Transfusion-dependent β-thalassemia PatientsPhase 2NCT07338344
Evaluating the Clinical Efficacy and Safety of Luspatercept Combined With Thalidomide in the Treatment of β-TDT Patients
A Phase 2 study testing Luspatercept combined with low-dose thalidomide in people with Transfusion-dependent β-thalassemia Patients, run by Rongrong Liu at 8 sites in China.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Transfusion-dependent β-thalassemia Patients
- What is being tested?
- Luspatercept combined with low-dose thalidomide
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 78 participants
- Where
- 8 sitesChina
- Study dates
- 1 Feb 2026to 31 Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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);
- 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);
- 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.
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- Use of long-term systemic glucocorticoids within the past 12 weeks.
- Use of cytotoxic drugs, immunosuppressants, or other investigational drugs within the past 28 days.
- HIV positive and/or active HCV or HBV infection.
- 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);
- Women who are pregnant, plan to become pregnant during the study, or are breastfeeding;
- Previous thalassemia gene therapy or hematopoietic stem cell transplantation (HSCT);
- Platelet count < 70 × 109/L, if not associated with hypersplenism, or platelet count > 1,000 × 109/L;
- Other conditions deemed unsuitable for participation in this clinical trial by the investigator.
Trial locations and contacts
8 sites in 1 country. Contact the study team or a site near you to ask about taking part.
8 sites in 1 country
China8 sites, 8 recruiting
- Recruiting
Affiliated Hospital of Youjiang Medical College for Nationalities
Baise City, Guangxi, 533000
Xiaochao Wang
- Recruiting
Baise People's Hospital
Baise City, Guangxi, 533000
Yan Huang
- Recruiting
Yunnan Provincial First People's Hospital
Kunming, Yunnan, 650100
Yajie Wang
- Recruiting
Liuzhou People's Hospital
Liuzhou, Guangxi, 545007
Qiangqiang Zhao, +86 07714712364
- Recruiting
Liuzhou Workers' Hospital
Liuzhou, Guangxi, 545007
Chunjie Qin, +86 0772 3805890, qinchunjie1@163.com
- Recruiting
The First Affiliated Hospital of Guangxi Medical University
Naning, 530000
Rongrong Liu, +86 0771 5356510, liurongrong@stu.gxmu.edu.cn
- Recruiting
Southern Medical University Shenzhen Hospital
Shenzhen, Guangdong, 518100
Zhiqiang Sun
- Recruiting
Yulin First People's Hospital
Yulin, Guangxi, 537000
Jun Wan
About this study
Official title: 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.
Read the detailed description
This is a prospective, multicenter, randomized, double-blind, placebo-controlled clinical trial designed to evaluate the clinical efficacy and safety of luspatercept combined with low-dose thalidomide versus luspatercept combined with placebo in adult patients with β-thalassemia requiring regular red blood cell transfusions.
The study is divided into the following phases: screening/run-in period, double-blind treatment period, and follow-up period. Participants are centrally randomized in a 1:1 ratio to either the intervention group or the control group. The primary objective is to compare the clinical response rate between the intervention group (luspatercept combined with low-dose thalidomide) and the control group (luspatercept combined with placebo) in adult patients with transfusion-dependent β-thalassemia. Clinical response is defined as the proportion of subjects achieving a reduction in red blood cell (RBC) transfusion burden by ≥50% and at least 2 units during weeks 13-24 after randomization compared with the baseline period (12 weeks prior to randomization). Secondary objectives mainly include assessments of other clinical efficacy indicators, iron metabolism, hemolysis, as well as the incidence of adverse events.
Statistical analyses in this clinical study are based on the Intention-To-Treat (ITT) principle. Subgroup analyses of the primary endpoint are planned according to baseline transfusion burden (low, medium, and high transfusion burden groups).
The study plans to consecutively enroll 78 participants across eight research centers: the First Affiliated Hospital of Guangxi Medical University, Liuzhou People's Hospital, Liuzhou Worker's Hospital, Yulin First People's Hospital, Affiliated Hospital of Youjiang Medical University for Nationalities, Baise People's Hospital,Yunnan Provincial First People's Hospital and Southern Medical University Shenzhen Hospital. During the screening and run-in period, patients with severe β-thalassemia major (β-TM) who are scheduled to receive luspatercept combined with low-dose thalidomide or luspatercept monotherapy are invited to participate. Written informed consent is provided to potential subjects, along with a detailed explanation of the study content. Written informed consent from the subject (or their legal representative) must be obtained before any study-specific procedures are conducted. After signing the informed consent form, baseline data are collected. During the double-blind treatment period, the intervention group receives luspatercept combined with low-dose thalidomide, while the control group receives luspatercept combined with placebo. Both groups may receive best supportive care, including RBC transfusions, iron chelators, antiplatelet therapy, antibiotic therapy, antiviral and antifungal therapy, and/or nutritional support as needed. During the follow-up period, subjects are followed up at weeks 12, 24, 36, and 48. Data on physical examination, vital signs, hematological tests, clinical biochemistry, transfusion status, iron parameters, quality of life, concomitant treatments, subject compliance, and adverse events are collected and recorded in detail for further analysis of clinical efficacy and safety.
- Being tested
- Luspatercept combined with low-dose thalidomide, Luspatercept plus placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Luspatercept combined with low-dose thalidomideDrug
The intervention group was treated with Luspatercept (starting dose level 1.0 mg/kg, once every 21 days) combined with low-dose thalidomide (starting dose level 50 mg/d) for 48 weeks.
Luspatercept plus placeboDrug
The control group was treated with Luspatercept (starting dose level 1.0 mg/kg every 21 days) plus placebo (starting dose level 50 mg/d) for 48 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Luspatercept(Reblozyl) combined with low-dose thalidomide | Experimental | The subjects in the intervention group were treated with Luspatercept (starting dose 1.0 mg/kg, once every 21 days) combined with low-dose thalidomide (starting dose level 50 mg/d) for 48 weeks. Receives: Drug: Luspatercept combined with low-dose thalidomide |
| Luspatercept plus placebo | Placebo comparator | The control group was treated with Luspatercept (starting dose level 1.0 mg/kg every 21 days) plus placebo (starting dose level 50 mg/d) for 48 weeks. Receives: Drug: Luspatercept plus placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants Who Achieved Erythroid Response - Week 13 to Week 24
Percentage of participants with a ≥50% reduction in RBC transfusion burden compared to baseline (RBC transfusion burden during the 12 weeks before randomization) and a reduction of at least 2 units for 12 consecutive weeks between Weeks 13 and 24 after randomization
Measured: Baseline: Day -83 to Day 1; Treatment: Weeks 13 to Week 24
Secondary outcomes (15)
Percentage of Participants Who Achieved ≥ 50% And a Reduction of ≥ 2 RBC units From Baseline in Transfusion Burden- Week 37 to Week 48
Percentage of participants who achieved a ≥50% reduction in RBC transfusion burden relative to baseline, with a reduction of ≥ 2 RBC units, for 12 consecutive weeks between 37 and 48 weeks after randomization.
Measured: Baseline: Day -83 to Day 1; Treatment: Weeks 37 to Week 48
Percentage Of Participants Who Achieved ≥ 33% And a Reduction of ≥ 2 RBC units Reduction From Baseline in Transfusion Burden - Week 37 to Week 48
Percentage Of participants who achieved a ≥33% reduction in RBC transfusion burden relative to baseline, with a reduction of ≥ 2 RBC units, for 12 consecutive weeks between 37 and 48 weeks after randomization.
Measured: Baseline: Day -83 to Day 1; Treatment: Weeks 37 to Week 48
Percentage Of Participants Who Achieved ≥ 50% Reduction From Baseline in Transfusion Burden - weeks 1-12, 13-24, 25-36, 37-48
Percentage Of participants who achieved a ≥50% reduction in RBC transfusion burden relative to baseline (RBC transfusion burden during the 12 weeks prior to randomization) for any consecutive 12 weeks (weeks 1-12, 13-24, 25-36, 37-48).
Measured: Baseline: Day -83 to Day 1; Treatment: Weeks1-12, 13-24, 25-36, 37-48
Percentage Of Participants Who Achieved ≥ 33% Reduction From Baseline in Transfusion Burden - weeks 1-12, 13-24, 25-36, 37-48
Percentage Of participants who achieved a ≥33% reduction in RBC transfusion burden relative to baseline (RBC transfusion burden during the 12 weeks prior to randomization) for any consecutive 12 weeks (weeks 1-12, 13-24, 25-36, 37-48).
Measured: Baseline: Day -83 to Day 1; Treatment: Weeks1-12, 13-24, 25-36, 37-48
Percentage Of Participants Who Achieved ≥ 50% Reduction From Baseline in Transfusion Burden - weeks 1-24, 25-48
Percentage Of participants with a ≥50% reduction in RBC transfusion burden relative to baseline (RBC transfusion burden during the 12 weeks prior to randomization) during any 24 consecutive weeks (weeks 1-24, 25-48);
Measured: Baseline: Day -83 to Day 1; Treatment: Weeks1-24, 25-48
Percentage Of Participants Who Achieved ≥ 33% Reduction From Baseline in Transfusion Burden - weeks 1-24, 25-48
Percentage Of participants with a ≥33% reduction in RBC transfusion burden relative to baseline (RBC transfusion burden during the 12 weeks prior to randomization) during any 24 consecutive weeks (weeks 1-24, 25-48);
Measured: Baseline: Day -83 to Day 1; Treatment: Weeks1-24, 25-48
Maximum percentage change in transfusion burden from baseline over 12 consecutive weeks
Percentage Of participants change in RBC transfusion burden relative to baseline (RBC transfusion burden during the 12 weeks prior to randomization) during any 12 consecutive weeks (weeks 1-12, 13-24, 25-36, 37-48);
Measured: Baseline: Day -83 to Day 1; Treatment: Weeks1-12, 13-24, 25-36, 37-48
Percentage of Participants Who Maintained Red Blood Cell (RBC) Transfusion Independence For ≥8 Weeks
Percentage of Participants who became transfusion-independent during any 8 consecutive weeks (weeks 1-8, 9-16, 17-24, 25-32, 33-40, 41-48);
Measured: weeks 1-8, 9-16, 17-24, 25-32, 33-40, 41-48
Percentage of Participants Who Maintained Red Blood Cell (RBC) Transfusion Independence For ≥12 Weeks
Percentage of Participants who became transfusion-independent during any 12 consecutive weeks (weeks 1-12, 13-24, 25-36, and 37-48);
Measured: weeks 1-12, 13-24, 25-36, and 37-48
Maximum percentage change from baseline in the number of transfusion events over 12 consecutive weeks
Maximum percentage change from baseline in the number of transfusion events (RBC transfusion burden during the 12 weeks prior to randomization) during any 12 consecutive weeks (weeks 1-12, 13-24, 25-36, and 37-48);
Measured: Baseline: Day -83 to Day 1; Treatment: Weeks1-12, 13-24, 25-36, 37-48
Concentration Change From Baseline to End of Treatment in serum ferritin
Concentration change from baseline in serum ferritin at weeks 12, 24, 36, and 48 after randomization;
Measured: Baseline (prior to first dose of study drug) and Treatment (weeks 12,24,36,48)
Change From Baseline to End of Treatment in Iron deposition in organs
Change from baseline in myocardial iron and liver iron concentrations at weeks 24 and 48 after randomization;
Measured: Baseline (prior to first dose of study drug) and Treatment (weeks 24,48)
Change From Baseline to End of Treatment in the average daily dose of iron chelation therapy
Change from baseline in the average daily dose of iron chelation therapy (ICT) during the treatment period (up to 48 weeks);
Measured: Baseline (prior to first dose of study drug) and Treatment (weeks 1-48)
Change from baseline in self-reported Health-related quality-of-life (HRQoL) assessed by TranQoL
The TranQol is a self-administered quality of life tool developed for beta-thalassemia patients. The adult self-report version used in this study, includes 36 questions assessed on a 5-point response, that are grouped into 5 domains (Physical Health, Emotional Health, Sexual Health, Family Functioning, School/Career Functioning). Scores are calculated according to specific scoring algorithms developed by the authors. Both individual domains score and the total score range from 0 (worst) to 100 (best). Total Score and Physical Health domain score are reported. Positive change from baseline values indicate improvement.
Measured: Baseline (prior to first dose of study drug) and Treatment (weeks 24,48)
Change from baseline in self-reported Health-related quality-of-life (HRQoL) assessed by SF-36
The SF-36 (version 2) is a generic, self-administered instrument consisting of 8 multi-item scales that assess 8 health domains. The raw score for each health domain is transformed into a 0 (worst) to 100 (best) domain score. The 0-100 scale score for each health domain is further converted to normbased scores using a T-score transformation, with a mean of 50 and a standard deviation (SD) of 10. Higher norm-based T-scores indicate better heath/QoL. The domains/summaries reported are: 1. Physical Functioning (Range of possible T-scores is 19.26 - 57.54) 2. General Health (Range of possible T-scores is 18.95 - 66.50) 3. Physical Component summary (PCS) (Range of possible T-scores is 5.02 - 79.78). Positive change from baseline values indicate improvement.
Measured: Baseline (prior to first dose of study drug) and Treatment (weeks 24,48)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2026
- Study start1 Feb 2026 (estimated)
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transfusion-dependent β-thalassemia PatientsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 78 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Rongrong Liu
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Rongrong Liu, First Affiliated Hospital of Guangxi Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07338344
- Sponsor's protocol ID
- 2025-K0444
- Status last verified
- Jan 2026
- First submitted
- 6 Dec 2025
- Last updated
- 13 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07338344
- Acronym / sponsor ID
- 2025-K0444
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 78 participants (estimated)
- Lead sponsor
- Rongrong LiuUniversity, hospital or other
- Study start
- 1 Feb 2026 (estimated)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 13 Jan 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is a prospective, multicenter, randomized, double-blind, placebo-controlled clinical trial designed to evaluate the clinical efficacy and safety of luspatercept combined with low-dose thalidomide versus luspatercept combined with placebo in adult patients with β-thalassemia requiring regular red blood cell transfusions.
The study is divided into the following phases: screening/run-in period, double-blind treatment period, and follow-up period. Participants are centrally randomized in a 1:1 ratio to either the intervention group or the control group. The primary objective is to compare the clinical response rate between the intervention group (luspatercept combined with low-dose thalidomide) and the control group (luspatercept combined with placebo) in adult patients with transfusion-dependent β-thalassemia. Clinical response is defined as the proportion of subjects achieving a reduction in red blood cell (RBC) transfusion burden by ≥50% and at least 2 units during weeks 13-24 after randomization compared with the baseline period (12 weeks prior to randomization). Secondary objectives mainly include assessments of other clinical efficacy indicators, iron metabolism, hemolysis, as well as the incidence of adverse events.
Statistical analyses in this clinical study are based on the Intention-To-Treat (ITT) principle. Subgroup analyses of the primary endpoint are planned according to baseline transfusion burden (low, medium, and high transfusion burden groups).
The study plans to consecutively enroll 78 participants across eight research centers: the First Affiliated Hospital of Guangxi Medical University, Liuzhou People's Hospital, Liuzhou Worker's Hospital, Yulin First People's Hospital, Affiliated Hospital of Youjiang Medical University for Nationalities, Baise People's Hospital,Yunnan Provincial First People's Hospital and Southern Medical University Shenzhen Hospital. During the screening and run-in period, patients with severe β-thalassemia major (β-TM) who are scheduled to receive luspatercept combined with low-dose thalidomide or luspatercept monotherapy are invited to participate. Written informed consent is provided to potential subjects, along with a detailed explanation of the study content. Written informed consent from the subject (or their legal representative) must be obtained before any study-specific procedures are conducted. After signing the informed consent form, baseline data are collected. During the double-blind treatment period, the intervention group receives luspatercept combined with low-dose thalidomide, while the control group receives luspatercept combined with placebo. Both groups may receive best supportive care, including RBC transfusions, iron chelators, antiplatelet therapy, antibiotic therapy, antiviral and antifungal therapy, and/or nutritional support as needed. During the follow-up period, subjects are followed up at weeks 12, 24, 36, and 48. Data on physical examination, vital signs, hematological tests, clinical biochemistry, transfusion status, iron parameters, quality of life, concomitant treatments, subject compliance, and adverse events are collected and recorded in detail for further analysis of clinical efficacy and safety.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Luspatercept(Reblozyl) combined with low-dose thalidomide | Experimental | Drug: Luspatercept combined with low-dose thalidomideThe subjects in the intervention group were treated with Luspatercept (starting dose 1.0 mg/kg, once every 21 days) combined with low-dose thalidomide (starting dose level 50 mg/d) for 48 weeks. |
| Luspatercept plus placebo | Placebo comparator | Drug: Luspatercept plus placeboThe control group was treated with Luspatercept (starting dose level 1.0 mg/kg every 21 days) plus placebo (starting dose level 50 mg/d) for 48 weeks. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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;
Key exclusion criteria (14 listed)
- 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);
- 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);
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Luspatercept combined with low-dose thalidomideThe intervention group was treated with Luspatercept (starting dose level 1.0 mg/kg, once every 21 days) combined with low-dose thalidomide (starting dose level 50 mg/d) for 48 weeks. | Drug | Luspatercept(Reblozyl) combined with low-dose thalidomide |
| Luspatercept plus placeboThe control group was treated with Luspatercept (starting dose level 1.0 mg/kg every 21 days) plus placebo (starting dose level 50 mg/d) for 48 weeks. | Drug | Luspatercept plus placebo |
Primary outcome
Percentage of Participants Who Achieved Erythroid Response - Week 13 to Week 24
Time frame: Baseline: Day -83 to Day 1; Treatment: Weeks 13 to Week 24
Percentage of participants with a ≥50% reduction in RBC transfusion burden compared to baseline (RBC transfusion burden during the 12 weeks before randomization) and a reduction of at least 2 units for 12 consecutive weeks between Weeks 13 and 24 after randomization
15 secondary outcomes
Percentage of Participants Who Achieved ≥ 50% And a Reduction of ≥ 2 RBC units From Baseline in Transfusion Burden- Week 37 to Week 48
Time frame: Baseline: Day -83 to Day 1; Treatment: Weeks 37 to Week 48
Percentage Of Participants Who Achieved ≥ 33% And a Reduction of ≥ 2 RBC units Reduction From Baseline in Transfusion Burden - Week 37 to Week 48
Time frame: Baseline: Day -83 to Day 1; Treatment: Weeks 37 to Week 48
Percentage Of Participants Who Achieved ≥ 50% Reduction From Baseline in Transfusion Burden - weeks 1-12, 13-24, 25-36, 37-48
Time frame: Baseline: Day -83 to Day 1; Treatment: Weeks1-12, 13-24, 25-36, 37-48
Percentage Of Participants Who Achieved ≥ 33% Reduction From Baseline in Transfusion Burden - weeks 1-12, 13-24, 25-36, 37-48
Time frame: Baseline: Day -83 to Day 1; Treatment: Weeks1-12, 13-24, 25-36, 37-48
Percentage Of Participants Who Achieved ≥ 50% Reduction From Baseline in Transfusion Burden - weeks 1-24, 25-48
Time frame: Baseline: Day -83 to Day 1; Treatment: Weeks1-24, 25-48
Percentage Of Participants Who Achieved ≥ 33% Reduction From Baseline in Transfusion Burden - weeks 1-24, 25-48
Time frame: Baseline: Day -83 to Day 1; Treatment: Weeks1-24, 25-48
Maximum percentage change in transfusion burden from baseline over 12 consecutive weeks
Time frame: Baseline: Day -83 to Day 1; Treatment: Weeks1-12, 13-24, 25-36, 37-48
Percentage of Participants Who Maintained Red Blood Cell (RBC) Transfusion Independence For ≥8 Weeks
Time frame: weeks 1-8, 9-16, 17-24, 25-32, 33-40, 41-48
Percentage of Participants Who Maintained Red Blood Cell (RBC) Transfusion Independence For ≥12 Weeks
Time frame: weeks 1-12, 13-24, 25-36, and 37-48
Maximum percentage change from baseline in the number of transfusion events over 12 consecutive weeks
Time frame: Baseline: Day -83 to Day 1; Treatment: Weeks1-12, 13-24, 25-36, 37-48
Concentration Change From Baseline to End of Treatment in serum ferritin
Time frame: Baseline (prior to first dose of study drug) and Treatment (weeks 12,24,36,48)
Change From Baseline to End of Treatment in Iron deposition in organs
Time frame: Baseline (prior to first dose of study drug) and Treatment (weeks 24,48)
Change From Baseline to End of Treatment in the average daily dose of iron chelation therapy
Time frame: Baseline (prior to first dose of study drug) and Treatment (weeks 1-48)
Change from baseline in self-reported Health-related quality-of-life (HRQoL) assessed by TranQoL
Time frame: Baseline (prior to first dose of study drug) and Treatment (weeks 24,48)
Change from baseline in self-reported Health-related quality-of-life (HRQoL) assessed by SF-36
Time frame: Baseline (prior to first dose of study drug) and Treatment (weeks 24,48)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jan 2026.
- Individual participant dataSharing planned: No
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Hemoglobinopathies (Transfusion-dependent β-thalassemia and Sickle Cell Disease)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Luspatercept combined with low-dose thalidomide in people with Transfusion-dependent β-thalassemia Patients, run by Rongrong Liu at 8 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 13 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Rongrong Liu (+86 0771 5356510, liurongrong@stu.gxmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants Who Achieved Erythroid Response - Week 13 to Week 24 (time frame: Baseline: Day -83 to Day 1; Treatment: Weeks 13 to Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Jan 2026.