A Phase 2, Open-label, Single Dose, Study of Subcutaneously Administered ALN-CC5 in Patients with Paroxysmal Nocturnal Hemoglobinuria who are Inadequate Responders to Eculizumab

Alnylam Pharmaceuticals Inc0 个研究点目标入组 15 人开始时间: 2016年9月30日最近更新: 2017年1月9日

试验速览

阶段: 1 期
状态: 进行中（未招募）
发起方
入组人数: 15

研究概览入排标准研究者记录与标识符相似试验

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. Age 18 years or older
•2. History of PNH
•3. Treatment with eculizumab at a stable dose for a minimum of 60 days before Screening
•4. Inadequate response to eculizumab as defined by elevated LDH level before eculizumab infusion at Screening as follows:
•LDH=3.0×ULN (with or without symptoms) attributable to hemolysis in the opinion of the Investigator; or,
•LDH =2.0×ULN, 1 or more of the following at Screening and while receiving eculizumab treatment, and which in the opinion of the Investigator is/are attributable to PNH disease: PNH symptoms (eg, fatigue, abdominal pain, dyspnea, dysphagia, or erectile dysfunction); Hemoglobin level =10 g/dL; Any blood transfusions within 90 days before Screening; and/or Spontaneous vascular thrombosis at any time since starting eculizumab treatment.
•5. Women of child-bearing potential (WOCBP) must have a negative pregnancy test, cannot be breast feeding, and must be willing to use a highly effective method of contraception 14 days before ALN-CC5 administration, throughout study participation, and for 6 months after ALN-CC5 administration.
•6. Willing and able to comply with the study requirements and to provide written informed consent
•7. Vaccinated against Neisseria meningitides according to standard practice (patients may be rescreened if not previously vaccinated)
•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 14
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 1

排除标准

•1. Alanine transaminase =2×ULN or total bilirubin =4×ULN (unless bilirubin elevation is due to Gilbert’s syndrome), which is considered clinically relevant in the opinion of the Investigator
•2. Clinically significant bone marrow suppression as defined by 2 or more of the following:
•Absolute neutrophil count =1.0×10 to power of 9/L
•Platelet count =50×10 to power of 9/L
•Absolute reticulocyte count =60×10 to power of 9/L
•3. Clinical laboratory test results considered clinically relevant and unacceptable in the opinion of the Investigator
•4. Planned change in eculizumab dose within 60 days of administration of ALN-CC5 (patients may be re-Screened)
•5. Spontaneous vascular thrombosis within 30 days of randomization (patients may be re Screened)
•6. Known or suspected hereditary asymptomatic complement deficiency
•7. Known clinical laboratory evidence or clinical diagnosis of human immunodeficiency virus infection (HIV), hepatitis C virus (HCV) infection, or chronic hepatitis B virus infection as shown by hepatitis B surface antigen positivity in blood
•8. Suspicion of active viral, bacterial, fungal, or parasitic infection as a cause for breakthrough hemolysis and their presence within 14 days before ALN-CC5 administration (patients may be rescreened)
•9. Travelled to Saudi Arabia or Africa within 90 days of Screening, or planning to do so during the study (patients may be rescreened)
•10. Received an investigational agent within the last 90 days or are in follow-up of another clinical study before study enrollment
•11. Active serious mental illness or psychiatric disorder, including, but not limited to, schizophrenia, bipolar disorder, or severe depression requiring current pharmacological intervention
•12. Other medical conditions or comorbidities which, in the opinion of the Investigator, would interfere with study compliance or data interpretation
•13. History of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc
•14. History of intolerance to SC injection(s) at the proposed injection site(s) that could potentially hinder study drug administration or evaluation of local tolerability
•15. History of meningococcal infection within 12 months before Screening
•16. Known history of chronic liver disease or cirrhosis

研究者

发起方

Alnylam Pharmaceuticals Inc

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