EUCTR2020-005854-90-AT进行中（未招募）1 期
A Phase 2, Single-Center, Open-Label, Repeat-Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Clinical Effect of Intravenous ANX005 in Participants with Primary Cold Agglutinin Disease
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 6
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Male or female = 18 years of age on the day of signing informed consent
- •2. Body weight < 150 kg
- •3. Diagnosis of CAD at least 3 months prior to screening based upon all of the following:
- •a. Positive direct antiglobulin test (DAT) = 1+ for C3d
- •b. Negative or only weakly positive (1+ or less) direct antiglobulin test for IgG
- •c. Cold agglutinin (IgM) titer =1:64 at 4°C or lower titer with signs and symptoms consistent with cold agglutinin disease and demonstration of positive IgM autoantibodies
- •d. Evidence of active hemolysis defined as presence of any one of the following:
- •i. Lactate dehydrogenase (LDH) > upper limit of normal (ULN)
- •ii. Indirect bilirubin > ULN
- •iii. Haptoglobin < lower limit of normal (LLN)
- •4. Evidence of active hemolysis (defined above) at screening
- •5. Hgb level = 10.0 g /dL at screening with no alternative explanation for anemia apart from CAD
- •6. Vaccinations against encapsulated bacterial organisms (N. meningitidis, H. influenzae and S. pneumoniae) within 5 years prior to screening or participant 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
- •7. Females must be of non-childbearing potential
- •8. Females with a history of a bilateral tubal occlusion/ligation procedure who do not meet any of the other criteria for non-childbearing potential (postmenopausal or permanently sterile), must have a negative serum pregnancy test at screening and a negative urine pregnancy test at Day 1
- •9. Males must agree to use adequate methods of contraception throughout the study and for at least 1 month after the final study visit (at least 2 months after the last administration of study medication)
- •10. Able to comply with the requirements of the study and complete the full sequence of protocol-related doses, procedures, and evaluations
- •11. Able to understand and provide written informed consent
- •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 3
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 3
排除标准
- •Exclusion Criteria:
- •1. History of cold agglutinin disease secondary to infection, rheumatologic disease, or active malignancy
- •2. History of hemolytic anemia due to another condition besides CAD including, but not limited to, paroxysmal nocturnal hemoglobinuria (PNH), Evan’s syndrome, sickle cell disease, thalassemia, thrombotic thrombocytopenia purpura, hemolytic-uremic syndrome (HUS), red cell membrane disorders (e.g. hereditary spherocytosis, hereditary elliptocytosis, hereditary pyropoikliocytosis, hereditary stomatocytosis, hereditary exocytosis), pyruvate kinase deficiency, glucose-6-phosphate deficiency, or hemolytic anemia secondary to autoimmunity (e.g. systemic lupus erythematosus, rheumatoid arthritis), infectious cause (e.g. Epstein-Barr Virus, cytomegalovirus, mycoplasma, hepatitis virus, human immunodeficiency virus), malignancy (e.g. lymphoproliferative disorder requiring treatment, lymphoma, leukemia, multiple myeloma), or medications (e.g. anti-neoplastics, non-steroidal anti-inflammatory drugs, antibiotics). Note: Mixed autoimmune hemolytic anemia may potentially be enrolled, but should first be discussed with the medical monitor
- •3. History of solid organ, bone marrow, or stem cell transplantation
- •4. History of splenectomy within the 3 months prior to screening
- •5. Clinically significant ongoing illness or medical condition affecting any major organ system, including chronic infections, or a medical history that would jeopardize the safety of the participant, limit participation in the study, or compromise the interpretation of the data derived from the participant
- •6. History of active lymphoma, lymphoproliferative disorder requiring therapy, or any other active malignant disease that has not been adequately treated
- •7. Signs and symptoms of, or a diagnosis consistent with, a chronic autoimmune disorder not resulting from primary CAD
- •8. History of meningitis or septicemia within the past 2 years
- •9. Clinically significant infection that required systemic medical intervention (not including antibiotic prophylaxis; i.e., viral, bacterial, fungal, or mycobacterial) within 1 month prior to screening
- •10. Positive serology for human immunodeficiency virus (HIV) 1 or 2 at screening
- •11. Evidence of hepatitis C virus (HCV) antibody at screening require reflex testing for HCV RNA. Participant with positive HCV RNA viral load at screening will be excluded. Participants with positive HCV Ab, but negative HCV RNA viral load are eligible but should be monitored throughout the study
- •12. Evidence of active or prior hepatitis B virus (HBV) infection. Participants with positive hepatitis B surface antigen (HBsAg) at screening are excluded from the study. Participants with positive HBV core Ab and HBV DNA viral load at screening are excluded from the study
- •13. Known genetic deficiencies of the complement cascade system
- •14. Active alcohol or substance abuse or any other reason that makes it unlikely that the participant will comply with study procedures
- •15. Females who are pregnant (positive serum pregnancy test at screening or positive urine pregnancy test on Day 1) or lactating
- •16. Hypersensitivity to any of the excipients in the ANX005 drug product
- •17. Hypersensitivity to or previous allergic reaction to the active substance or to any of the excipients in any of the immunizations required for this study
- •18. History of previous sensitivities or allergic or anaphylactic reactions to previous IV medication
- •19. Platelet count <30 × 109/L
研究者
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