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

Last updated: May 16, 2024
Sponsor: Peking Union Medical College Hospital
Overall Status: Active - Not Recruiting

Phase

4

Condition

Aplastic Anemia

Anemia

Red Blood Cell Disorders

Treatment

Luspatercept

cyclosporine

Clinical Study ID

NCT06424639
LC-001
  • Ages 18-90
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

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 (atleast two of the following conditions can be diagnosed as AA: (i) Hemoglobin < 100g/L; (ii) Platelet count < 50×10^9/L; (iii) Neutrophil count < 1.5×10^9/L. SAAdiagnosis criteria include less than 25% (or 25-50%, but residual hematopoieticcells < 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, butwith 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.

Exclusion

Exclusion Criteria:

  1. Congenital aplastic anemia

  2. Presence of chromosomal aberrations

  3. Cytogenetic evidence of clonal hematological myelodysplastic syndrome (MDS) or acutemyelogenous leukemia (AML)

  4. PNH clone ≥50%

  5. Previous use of alemtuzumab, any ATG, or any dose of cyclosporine forimmunosuppressive 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 lessthan 480 milliseconds of bundle branch block determined by three ECG averages, andassessed on-site; unstable angina; uncontrolled hypertension (>180/100 mmHg);pulmonary hypertension

  12. Any malignant tumor within 5 years, except local basal cell carcinoma; previousthromboembolic event, history of myocardial infarction or stroke (includingantiphospholipid syndrome); currently using anticoagulants

  13. Pregnant or lactating women

  14. Participated in another clinical trial within 3 months

Study Design

Total Participants: 58
Treatment Group(s): 2
Primary Treatment: Luspatercept
Phase: 4
Study Start date:
May 01, 2024
Estimated Completion Date:
December 31, 2025

Study 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: received Luspatercept (1.0 mg/kg, subcutaneous injection every 3 weeks), cyclosporine (3-5mg/kg/day), adjusted based on hematological parameters, for at least 6 months to assess efficacy. Effective patients continued to receive cyclosporine treatment for at least 1.5 years, with a gradual reduction in dosage; Group B, cyclosporine: received 3-5mg/kg/day, adjusted based on hematological parameters, for at least 6 months to assess efficacy, with effective patients continuing to receive cyclosporine treatment for at least 1.5 years, with a gradual reduction in dosage. Hgb below 60g/L was allowed, or in emergency conditions, blood transfusion was allowed. Platelets below 20×10^9/L or with obvious bleeding tendency were allowed to receive platelet transfusion. If neutrophil count was below 1.0×10^9/L, G-CSF was allowed until neutrophil count recovered to above 1.0×10^9/L. Symptoms, treatment-related adverse events, signs, blood transfusion volume, and laboratory tests (including reticulocyte count) were recorded at least every 3 months for the first 3 months, and every 6 months thereafter until 6 months, and bone marrow aspiration, biopsy, and chromosome examination were performed at least every 6 months to observe efficacy and safety.

Connect with a study center

  • Peking Union Medical College Hospital

    Beijing, Beijing 100730
    China

    Site Not Available

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