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Evaluating the Clinical Efficacy and Safety of Luspatercept Combined With Thalidomide in the Treatment of β-TDT Patients
Led by Rongrong Liu · Updated on 2026-01-13
78
Participants Needed
8
Research Sites
99 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Evaluating the Clinical Efficacy and Safety of Luspatercept Combined With Thalidomide in the Treatment of β-TDT Patients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older, any gender
- Diagnosed with transfusion-dependent beta-thalassemia
- Planned treatment with luspatercept combined with low-dose thalidomide or luspatercept alone
- Requires regular red blood cell transfusions totaling 6 to 30 units within 24 weeks before randomization, with transfusion intervals no longer than 42 days
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
- Voluntary participation with signed informed consent from the patient or legal guardian
You will not qualify if you...
- Diagnosis of alpha-thalassemia minor, Hb Bart's edema, hemoglobin S/beta-thalassemia, or myelodysplastic anemia (combination of beta-thalassemia and alpha-thalassemia is allowed)
- Anemia due to nutritional deficiency, chronic disease, autoimmune hemolytic anemia, or other hemolytic anemias like severe G6PD deficiency or pyruvate kinase deficiency
- Frequent bleeding disorders such as menorrhagia, epistaxis, or coagulopathy
- Hemolysis not related to thalassemia within the past 8 weeks, including from certain medications
- Use of long-term anticoagulant therapy unless stopped at least 28 days before randomization (some exceptions apply)
- Use of thalidomide alone, erythropoiesis-stimulating agents, or hydroxyurea within the last 24 weeks
- Use of long-term systemic glucocorticoids within the past 12 weeks
- Use of cytotoxic drugs, immunosuppressants, or investigational drugs within the past 28 days
- Positive for HIV or active hepatitis B or C infection
- Significant liver or kidney dysfunction as defined by lab tests
- History of malignancy unless cured and inactive
- Pregnant, planning pregnancy, or breastfeeding
- Prior gene therapy or hematopoietic stem cell transplantation for thalassemia
- Platelet count below 70 x 10^9/L (unless due to hypersplenism) or above 1000 x 10^9/L
- Any other condition the investigator believes makes participation unsuitable
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Trial Site Locations
Total: 8 locations
1
Southern Medical University Shenzhen Hospital
Shenzhen, Guangdong, China, 518100
Actively Recruiting
2
Affiliated Hospital of Youjiang Medical College for Nationalities
Baise City, Guangxi, China, 533000
Actively Recruiting
3
Baise People's Hospital
Baise City, Guangxi, China, 533000
Actively Recruiting
4
Liuzhou People's Hospital
Liuzhou, Guangxi, China, 545007
Actively Recruiting
5
Liuzhou Workers' Hospital
Liuzhou, Guangxi, China, 545007
Actively Recruiting
6
Yulin First People's Hospital
Yulin, Guangxi, China, 537000
Actively Recruiting
7
Yunnan Provincial First People's Hospital
Kunming, Yunnan, China, 650100
Actively Recruiting
8
The First Affiliated Hospital of Guangxi Medical University
Naning, China, 530000
Actively Recruiting
Research Team
R
Rongrong Liu
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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