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

Launched by PEKING UNION MEDICAL COLLEGE HOSPITAL · May 16, 2024

Keywords

ClinConnect Summary

This clinical trial is studying the effectiveness and safety of a new treatment called luspatercept when combined with a medication called cyclosporine (CsA) for patients who have recently been diagnosed with a type of blood disorder called aplastic anemia. Specifically, it focuses on patients who do not require blood transfusions and have certain blood count levels. The goal is to see if adding luspatercept can improve outcomes compared to using cyclosporine alone.

To be eligible for this trial, participants must be at least 18 years old, have specific blood counts that indicate they have aplastic anemia (but not the more severe forms), and must not have active infections or other serious health issues. Participants can expect to undergo regular check-ups and monitoring while receiving the treatment. The trial is not yet recruiting, so interested individuals will need to wait for it to begin. It's important to note that certain health conditions and previous treatments may exclude someone from participating, so talking to a healthcare provider is essential for anyone considering this option.

Gender

ALL

Eligibility criteria

• 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.
• 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