Transfusion-dependent Non-severe Aplastic AnemiaNCT06964971
Treatment of Transfusion-dependent Nonsevere Aplastic Anemia With Luspatercept: a Multicenter Prospective Clinical Study
A study testing Luspatercept and Luspatercept combine with Deferasirox in people with Transfusion-dependent Non-severe Aplastic Anemia, run by The First Affiliated Hospital of Zhejiang Chinese Medical University at 1 site in China.
Trial summary
The goal of this clinical trial is to learn whether Luspatercept alone or in combination with Deferasirox can promote hematopoietic function in patients with transfusion-dependent non-severe aplastic anemia, as well as to assess the safety and efficacy of this treatment approach. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Transfusion-dependent Non-severe Aplastic Anemia
- What is being tested?
- Luspatercept and Luspatercept combine with Deferasirox
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 90 participants
- Where
- 1 siteChina
- Study dates
- 20 May 2025to 30 Jun 2027
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
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 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 1.Age >= 18 years; 2.According to the "Chinese Guidelines for the Diagnosis and Treatment of Aplastic Anemia (2022 Edition)", the patient must be diagnosed with transfusion-dependent non-severe aplastic anemia (TD-NSAA) and meet the requirement of erythroid hyperplasia in bone marrow aspiration from the posterior iliac crest and/or sternum being more than 15%; 3.If not newly diagnosed with TD-NSAA, and there are combined primary disease maintenance medications, the following conditions must be met:
- The patient has not received and does not consider HSCT or ATG treatment for at least the next six months;
- If maintaining oral immunosuppressive therapy, the course must be at least 6 months and assessed as ineffective;
- If maintaining androgen therapy, the course must be at least 3 months and assessed as ineffective;
- If maintaining recombinant human erythropoietin therapy, the course must be at least 3 months and assessed as ineffective;
- If maintaining thrombopoietin receptor agonist (TPO-RA) therapy, the duration must be >=6 months with confirmed inefficacy, and a washout period of >=1 month is required before study enrollment;
- If the above maintenance medication durations are not met, a washout period of at least 1 month is required; 4.Serum ferritin level >= 1000 ng/ml; 5.Complete whole exome sequencing and MDS/AA next-generation sequencing testing are required.
Who cannot join
- 1. Severe hepatic dysfunction (ALT or AST ≥ 3 × ULN); 2.Severe renal impairment (eGFR < 30 ml/min/1.73m² or patients with end-stage renal disease); 3.Cardiac disease, including New York Heart Association (NYHA) Class 3 or higher heart failure, or severe arrhythmia requiring treatment, or recent myocardial infarction within 6 months of randomization; 4.Patients with uncontrolled hypertension, with controlled hypertension according to NCI CTCAE version 5.0 considered as ≤ Grade 1 for this protocol; 5.Patients with a PNH clone > 1%; 6.Patients planning to become pregnant or who are pregnant; 7.Surgical or clinical conditions that may significantly alter drug absorption, distribution, metabolism, or excretion (e.g., gastritis, ulcers, history of gastrointestinal or rectal bleeding; history of major gastrointestinal surgery); 8.Patients carrying congenital bone marrow failure-related gene mutations (homozygous or heterozygous, regardless of whether they are pathogenic/benign/likely benign/ of uncertain significance).
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
The First Affiliated Hospital of Zhejiang Chinese Medical University
Hangzhou, Zhejiang, 310000
Hangping Ge, +86 13738092542, canthy1216@126.com
About this study
The goal of this clinical trial is to learn whether Luspatercept alone or in combination with Deferasirox can promote hematopoietic function in patients with transfusion-dependent non-severe aplastic anemia, as well as to assess the safety and efficacy of this treatment approach.
The main questions it aims to answer is:
whether the combination therapy of Luspatercept and Deferasirox can improve hemoglobin levels in these patients.
Participants will receive Luspatercept every 3 to 5 weeks based on hemoglobin response, undergo complete blood counts every 1 to 3 weeks, and receive other necessary evaluations as required.
- Being tested
- Luspatercept, Luspatercept combine with Deferasirox
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LuspaterceptDrug
The recommended starting dose of luspatercept is 1.5 mg/kg administered subcutaneously (SC) once every 3 weeks. Treatment response should be evaluated and dosage adjusted after at least 3 treatment cycles (9 weeks).
Luspatercept combine with DeferasiroxDrug
The recommended starting dose of luspatercept is 1.5 mg/kg administered subcutaneously (SC) once every 3 weeks. Treatment response should be evaluated and dosage adjusted after at least 3 treatment cycles (9 weeks).
For patient with platelet counts < 50×10⁹/L:
Deferasirox dosage: 8-10 mg/kg/day, orally.
For platelet counts ≥ 50×10⁹/L:
Deferasirox dosage: 20 mg/kg/day, orally. Discontinue deferasirox if serum ferritin drops below 500 μg/L.
| Study group | Role | What they receive |
|---|---|---|
| Treatment Group1 | Experimental | basic treatment regimen + Luspatercept alone Receives: Drug: Luspatercept |
| Treatment Group2 | Experimental | basic treatment regimen + Luspatercept combine with Deferasirox Receives: Drug: Luspatercept combine with Deferasirox |
What is being measured
The primary outcome is the main question the study is designed to answer.
Hemoglobin
Mild anemia: Defined as a hemoglobin level <120 g/L in adult males or <110 g/L in adult females, but >90 g/L.
Moderate anemia: Hemoglobin level between 60-90 g/L. Severe anemia: Hemoglobin level between 30-60 g/L. Extremely severe anemia: Hemoglobin level <30 g/L.
Measured: Baseline (1 month before treatment), treatment phase: 12 weeks (twice weekly in weeks 1 / 2, once weekly in week 3, once every 3 weeks ), mid-treatment phase: 12 weeks (once every 3 weeks), follow-up period: 48 weeks (once every 12 weeks).
Secondary outcomes (4)
platelets
Measured: Baseline (30 days before treatment), treatment phase: 12 weeks (twice weekly in weeks 1 / 2, once weekly in week 3, once every 3 weeks ), mid-treatment phase: 12 weeks (once every 3 weeks).
Ferritin
Measured: Baseline (30 days before treatment), treatment phase: 12 weeks ( once every 3 weeks ), mid-treatment phase: 12 weeks (once every 3 weeks)
Proportion of patients transfusion-free for ≥8 weeks and ≥12 weeks
Measured: treatment phase: 12 weeks (twice weekly in weeks 1 / 2, once weekly in week 3, once every 3 weeks ), mid-treatment phase: 12 weeks (once every 3 weeks)
Overall response rate (ORR; CR + PR) at Week 12 and Week 24 of treatment
ORR: Overall Response Rate (defined as the proportion of patients achieving complete response [CR] or partial response [PR]).
Measured: Week 12 and Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 May 2025
- Study start20 May 2025 (estimated)
- Primary completion20 May 2027 (estimated)
- Study completion30 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transfusion-dependent Non-severe Aplastic AnemiaStudies 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
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- Control Group: Patients will continue with their original basic treatment regimen for the disease (immunosuppressants, androgens（either alone or in combination）) under observation. Treatment Group: In addition to the original basic treatment for the disease, patients will be further divided based on whether iron chelation therapy is used, into two subgroups: Luspatercept monotherapy group and Luspatercept combined with deferasirox group.
- Masking
- None (open label)
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The First Affiliated Hospital of Zhejiang Chinese Medical University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06964971
- Sponsor's protocol ID
- 2025-KLS-046-02
- Status last verified
- Apr 2025
- First submitted
- 7 May 2025
- Last updated
- 11 May 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of Transfusion-dependent Nonsevere Aplastic Anemia With Luspatercept: a Multicenter Prospective Clinical Study
- ClinicalTrials.gov ID
- NCT06964971
- Acronym / sponsor ID
- 2025-KLS-046-02
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 90 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Zhejiang Chinese Medical UniversityUniversity, hospital or other
- Study start
- 20 May 2025 (estimated)
- Primary completion
- 20 May 2027 (estimated)
- Study completion
- 30 Jun 2027 (estimated)
- First posted
- 11 May 2025 (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.
The goal of this clinical trial is to learn whether Luspatercept alone or in combination with Deferasirox can promote hematopoietic function in patients with transfusion-dependent non-severe aplastic anemia, as well as to assess the safety and efficacy of this treatment approach.
The main questions it aims to answer is:
whether the combination therapy of Luspatercept and Deferasirox can improve hemoglobin levels in these patients.
Participants will receive Luspatercept every 3 to 5 weeks based on hemoglobin response, undergo complete blood counts every 1 to 3 weeks, and receive other necessary evaluations as required.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Control Group: Patients will continue with their original basic treatment regimen for the disease (immunosuppressants, androgens（either alone or in combination）) under observation. Treatment Group: In addition to the original basic treatment for the disease, patients will be further divided based on whether iron chelation therapy is used, into two subgroups: Luspatercept monotherapy group and Luspatercept combined with deferasirox group.
| Arm | Type | Receives |
|---|---|---|
| Treatment Group1 | Experimental | Drug: Luspaterceptbasic treatment regimen + Luspatercept alone |
| Treatment Group2 | Experimental | Drug: Luspatercept combine with Deferasiroxbasic treatment regimen + Luspatercept combine with Deferasirox |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 1.Age >= 18 years; 2.According to the "Chinese Guidelines for the Diagnosis and Treatment of Aplastic Anemia (2022 Edition)", the patient must be diagnosed with transfusion-dependent non-severe aplastic anemia (TD-NSAA) and meet the requirement of erythroid hyperplasia in bone marrow aspiration from the posterior iliac crest and/or sternum being more than 15%; 3.If not newly diagnosed with TD-NSAA, and there are combined primary disease maintenance medications, the following conditions must be met:
Key exclusion criteria (1 listed)
- 1. Severe hepatic dysfunction (ALT or AST ≥ 3 × ULN); 2.Severe renal impairment (eGFR < 30 ml/min/1.73m² or patients with end-stage renal disease); 3.Cardiac disease, including New York Heart Association (NYHA) Class 3 or higher heart failure, or severe arrhythmia requiring treatment, or recent myocardial infarction within 6 months of randomization; 4.Patients with uncontrolled hypertension, with controlled hypertension according to NCI CTCAE version 5.0 considered as ≤ Grade 1 for this protocol; 5.Patients with a PNH clone > 1%; 6.Patients planning to become pregnant or who are pregnant; 7.Surgical or clinical conditions that may significantly alter drug absorption, distribution, metabolism, or excretion (e.g., gastritis, ulcers, history of gastrointestinal or rectal bleeding; history of major gastrointestinal surgery); 8.Patients carrying congenital bone marrow failure-related gene mutations (homozygous or heterozygous, regardless of whether they are pathogenic/benign/likely benign/ of uncertain significance).
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 |
|---|---|---|
| LuspaterceptThe recommended starting dose of luspatercept is 1.5 mg/kg administered subcutaneously (SC) once every 3 weeks. Treatment response should be evaluated and dosage adjusted after at least 3 treatment cycles (9 weeks). | Drug | Treatment Group1 |
| Luspatercept combine with DeferasiroxThe recommended starting dose of luspatercept is 1.5 mg/kg administered subcutaneously (SC) once every 3 weeks. Treatment response should be evaluated and dosage adjusted after at least 3 treatment cycles (9 weeks). For patient with platelet counts < 50×10⁹/L: Deferasirox dosage: 8-10 mg/kg/day, orally. For platelet counts ≥ 50×10⁹/L: Deferasirox dosage: 20 mg/kg/day, orally. Discontinue deferasirox if serum ferritin drops below 500 μg/L. | Drug | Treatment Group2 |
Primary outcome
Hemoglobin
Time frame: Baseline (1 month before treatment), treatment phase: 12 weeks (twice weekly in weeks 1 / 2, once weekly in week 3, once every 3 weeks ), mid-treatment phase: 12 weeks (once every 3 weeks), follow-up period: 48 weeks (once every 12 weeks).
Mild anemia: Defined as a hemoglobin level <120 g/L in adult males or <110 g/L in adult females, but >90 g/L. Moderate anemia: Hemoglobin level between 60-90 g/L. Severe anemia: Hemoglobin level between 30-60 g/L. Extremely severe anemia: Hemoglobin level <30 g/L.
4 secondary outcomes
platelets
Time frame: Baseline (30 days before treatment), treatment phase: 12 weeks (twice weekly in weeks 1 / 2, once weekly in week 3, once every 3 weeks ), mid-treatment phase: 12 weeks (once every 3 weeks).
Ferritin
Time frame: Baseline (30 days before treatment), treatment phase: 12 weeks ( once every 3 weeks ), mid-treatment phase: 12 weeks (once every 3 weeks)
Proportion of patients transfusion-free for ≥8 weeks and ≥12 weeks
Time frame: treatment phase: 12 weeks (twice weekly in weeks 1 / 2, once weekly in week 3, once every 3 weeks ), mid-treatment phase: 12 weeks (once every 3 weeks)
Overall response rate (ORR; CR + PR) at Week 12 and Week 24 of treatment
Time frame: Week 12 and Week 24
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 May 2025.
- Individual participant dataSharing planned: No
Related trials for Transfusion-dependent Non-severe Aplastic Anemia
RecruitingPhase 1/2
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes
Bone Marrow Failure Syndromes, Aplastic Anemias
Not yet recruitingPhase 2
CsA+EPAG/HPAG+Romiplostim N01 in Newly-diagnosed SAA/TD-NSAA
Severe Aplastic Anemia (SAA), Transfusion-dependent Non-severe Aplastic Anemia
Not yet recruitingPhase 3
Enarodustat + CsA vs CsA in Newly Diagnosed TD-NSAA
Transfusion-dependent Non-severe Aplastic Anemia
RecruitingPhase 2
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
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.
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 and Luspatercept combine with Deferasirox in people with Transfusion-dependent Non-severe Aplastic Anemia, run by The First Affiliated Hospital of Zhejiang Chinese Medical 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 7 inclusion and 1 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: opening soon, as of the sponsor's last update on 11 May 2025. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Dijiong Wu, Ph.D. (+86 13989463963, wudijiong@zcmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Hemoglobin (time frame: Baseline (1 month before treatment), treatment phase: 12 weeks (twice weekly in weeks 1 / 2, once weekly in week 3, once every 3 weeks ), mid-treatment phase: 12 weeks (once every 3 weeks), follow-up period: 48 weeks (once every 12 weeks).). 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 11 May 2025.