Trial studying safety and effects for Transfusion-dependent β-thalassemia Patients
Official title Evaluating the Clinical Efficacy and Safety of Luspatercept Combined With Thalidomide in the Treatment of β-TDT Patients
ClinicalTrials.gov ID: NCT07338344
What this study is testing
What is Luspatercept combined with low-dose thalidomide?
An investigational combination therapy being studied for transfusion-dependent β‑thalassemia
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The study compares luspatercept with or without low‑dose thalidomide to see if the combination reduces how often patients need transfusions and to check for any medical problems that might arise.
- Who it's for
- Adults who have transfusion‑dependent β‑thalassemia and need regular blood transfusions.
- What's involved
- Participants receive either luspatercept plus low‑dose thalidomide or luspatercept plus a placebo. They will have regular clinic visits and blood tests to monitor transfusion needs and safety.
- Time commitment
- The trial lasts several months, with scheduled visits for medication, blood tests, and safety monitoring throughout the study period.
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 75
You may be able to join if
- 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...
- ECOG performance status 0-1;
You likely can't join if
- A diagnosis of α-thalassemia minor, Hb Bart's edema, hemoglobin S/β-thalassemia, or myelodysplastic anemia (combination of β-thalassemia and...
- Anemia related to nutritional deficiency, anemia of chronic disease, autoimmune hemolytic anemia, or any other hemolytic anemia (e.g., severe G6PD...
- 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...
- Use of long-term anticoagulant therapy, unless discontinued at least 28 days before randomization.
- Use of thalidomide alone, erythropoiesis-stimulating drugs (ESA), or hydroxyurea within the past 24 weeks.
See the full eligibility 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.
- 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.
- 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)
- 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 \ 1,000 × 109/L;
- Other conditions deemed unsuitable for participation in this clinical trial by the investigator.
The study team makes the final eligibility decision.
Where it is enrolling
- Shenzhen, Guangdong, China
- Baise City, Guangxi, China
- Liuzhou, Guangxi, China
- Yulin, Guangxi, China
- Kunming, Yunnan, China
- Naning, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 75 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Shenzhen, Guangdong, China; Baise City, Guangxi, China; Liuzhou, Guangxi, China; Yulin, Guangxi, China; Kunming, Yunnan, China; Naning, China. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.