- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT06164821
Clinical Observation of Luspatercept in Treatment of Chinese Adult β-thalassaemia Patients With TD β-thalassemia
Efficary and Safety of Luspatercept for Treatment of Anaemia in Transfusion-dependent β-thalassaemia in Southwest China
Study Overview
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.
Study Type
Enrollment (Estimated)
Phase
- Not Applicable
Contacts and Locations
Study Contact
- Name: Chaoen Zhen, MD
- Phone Number: +86 18087991755
- Email: zce163163@163.com
Study Contact Backup
- Name: Liu Liu, MD
- Phone Number: +86 0871-64774206
Study Locations
-
-
Yunnan
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Kunming, Yunnan, China, 650000
- Recruiting
- 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
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Contact:
- Lin Liu
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-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Adult
- Older Adult
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- ≥18 years old;
- 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);
- Voluntarily participate in the study and sign the informed consent;
Exclusion Criteria:
- pregnant or lactating women;
- Allergic to luspatercept and/or luspatercept for injection excipients;
- Severe liver dysfunction: Liver enzymes (alanine aminotransferase ALT or aspartate aminotransferase AST) ≥ 3 times normal value.;
- Severe renal injury: eGFR<30 ml/min/1.73m3 or end-stage renal disease;
- 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;
- The patient had uncontrolled hypertension;
- Patients with a history of deep vein thrombosis or stroke within 24 weeks prior to enrollment.;
- Treatment with ESA, luspatercept, thalidomide or hydroxyurea within 12 weeks before enrollment;
- Any significant other medical condition, laboratory abnormality, or mental illness;
- Investigators deemed enrollment inappropriate.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: 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.
|
Adult patients with TD thalassemia were given luspatercept subcutaneous injection.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of units in cumulative transfusion volume
Time Frame: Within 24 weeks
|
The decrease of cumulative blood transfusion volume in low transfusion burden group (<7.5 units /24 weeks) and high transfusion burden group (7.5-15 units /24 weeks) after 24 weeks of treatment;
|
Within 24 weeks
|
Proportion of patients with a 33% reduction in transfusion burden
Time Frame: Within 24 weeks
|
33% reduction in blood transfusion burden at week 24 in low transfusion burden group (<7.5 units /24 weeks) and high transfusion burden group (7.5-15 units /24 weeks);
|
Within 24 weeks
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Blood transfusion burden reduction ratio
Time Frame: Within 24 weeks
|
The proportion of RBC transfusion burden reduced by 50% at any 12 weeks; (2) transfusion independence (TI) rates at any 8-week and any 12-week in the whole study population; |
Within 24 weeks
|
Ratio of TI
Time Frame: Within 24 weeks
|
Transfusion independence (TI) rates at any 8 weeks and at any 12 weeks in the entire study population;
|
Within 24 weeks
|
Other Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Security Index
Time Frame: Within 33 weeks
|
Vital signs, laboratory measures, adverse events (AES), and serious adverse events (SAEs)
|
Within 33 weeks
|
Collaborators and Investigators
Study record dates
Study Major Dates
Study Start (Estimated)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- B-thal01
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
product manufactured in and exported from the U.S.
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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