Luspatercept plus low-dose thalidomide for adults with transfusion-dependent β-thalassemia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 2, multicenter, randomized, double-blind, placebo-controlled trial enrolling adults with transfusion-dependent β-thalassemia. Participants are centrally randomized 1:1 to receive luspatercept plus low-dose thalidomide or luspatercept plus placebo through a screening/run-in period, a 24-week double-blind treatment period and a follow-up period. The primary endpoint is the proportion of subjects who reduce RBC transfusion burden by ≥50% and at least 2 units during weeks 13–24 versus their 12-week baseline. Secondary outcomes include other efficacy measures, iron metabolism, hemolysis markers, safety, and adverse event rates.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* 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 Criteria:

* 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.
* 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.

Where it's running

• Southern Medical University Shenzhen Hospital
  Shenzhen, Guangdong, China Recruiting
• Affiliated Hospital of Youjiang Medical College for Nationalities
  Baise City, Guangxi, China Recruiting
• Baise People's Hospital
  Baise City, Guangxi, China Recruiting
• Liuzhou People's Hospital
  Liuzhou, Guangxi, China Recruiting
• Liuzhou Workers' Hospital
  Liuzhou, Guangxi, China Recruiting
• Yulin First People's Hospital
  Yulin, Guangxi, China Recruiting

Study contacts

• Study coordinator: Rongrong Liu
• Email: liurongrong@stu.gxmu.edu.cn
• Phone: +86 0771 5356510

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Using a monoclonal antibody to prevent graft-versus-host disease in thalassemia patients after stem cell transplantation

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.