A Phase 2, Open-Label, Repeat Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Proof-of-Concept of Intravenous ANX005 in Subjects with Warm Autoimmune Hemolytic Anemia

Annexon, Inc.0 个研究点目标入组 12 人开始时间: 2020年12月10日最近更新: 2021年10月26日

研究概览

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•Subjects must meet ALL the following criteria to be eligible to participate in the study:
•1. Male or female = 18 years of age on the day of signing informed consent
•2. Diagnosis of wAIHA at least 3 months prior to screening
•3. hemoglobin level = 10.0 g/dL (pre-transfusion) at screening with no alternative explanation for anemia apart from wAIHA
•4. Historical positive DAT = 1 + for C3d and IgG
•5. Evidence of classical complement pathway activation by at least one of the following within 6 months prior to dosing:
•a. Serum complement component 4 (C4) below the lower limit of normal
•b. CH50 below the lower limit of normal
•c. Complement component deposition on the cell surface assessed by flow cytometry
•6. Evidence of active hemolysis based on at least one of the following:
•a. LDH above the upper limit of normal (ULN)
•b. Indirect bilirubin above the ULN
•c. Haptoglobin below the LLN
•7. Patients must have been previously vaccinated for encapsulated
•bacteria within 5 years prior to screening or be willing to receive
•prophylaxis against infections with encapsulated bacteria via vaccination
•and/or the use of prophylactic antibiotics in accordance with local
•standards of practice and/or guidelines.
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 10
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 2

•Subjects must not meet any of the following criteria:
•1. Active lymphoma, lymphoproliferative disorder, or other malignancy
•requiring therapy and/or not clinically stable or in remission for at least
•3 months prior to screening
•2. Elevated aspartate aminotransferase or alanine aminotransferase levels > 2.5 times the upper
•limit of normal at screening
•3. Platelet count < 30 x 109/L
•4. History of cold agglutinin disease or IgM cold agglutinin titer > 1:64
•5. History of solid organ, bone marrow, or stem cell transplantation
•6. History of splenectomy within the 3 months prior to screening
•7. Received rituximab or other B cell depleting monoclonal antibody (eg.
•daratumumab) <90 days prior to screening
•8. History of a systemic autoimmune disorder (eg. lupus) or primary
•immunodeficiency disorder (eg. common variable immune deficiency)
•9. Known genetic deficiencies of the complement cascade system

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