Effect of DrugNCT06164821
Clinical Observation of Luspatercept in Treatment of Chinese Adult β-thalassaemia Patients With TD β-thalassemia
A study testing luspatercept in people with Effect of Drug, run by Hematology department of the 920th hospital at 1 site in China.
Trial summary
To assesse the efficacy and safety of luspatercept versus placebo in China patients with transfusion-dependent β-thalassaemia. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Effect of Drug
- What is being tested?
- luspatercept
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- 1 Jan 2024to 30 Nov 2024
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- ≥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;
Who cannot join
- 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;
Show all 10Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
Liu Liu, MD contact
China1 site, 1 recruiting
- Recruiting
920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
Kunming, Yunnan, 650000
Lin Liu
About this study
Official title: Efficary and Safety of Luspatercept for Treatment of Anaemia in Transfusion-dependent β-thalassaemia in Southwest China
To assesse the efficacy and safety of luspatercept versus placebo in China patients with transfusion-dependent β-thalassaemia.
Read the 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
- Being tested
- luspatercept
- Keywords
- luspatercept, β-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
luspaterceptDrug
Adult patients with TD thalassemia were given luspatercept subcutaneous injection.
| Study group | Role | What they receive |
|---|---|---|
| luspatercept arm | Experimental | 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. Receives: Drug: luspatercept |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of units in cumulative transfusion volume
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;
Measured: Within 24 weeks
Proportion of patients with a 33% reduction in transfusion burden
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);
Measured: Within 24 weeks
Secondary outcomes (2)
Blood transfusion burden reduction ratio
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;
Measured: Within 24 weeks
Ratio of TI
Transfusion independence (TI) rates at any 8 weeks and at any 12 weeks in the entire study population；
Measured: Within 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Dec 2023
- Study start1 Jan 2024 (estimated)
- Primary completion1 Jan 2024 (estimated)
- Study completion30 Nov 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Effect of DrugStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- 5 cases with transfusion burden\<7.5 units/24 weeks, and 5 cases with transfusion burden 7.5-15 units/24 weeks
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hematology department of the 920th hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06164821
- Sponsor's protocol ID
- B-thal01
- Status last verified
- Dec 2023
- First submitted
- 1 Dec 2023
- Last updated
- 22 Dec 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 22 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficary and Safety of Luspatercept for Treatment of Anaemia in Transfusion-dependent β-thalassaemia in Southwest China
- ClinicalTrials.gov ID
- NCT06164821
- Acronym / sponsor ID
- B-thal01
- Conditions
- Effect of Drug
- Keywords
- luspatercept, β-Thalassemia
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Hematology department of the 920th hospitalUniversity, hospital or other
- Study start
- 1 Jan 2024 (estimated)
- Primary completion
- 1 Jan 2024 (estimated)
- Study completion
- 30 Nov 2024 (estimated)
- First posted
- 11 Dec 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- 5 cases with transfusion burden<7.5 units/24 weeks, and 5 cases with transfusion burden 7.5-15 units/24 weeks
| Arm | Type | Receives |
|---|---|---|
| luspatercept arm | Experimental | Drug: luspaterceptLuspatercept 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- ≥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;
Key exclusion criteria (10 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| luspaterceptAdult patients with TD thalassemia were given luspatercept subcutaneous injection. | Drug | luspatercept arm |
Primary outcome
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;
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);
2 secondary outcomes
Blood transfusion burden reduction ratio
Time frame: Within 24 weeks
Ratio of TI
Time frame: Within 24 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Dec 2023.
- Individual participant dataSharing planned: Yes
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Liu Liu, MD contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing luspatercept in people with Effect of Drug, run by Hematology department of the 920th hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 22 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Chaoen Zhen, MD (18087991755 ext. +86, zce163163@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of units in cumulative transfusion volume (time frame: Within 24 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Dec 2023.