CTRI/2023/10/058601尚未招募2 期
A Phase II, Open-Label Study of NM8074 in Patients with Atypical Hemolytic Uremic Syndrome (aHUS) - NI
ovelMed Therapeutics0 个研究点目标入组 0 人开始时间: 待定最近更新:
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
入选标准
- •1. Patients, 14 Years and older, with evidence of complement-mediated aHUS with symptoms of thrombocytopenia, hemolysis, reduced eGFR, elevated serum creatinine, and kidney injury
- •2. Male or female = 14 years of age and weighing = 45 kg at the time of consent
- •3. Platelets less than 150,000 per microliter (thrombocytopenia) and Microangiopathic hemolysis leading to thrombocytopenia, hemolysis, and decreased kidney function
- •4. Prescence of Schistocytes
- •5. Decreased level of haptoglobin and hemoglobin (Hemoglobin =10 g/dL)
- •6. Lactate dehydrogenase (LDH) level = 1.5 times the upper limit of normal (xULN) during Screening.
- •7. Negative for Shiga toxin induced HUS (STEC-HUS)
- •8. ADAMTS13 activity greater than 10%
- •9. Negative (-) direct Coombs test
- •10. Detectable complement gene mutation (eg. Complement Factor H or Complement Factor I)
- •11. All patients must be vaccinated prior to dosing with MenACWY Menactra® polysaccharide diphtheria toxoid conjugate vaccination against Neisseria meningitidis serogroups A, C, Y, and W-135 and MenB meningococcal serogroup B vaccine (Bexsero®). If the window of vaccination is short, then patients will be prophylactically treated with appropriate antibiotics.
- •12. Willing and able to understand and complete informed consent procedures, including signing and dating the informed consent form (ICF), and complying with the study visit schedule.
- •13. Female partners of child-bearing potential (WOCBP), defined as all women physiologically capable of becoming pregnant, must have a negative pregnancy test at screening and must agree to use highly effective methods of contraception during dosing and for 1 month after stopping the investigational drug.
- •14. Male patients and partners of child-bearing potential must agree to use contraceptives and male patients must agree to refrain from donating sperm for the duration of the study.
排除标准
- •1. History of bone marrow, hematopoietic stem cell, or solid organ transplantation
- •2. History or current treatment with complement blockers
- •3. Patients with infections
- •4. HUS due to 13 (ADAMTS-13) deficiency ( <10%)
- •5. Kidney disease other than aHUS
- •6. Chronic dialysis (hemo or peritoneal)
- •7. Liver disease or other major autoimmune diseases
- •8. Positive direct Coombs test
- •9. Shiga toxin related hemolytic uremic syndrome
- •10. Known Systemic Lupus Erythematosus (SLE), Systemic Sclerosis, or antiphospholipid antibody positivity or syndrome
- •11. History of currently active primary or secondary immunodeficiency
- •12. Identified drug exposure-related HUS or HUS related to known genetic defects of cobalamin C metabolism or known diacylglycerol kinase e (DGKE) mediated aHUS
- •13. Currently active systemic infection or suspicion of active bacterial, viral, or fungal infection within 2 weeks prior to first dose, or history of unexplained, recurrent bacterial infections
- •14. Has a currently active or known history of meningococcal disease or N. meningitidis infection
- •15. Severe concurrent co-morbidities not amenable to active treatment, e.g., patients with severe kidney disease (CKD stage 4, chronic dialysis)
- •16. Pregnant, planning to become pregnant, or nursing female subjects.
- •17. Females who have a positive pregnancy test result at Screening or on Day 1.
研究者
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