Luspatercept Plus CsA vs CsA for the Treatment of Newly Diagnosed Non-Transfusion-Dependent NSAA

Phase 4

Not yet recruiting

• Conditions: Aplastic Anemia
• Interventions: Drug: Luspatercept; Drug: cyclosporine

• Registration Number: NCT06424639
• Lead Sponsor: Peking Union Medical College Hospital

Brief Summary

In a randomized, controlled clinical trial, the efficacy and safety of rodsipil combined with cyclosporine versus cyclosporine alone in the treatment of newly diagnosed non-transfusion-dependent NSAA were compared.

Detailed Description

Conduct a comparative evaluation of the effectiveness and safety of Luspatercept combined with cyclosporine versus cyclosporine monotherapy in the treatment of newly diagnosed non-transfusion-dependent non-severe aplastic anemia (NSAA). Patients were randomized in a 1:1 ratio and assigned to one of two groups: Group A, Luspatercept combined with cyclosporine...

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Status Verified: 2024-05
• Study Start: 2024-05
• Study First Submitted: 2024-05-16
• Study First Submitted QC: 2024-05-16
• Last Update Submitted: 2024-05-16
• Study First Posted: 2024-05-22
• Last Update Posted: 2024-05-22
• Primary Completion: 2025-05
• Study Completion: 2025-12

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 58

Inclusion Criteria

1. Age ≥ 18 years
2. Hemoglobin level of 6-10 g/dL
3. Definition of NSAA: Patients with AA diagnosis but not SAA or VSAA diagnosis (at least two of the following conditions can be diagnosed as AA: (i) Hemoglobin < 100 g/L; (ii) Platelet count < 50×10^9/L; (iii) Neutrophil count < 1.5×10^9/L. SAA diagnosis criteria include less than 25% (or 25-50%, but residual hematopoietic cells < 30%) of bone marrow cells, plus at least two of the following conditions: (i) Neutrophil count < 0.5×10^9/L; (ii) Platelet count < 20×10^9/L; (iii) Retroperitoneal lymph node count < 20×10^9/L. VSAA meets the criteria for SAA, but with neutrophil count < 0.2×10^9/L. (British guidelines, 2015))
4. No active infection
5. No other concurrent neoplasms (except in situ carcinoma)
6. Baseline liver and renal function within 1.5 times of normal value
7. No pregnancy or breastfeeding
8. Agree to sign informed consent form
9. Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.

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Exclusion Criteria

1. Congenital aplastic anemia
2. Presence of chromosomal aberrations
3. Cytogenetic evidence of clonal hematological myelodysplastic syndrome (MDS) or acute myelogenous leukemia (AML)
4. PNH clone ≥50%
5. Previous use of alemtuzumab, any ATG, or any dose of cyclosporine for immunosuppressive treatment
6. Previous hematopoietic stem cell transplant (HSCT)
7. Uncontrolled infection or bleeding under standard treatment
8. Allergy to rituximab, cyclosporine, or excipients
9. History of allergy to polyethylene glycol (PEG) 80
10. Active infection or cirrhosis of the liver or portal hypertension due to HIV, HCV, or HBV
11. Screening QTcF (Fridericia QT corrected formula) less than 450 milliseconds or less than 480 milliseconds of bundle branch block determined by three ECG averages, and assessed on-site; unstable angina; uncontrolled hypertension (>180/100 mmHg); pulmonary hypertension
12. Any malignant tumor within 5 years, except local basal cell carcinoma; previous thromboembolic event, history of myocardial infarction or stroke (including antiphospholipid syndrome); currently using anticoagulants
13. Pregnant or lactating women
14. Participated in another clinical trial within 3 months

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Luspatercept combined with cyclosporine	Luspatercept	Administered Luspatercept (1.0 mg/kg, subcutaneous injection every 3 weeks), and Cyclosporine (3-5mg/kg/day) adjusted according to hematological parameters, for at least 6 months to evaluate efficacy. Effective patients will continue to receive Cyclosporine treatment for at least 1.5 years, followed by a gradual reduction in dosage.
Luspatercept combined with cyclosporine	cyclosporine	Administered Luspatercept (1.0 mg/kg, subcutaneous injection every 3 weeks), and Cyclosporine (3-5mg/kg/day) adjusted according to hematological parameters, for at least 6 months to evaluate efficacy. Effective patients will continue to receive Cyclosporine treatment for at least 1.5 years, followed by a gradual reduction in dosage.
cyclosporine	cyclosporine	Give cyclosporine 3-5mg/kg/day, adjust the dose based on the blood count, and administer it for at least 6 months to evaluate the efficacy. If effective, the patient will continue to receive cyclosporine treatment for at least 1.5 years, followed by a gradual reduction in dosage.

Primary Outcome Measures

Name	Time	Method
overall response rate (ORR)	6 month	Proportion of patients who achieved complete response, partial response and hematological response

Secondary Outcome Measures

Name	Time	Method
overall response rate (ORR)	12 month	Proportion of patients who achieved complete response, partial response and hematological response
adverse event rate	12 month	Proportion of patients with adverse events

Trial Locations

Locations (1)

Peking Union Medical College Hospital; 🇨🇳 Beijing, Beijing, China