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

NCT ID: NCT06164821

Last Updated: 2023-12-22

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: NA
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-01-01
• Study Completion Date: 2024-11-30

Brief Summary

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

Detailed 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.

Conditions

Effect of Drug

Keywords

luspatercept; β-Thalassemia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

luspatercept arm

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 patients before each injection of luspatercept should be monitored, and the common adverse reactions (AE) should be monitored. 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. If the patient has blood transfusion, it is necessary to obtain the blood transfusion record from the hospital system.Concomitant use of iron chelating agents was also recorded.

Group Type: EXPERIMENTAL

luspatercept

Intervention Type: DRUG

Adult patients with TD thalassemia were given luspatercept subcutaneous injection.

Interventions

luspatercept

Adult patients with TD thalassemia were given luspatercept subcutaneous injection.

Intervention Type: DRUG

Eligibility Criteria

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 of red 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 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 aspartate aminotransferase 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, or the need for treatment Severe arrhythmia, or recent myocardial infarction within 6 months;

6. The patient had uncontrolled hypertension;

7. Patients with a history of deep vein thrombosis or stroke within 24 weeks prior to enrollment.;

8. Treatment with ESA, luspatercept, thalidomide or hydroxyurea within 12 weeks before enrollment;

9. Any significant other medical condition, laboratory abnormality, or mental illness;

10. Investigators deemed enrollment inappropriate.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Hematology department of the 920th hospital

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

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

Kunming, Yunnan, China

Site Status: RECRUITING

Countries

China

Central Contacts

Chaoen Zhen, MD

Role: CONTACT

Phone: 18087991755

Email: zce163163@163.com

Liu Liu, MD

Role: CONTACT

Phone: 0871-64774206

Facility Contacts

Lin Liu

Role: primary

Other Identifiers

B-thal01

Identifier Type: -

Identifier Source: org_study_id