed to treat patients with β-thalassemia. This clinical trial aims to evaluate the efficacy and safety of luspatercept combined with low-dose thalidomide compared with luspatercept alone in patients with thalassemia. Key questions to be answered include:Read full original descriptionShow less
Evaluation of the Clinical Efficacy and Safety of Luspatercept Combined With Low-dose Thalidomide Versus Luspatercept Alone in the Treatment of Adult Patients With Transfusion-dependent β-thalassemia
β-thalassemia is one of the most common inherited hemoglobinopathies worldwide and a major public health issue that severely impacts birth quality, human health, and social progress. Currently, there are limited clinical drugs specifically designed to treat patients with β-thalassemia. This clinical trial aims to evaluate the efficacy and safety of luspatercept combined with low-dose thalidomide compared with luspatercept alone in patients with thalassemia. Key questions to be answered include:
* Does luspatercept combined with low-dose thalidomide reduce the transfusion burden in patients with β-thalassemia major? * What medical problems may occur when patients receive luspatercept combined with low-dose thalidomide? In this clinical trial, participants were randomly assigned in a 1:1 ratio to either an intervention group (luspatercept combined with low-dose thalidomide) or a control group (luspatercept combined with placebo) using a central randomization system. The clinical efficacy and safety of the two groups were evaluated. The primary outcome measure was the clinical efficacy of luspatercept combined with low-dose thalidomide in reducing the transfusion burden in patients with β-thalassemia major.
Inclusion: Age ≥ 18 years, regardless of gender; Patients with transfusion-dependent β-thalassemia; Intended treatment with rotecept combined with low-dose thalidomide or rotecept alone; Requires regular red blood cell transfusions (6-30 RBC units (International Units) within 24 weeks prior to randomization, with a transfusion-free interval of ≤ 42 days); ECOG performance status 0-1; Patients (or legal guardians) voluntarily participate in the study and provide signed informed consent.
Exclusion: Hemolysis unrelated to thalassemia within the past 8 weeks, such as after use of hemolytic-inducing medications (e.g., antimalarials, nonsteroidal anti-inflammatory drugs [NSAIDs]); Use of long-term anticoagulant therapy, unless discontinued at least 28 days before randomization. Prophylactic anticoagulant therapy for surgery or high-risk procedures, as well as low-molecular-weight heparin and long-term aspirin therapy for superficial venous thrombosis, are permitted; Use of thalidomide alone, erythropoiesis-stimulating drugs (ESA), or hydroxyurea within the past 24 weeks; Use of cytotoxic drugs, immunosuppressants, or other investigational drugs within the past 28 days; Previous thalassemia gene therapy or hematopoietic stem cell transplantation (HSCT); Anemia related to nutritional deficiency, anemia of chronic disease, autoimmune hemolytic anemia, or any other hemolytic anemia (e.g., severe G6PD deficiency, pyruvate kinase deficiency); A bleeding disorder manifesting as frequent bleeding (e.g., menorrhagia, epistaxis, coagulopathy); HIV positive and/or active HCV or HBV infection; Other conditions deemed unsuitable for participation in this clinical trial by the investigator; A diagnosis of α-thalassemia minor, Hb Bart's edema, hemoglobin S/β-thalassemia, or myelodysplastic anemia (combination of β-thalassemia and α-thalassemia is permitted); Hepatic and renal insufficiency (liver insufficiency, i.e., aspartate aminotransferase (AST), alanine aminotransferase (ALT) ≥3× upper limit of normal (ULN); renal insufficiency, i.e., serum creatinine ≥3× upper limit of normal (ULN) or creatinine clearance less than 30). mL/min), history of malignancy (unless cured and/or with no known active disease); Platelet count < 70 × 109/L, if not associated with hypersplenism, or platelet count > 1,000 × 109/L; Use of long-term systemic glucocorticoids within the past 12 weeks; Women who are pregnant, plan to become pregnant during the study, or are breastfeeding.