This is a phase I/II clinical study in adult patients with refractory severe aplastic anemia (SAA). Eligible patients must meet the criteria for refractory SAA and have a platelet count (PLT) \<30 × 10\^9/L and/or hemoglobin (HGB) \<90 g/L at enrollment. If the phase I results demonstrate an acceptable safety profile and allow determination of the maximum tolerated dose (MTD), the phase II part will be initiated directly to evaluate the efficacy of isatuximab.
Diagnosed with primary acquired aplastic anemia according to the 2024 British Society for Haematology guideline, Guidelines for the Diagnosis and Management of Adult Aplastic Anaemia, and the Chinese Guideline for the Diagnosis and Treatment of Aplastic Anemia (2022 edition) issued by the Hematology Branch of the Chinese Medical Association.
Previously diagnosed with severe aplastic anemia (SAA) or very severe aplastic anemia (VSAA), with no response or relapse after receiving anti-thymocyte/anti-lymphocyte globulin (ATG/ALG) in combination with standard-dose cyclosporine for at least 6 months, and standard-dose thrombopoietin receptor agonist (TPO-RA) therapy for at least 4 months.
Hemoglobin <90 g/L or platelet count <30×10^9/L
Unsuitable for or unwilling to undergo hematopoietic stem cell transplantation, with no better available treatment options.
Diagnosed with congenital bone marrow failure syndromes
Bone marrow reticulin fibrosis grade ≥2
Subjects with a paroxysmal nocturnal hemoglobinuria (PNH) clone ≥50% or active hemolysis
Subjects with clonal cytogenetic abnormalities characteristic of myelodysplastic syndromes, except +8, del(20q), and -Y
Single group
Isatuximab
DrugPhase I: Eligible subjects will receive isatuximab at 5 mg/kg per dose or 10 mg/kg per dose. The treatment period will last 6 weeks. Isatuximab will be administered by intravenous infusion once weekly (QW) for six consecutive doses. Phase II: Eligible subjects will receive isatuximab by intravenous infusion once weekly for six consecutive doses. The specific dose will be the recommended phase II dose from the phase I part.
Incidence of adverse events
Use Common Terminology Criteria for Adverse Events (CTCAE) Version 6 to assess the adverse event
Overall response rate
Percentage of patients with hematological response, including complete response (CR) or partial response (PR). Hematological response is evaluated by hemoglobin (Hb), platelet count (PLT) and absolute neutrophil count (ANC).
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Institute of Hematology & Blood Diseases Hospital, China
Lead sponsor
Sanofi
Collaborator
