Efficacy and Safety in Transfusion Independent Non-severe Aplastic Anemia

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

58 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-12-19

Study Completion Date

2025-04-15

Brief Summary

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Aplastic anemia (AA) is a rare bone marrow failure disease characterized by bone marrow hypocellularity and peripheral blood pancytopenia. AA is divided into severe AA (SAA) and non-severe AA (NSAA) based on the degree of cytopenia. The first line therapy for SAA or transfusion dependent NSAA is either immunosuppression therapy (IST) or hematopoietic stem cell transplantation (HSCT). Little attention has been paid to patients with anemia but not transfusion dependent, whose quality of life is significantly impaired due to the anemia and other complications.

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Detailed Description

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Recombined human erythropoietin (rhEPO) has been shown to increase the erythroid response and response rate when combined with IST for patients with newly diagnosed AA, either SAA or NSAA. Different from rhEPO, luspatercept is a recombinant fusion protein that binds to select transforming growth factor β superfamily ligands and enhances late-stage erythropoiesis, and has been shown the promising efficiency in the erythropoiesis in patients with lower risk myelodysplastic syndrome (MDS) in the phase II and III clinical trials. This randomized control study aimed to compare the 6-month efficacy and safety of the combination of luspatercept and cyclosporine versus cyclosporine monotherapy in patients with newly diagnosed transfusion independent NSAA.

Conditions

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Aplastic Anemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Cyclosporine soft capsules（25mg） are produced by North China pharmaceutical Group Corporation

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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efficiency and safety in luspatercept plus cyclosporine

luspatercept is at a dose of 1.0 mg per kilogram of body weight, administered subcutaneously every 3 weeks，and cyclosporine is at a dose of 3\~5mg/kg /day for at least 6 months.

Group Type EXPERIMENTAL

Luspatercept

Intervention Type DRUG

Patients in each group will be treated for at least 6 months and continue the treatment for an additional 6 months unless disease progress or have intolerable side effects.

Cyclosporine

Intervention Type DRUG

Cyclosporine was administered at a 3-5 mg/(kd/d) and maintained at a 100-200 ng/ml trough plasma concentration.

controll group in cyclosporine alone

cyclosporine is at a dose of 3\~5mg/kg /day for at least 6 months.

Group Type ACTIVE_COMPARATOR

Cyclosporine

Intervention Type DRUG

Cyclosporine was administered at a 3-5 mg/(kd/d) and maintained at a 100-200 ng/ml trough plasma concentration.

Interventions

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Luspatercept

Patients in each group will be treated for at least 6 months and continue the treatment for an additional 6 months unless disease progress or have intolerable side effects.

Intervention Type DRUG

Cyclosporine

Cyclosporine was administered at a 3-5 mg/(kd/d) and maintained at a 100-200 ng/ml trough plasma concentration.

Intervention Type DRUG

Other Intervention Names

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Ciclosporin Cyclosporin

Eligibility Criteria

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Inclusion Criteria

1. age≥18 year-old;
2. hemoglobin level between 60g/L\~10 g/dL;
3. newly diagnosed patients have at least one of the followings: #absolute neutrophil count \<1.5×109/L, #platelet count \< 30×109/L, # hemoglobin level \< 100g/L;
4. with normal baseline liver and kidney function;
5. with no active infection; are not pregnant or nursing;
6. agree to sign consent forms;
7. Eastern Cooperative Oncology Group (ECOG) performance status 0-2

Exclusion Criteria

1. Congenital aplastic anemia;
2. Presence of chromosomal aberration;
3. Evidence of a clonal hematologic bone marrow disorder (MDS, AML) on cytogenetics;
4. Presence with PNH clone ≥50%;
5. Patients received HSCT before;
6. Uncontrolled infection or bleeding with standard treatment;
7. Allergic to luspatercept CsA or accessories;
8. HIV, HCV or HBV active infection or liver cirrhosis or portal hypertension;
9. Patient with QTcF (Fridericia's QT correction formula) at screening \<450 msec, or\<480 msec with bundle branch block, as determined via the mean of a triplicate ECG and assessed at site, unstable angina pectoris, uncontrolled hypertension(\>180/100mmHg)#pulmonary artery hypertension;
10. Have any concomitant malignancies within 5 years expect for local basal cell carcinoma of the skin;
11. Past history of thromboembolic event, heart attack or stroke (including anti-phospholipid antibody syndrome) and current use of anticoagulants;
12. Pregnant or nursing (lactating) woman;
13. Have attended other clinical trials within 3 months

Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No