Clinical Observation of Luspatercept in Treatment of Chinese Adult β-thalassaemia Patients With TD β-thalassemia

Last updated: December 19, 2023
Sponsor: Hematology department of the 920th hospital
Overall Status: Active - Recruiting

Phase

N/A

Condition

Thalassemia

Treatment

luspatercept

Clinical Study ID

NCT06164821
B-thal01
  • Ages > 18
  • All Genders

Study Summary

To assesse the efficacy and safety of luspatercept versus placebo in China patients with transfusion-dependent β-thalassaemia.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. ≥18 years old;
  2. A clear diagnosis of transfusion-dependent β-thalassemia (including αβ mixed type)with red blood cell transfusion ≤15u within 24 weeks before enrollment (one unit ofred blood cell in overseas clinical research is 200-350ml packed red blood cells,which should be converted according to Chinese clinical practice);
  3. Voluntarily participate in the study and sign the informed consent;

Exclusion

Exclusion Criteria:

  1. pregnant or lactating women;
  2. Allergic to luspatercept and/or luspatercept for injection excipients;
  3. Severe liver dysfunction: Liver enzymes (alanine aminotransferase ALT or aspartateaminotransferase AST) ≥ 3 times normal value.;
  4. Severe renal injury: eGFR<30 ml/min/1.73m3 or end-stage renal disease;
  5. heart disease, New York Heart Association (NYHA) class 3 or higher heart failure, orthe need for treatment Severe arrhythmia, or recent myocardial infarction within 6months;
  6. The patient had uncontrolled hypertension;
  7. Patients with a history of deep vein thrombosis or stroke within 24 weeks prior toenrollment.;
  8. Treatment with ESA, luspatercept, thalidomide or hydroxyurea within 12 weeks beforeenrollment;
  9. Any significant other medical condition, laboratory abnormality, or mental illness;
  10. Investigators deemed enrollment inappropriate.

Study Design

Total Participants: 10
Treatment Group(s): 1
Primary Treatment: luspatercept
Phase:
Study Start date:
January 01, 2024
Estimated Completion Date:
November 30, 2024

Study Description

This was a prospective, multicenter, open-label, multicenter clinical study to observe the efficacy and safety of luspatercept in the treatment of adult patients with transfusion-dependent β-thalassemia in Chinese clinical practice.

Luspatercept has been approved for listing in China for the treatment of that requires regular infusion of red blood cells and red blood cell infusion ≤ 15 units/24 weeks β- Adult patients with thalassemia. The BELIEVE study enrolled 117 patients of Asian descent from Malaysia, Thailand, Taiwan, Australia, the United States, the United Kingdom, and Canada. Efficacy results similar to those in the global intention-to-treat population were observed in Asian patients with a baseline transfusion load of 6 to 15 units per 24 weeks. The results in the Asian population provide valuable reference for the safety and efficacy of luspatercept in the Chinese population.

Ten adult patients with β-thalassemia were enrolled in this study,divided into two groups (5 cases with transfusion burden<7.5 units/24 weeks, and 5 cases with transfusion burden 7.5-15 units/24 weeks).Luspatercept was given once subcutaneously every 3 weeks for 24 weeks in the treatment period, . Luspatercept was started at 1·0 mg/kg with titration up to 1·25 mg/kg, or reduction in the event of toxicity or excessive haemoglobin concentration increase. During the treatment, the hemoglobin of the patients before each injection of luspatercept was monitored, and the common adverse reactions (AE) were monitored.The primary end point was the percentage of the LTB and HTB group patients who had a reduction in the transfusion burden of at least 33% and reduce blood transfusion unit from baseline group.According to the judgment and practice of clinicians, the best supportive treatment, including blood transfusion, iron chelation therapy, and anti-infection treatment, should be provided for patients receiving luspatercept treatment. Thus，this clinical trial is going to further explore its efficacy and safety.

Connect with a study center

  • 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China

    Kunming, Yunnan 650000
    China

    Active - Recruiting

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