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 - Study with intravenous ANX005 in patients with wAIHA

Annexon, Inc0 个研究点目标入组 12 人开始时间: 2021年8月2日最近更新: 2021年8月30日

适应症

试验速览

暂无简介。

•1. Male or non-pregnant, non-lactating female = 18 years of age on the day of signing informed consent
•2. Diagnosis of wAIHA at least 3 months prior to screening
•3. Hgb level > 10.0 g/dL (pre-transfusion) with no alternative explanation for anemia apart from wAIHA at screening
•4. Positive direct antiglobulin test (DAT) = 1 + for C3d and IgG
•5. Evidence of classical complement pathway activation by one of the
•a. Serum complement component 4 (C4) below the lower limit of normal
•b. CH50 below the lower limit of normal
•6. Evidence of active hemolysis based on at least one of the following:
•a. LDH above the ULN
•b. Indirect bilirubin above the ULN
•c. Haptoglobin below the LLN
•7. Vaccinations against encapsulated bacterial organisms (N. meningitidis, H. influenzae and S. pneumoniae) within 5 years prior to screening or subject must be willing
•to complete vaccinations at least 2 weeks prior to dosing with ANX005.
•Documentation of vaccinations must be received at the study site in advance of the scheduled screening visit
•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

•1. History of or current lymphoma or lymphoproliferative disorder requiring therapy
•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. Other wAIHA treatments:
•- Received rituximab or other anti-CD20 monoclonal antibody < 3 months prior to screening
•- Receiving steroids > 1 mg/kg of prednisone or equivalent daily at screening
•8. Signs and symptoms of, or a diagnosis consistent with, a chronic autoimmune disorder not resulting from primary wAIHA and/or an ANA titer of = 1:160
•9. Known genetic deficiencies of the complement cascade system

发起方