Thalassemia MajorPhase 4NCT05462548
The Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-world Study
A Phase 4 study testing Luspatercept Injectable Product in people with Thalassemia Major and Transfusion-dependent Anemia, run by Sun Yat-sen University at 1 site in China.
Trial summary
This is a prospective, single-arm, open-label study. Twenty adult patients with transfusion-dependent β -thalassemia will be enrolled to receive Luspatercept with optimal supportive care, including blood transfusion and iron removal, based on the clinician's judgment and practice. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thalassemia Major, Transfusion-dependent Anemia
- What is being tested?
- Luspatercept Injectable Product
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- Jul 2022to 30 Apr 2023
- Phase
- Phase 4
- 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
- The patient was clearly diagnosed as transfusion-dependent β -thalassemia, the blood transfusion period was ≤60 days, and the red blood cell infusion volume was not less than 3-12U in the 12 weeks before enrollment, which could provide the red blood cell infusion volume record in the 12 weeks before enrollment
- Patients who were treated with thalidomide before enrollment were required to stop the treatment for more than 4 weeks, and their hemoglobin decreased to below 90g/L. Blood transfusion was required, and records of blood transfusion within 12 weeks could be provided
- Voluntarily participate in the study and sign the informed consent;
Who cannot join
- (1) Pregnant or lactating women
- persons known to be allergic to Luspatercept and/or Luspatercept excipients for injection
- Severe liver dysfunction (liver enzyme (ALT or AST) ≥3 TIMES ULN)
- Severe renal impairment (eGFR < 30 ml/min/1.73m3 or patients with end-stage renal disease)
- Heart disease, heart failure classified as Class 3 or higher by the New York Heart Association (NYHA), or severe arrhythmia requiring treatment, or recent myocardial infarction within 6 months of randomization.
- The patient has uncontrolled hypertension. According to NCI CTCAE version 5.0, controlled hypertension in this protocol is considered to be ≤1.
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- Patients with a history of deep vein thrombosis or stroke within 24 weeks prior to enrollment
- Erythropoietin, Luspatercept or hydroxyurea were used 8 weeks before enrollment
- Any other significant medical condition, laboratory abnormality, or mental illness that the Investigator deems inappropriate for inclusion.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
The second affiliated hospital of Sun Yat-sen University
Guangzhou, Guangdong, 510000
Jianpei Fang, M.D., +8620-81332003, jpfang2005@163.com
Jianpei Fang, M.D.
About this study
This is a prospective, single-arm, open-label study. Twenty adult patients with transfusion-dependent β -thalassemia will be enrolled to receive Luspatercept with optimal supportive care, including blood transfusion and iron removal, based on the clinician's judgment and practice. The main objective of this study was to evaluate the efficacy and safety of Luspatercept in the treatment of adult patients with transfusion-dependent β -thalassaemia in Chinese clinical practice, and to provide evidence reference for subsequent clinical use.
Read the detailed description
Twenty adult patients with transfusion-dependent β -thalassemia will be enrolled to receive Luspatercept with optimal supportive care, being injected subcutaneously every 3 weeks ,by the dose of 1-1.25 mg/kg. The main objective of this study was to evaluate the efficacy and safety of Luspatercept in the treatment of adult patients with transfusion-dependent β -thalassaemia in Chinese clinical practice, and to provide evidence reference for subsequent clinical use.
- Conditions
- Thalassemia Major, Transfusion-dependent Anemia
- Being tested
- Luspatercept Injectable Product
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Luspatercept Injectable ProductDrug
Also called luspatercept
1-1.25mg/kg every 3 weeks subcutaneous injection
| Study group | Role | What they receive |
|---|---|---|
| treatment group | Experimental | accept Luspatercept treatment Receives: Drug: Luspatercept Injectable Product |
What is being measured
The primary outcome is the main question the study is designed to answer.
A 33% reduction in the red cell (RBC) transfusion burden for any 12 consecutive weeks within 24 weeks of Luspatercept treatment compared with baseline
the incidence of 33% reduction in RBC transfusion
Measured: 24 weeks
The incidence of adverse events within 24 consecutive weeks of Luspatercept treatment
incidence of reported adverse events
Measured: 24weeks
Secondary outcomes (7)
the incidence of 50% reduction in transfusion in any 12 consecutive weeks
the incidence of 50% reduction in transfusion
Measured: 24 weeks
Changes in mean cumulative transfusion volume from baseline at weeks 1-9, 1-12, and 1-24
volume of transfusion
Measured: 24 weeks
The rate of transfusion free at any 8 and 12 consecutive weeks in the entire study population
volume of transfusion
Measured: 24 weeks
Changes in mean serum ferritin (SF) levels in the population from baseline
serum ferritin (SF) levels
Measured: 24 weeks
Changes of cardiac and liver iron concentrations at 24 weeks from baseline (MRI T2*)
MR T2*
Measured: 24 weeks
changes in reticulocyte levels from baseline during treatment
blood routine
Measured: 24 weeks
changes in fetal hemoglobin levels from baseline during treatment
Hemoglobin electrophoresis
Measured: 24weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jul 2022
- Study startJul 2022 (estimated)
- Primary completion28 Feb 2023 (estimated)
- Study completion30 Apr 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thalassemia MajorStudies of the same condition, open and under way.
- More trials run by Sun Yat-sen UniversityThe sponsor's other registered studies.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- 18 years old or above
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sun Yat-sen University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jianpei FANG, Sun Yat-sen University
- Investigators
- Jianpei Fang, Dr., Study director, SunYat-senU2H
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05462548
- Sponsor's protocol ID
- SYSKY-2022-088-02
- Status last verified
- Jul 2022
- First submitted
- 7 Jul 2022
- Last updated
- 18 Jul 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: privacy information of individual participant is not available
Study at a glance
From the registry record, last updated by the sponsor on 18 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-world Study
- ClinicalTrials.gov ID
- NCT05462548
- Acronym / sponsor ID
- SYSKY-2022-088-02
- Conditions
- Thalassemia Major, Transfusion-dependent Anemia
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Sun Yat-sen UniversityUniversity, hospital or other
- Study start
- Jul 2022 (estimated)
- Primary completion
- 28 Feb 2023 (estimated)
- Study completion
- 30 Apr 2023 (estimated)
- First posted
- 18 Jul 2022 (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.
This is a prospective, single-arm, open-label study. Twenty adult patients with transfusion-dependent β -thalassemia will be enrolled to receive Luspatercept with optimal supportive care, including blood transfusion and iron removal, based on the clinician's judgment and practice. The main objective of this study was to evaluate the efficacy and safety of Luspatercept in the treatment of adult patients with transfusion-dependent β -thalassaemia in Chinese clinical practice, and to provide evidence reference for subsequent clinical use.
Detailed description
Twenty adult patients with transfusion-dependent β -thalassemia will be enrolled to receive Luspatercept with optimal supportive care, being injected subcutaneously every 3 weeks ,by the dose of 1-1.25 mg/kg. The main objective of this study was to evaluate the efficacy and safety of Luspatercept in the treatment of adult patients with transfusion-dependent β -thalassaemia in Chinese clinical practice, and to provide evidence reference for subsequent clinical use.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- 18 years old or above
| Arm | Type | Receives |
|---|---|---|
| treatment group | Experimental | Drug: Luspatercept Injectable Productaccept Luspatercept treatment |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- -≥18 years old
- The patient was clearly diagnosed as transfusion-dependent β -thalassemia, the blood transfusion period was ≤60 days, and the red blood cell infusion volume was not less than 3-12U in the 12 weeks before enrollment, which could provide the red blood cell infusion volume record in the 12 weeks before enrollment
- Patients who were treated with thalidomide before enrollment were required to stop the treatment for more than 4 weeks, and their hemoglobin decreased to below 90g/L. Blood transfusion was required, and records of blood transfusion within 12 weeks could be provided
- Voluntarily participate in the study and sign the informed consent;
Key exclusion criteria (9 listed)
- (1) Pregnant or lactating women
- persons known to be allergic to Luspatercept and/or Luspatercept excipients for injection
- Severe liver dysfunction (liver enzyme (ALT or AST) ≥3 TIMES ULN)
- Severe renal impairment (eGFR < 30 ml/min/1.73m3 or patients with end-stage renal disease)
- Heart disease, heart failure classified as Class 3 or higher by the New York Heart Association (NYHA), or severe arrhythmia requiring treatment, or recent myocardial infarction within 6 months of randomization.
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 |
|---|---|---|
| Luspatercept Injectable ProductAlso: luspatercept1-1.25mg/kg every 3 weeks subcutaneous injection | Drug | treatment group |
Primary outcome
A 33% reduction in the red cell (RBC) transfusion burden for any 12 consecutive weeks within 24 weeks of Luspatercept treatment compared with baseline
Time frame: 24 weeks
the incidence of 33% reduction in RBC transfusion
The incidence of adverse events within 24 consecutive weeks of Luspatercept treatment
Time frame: 24weeks
incidence of reported adverse events
7 secondary outcomes
the incidence of 50% reduction in transfusion in any 12 consecutive weeks
Time frame: 24 weeks
Changes in mean cumulative transfusion volume from baseline at weeks 1-9, 1-12, and 1-24
Time frame: 24 weeks
The rate of transfusion free at any 8 and 12 consecutive weeks in the entire study population
Time frame: 24 weeks
Changes in mean serum ferritin (SF) levels in the population from baseline
Time frame: 24 weeks
Changes of cardiac and liver iron concentrations at 24 weeks from baseline (MRI T2*)
Time frame: 24 weeks
changes in reticulocyte levels from baseline during treatment
Time frame: 24 weeks
changes in fetal hemoglobin levels from baseline during treatment
Time frame: 24weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jul 2022.
- Individual participant dataSharing planned: Yes. privacy information of individual participant is not available
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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.
Jianpei Fang, Dr. study director
SunYat-senU2H
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Luspatercept Injectable Product in people with Thalassemia Major and Transfusion-dependent Anemia, run by Sun Yat-sen University 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 4 inclusion and 9 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 18 Jul 2022. 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.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 Jianpei Fang, MD (008613682290830, jpfang2005@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
A 33% reduction in the red cell (RBC) transfusion burden for any 12 consecutive weeks within 24 weeks of Luspatercept treatment compared with baseline (time frame: 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 18 Jul 2022; AzTrials last refreshed its copy on 8 Nov 2026.