EUCTR2015-002603-29-SE进行中（未招募）1 期
An Open-Label Study To Evaluate The Efficacy And Safety Of Revusiran In Patients With Transthyretin-Mediated Familial Amyloidotic Polyneuropathy With Disease Progression Post Orthotopic Liver Transplant
Alnylam Pharmaceuticals, Inc0 个研究点目标入组 12 人开始时间: 2015年7月10日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 12
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
- •2. Diagnosis of FAP with documented TTR mutation
- •3. Received an OLT =12 months before the date of informed consent
- •4. An increase in polyneuropathy disability (PND) score compared to a preliver transplant assessment OR increase in PND score between any 2 assessments postliver transplant, which in the opinion of the Investigator is due to underlying FAP progression
- •5. On stable immunosuppressive regimen with =10 mg/day of prednisone for at least 3 months before the date of informed consent
- •6. Neurological impairment score (NIS) of 5 to 130 (inclusive)
- •7. Polyneuropathy Disability score of =3b
- •8. Karnofsky Performance Status =60%
- •9. No liver allograft rejection episodes (chronic, acute, or subacute) in the past 6 months before the date of informed consent
- •10. Normal liver function, including aspartate transaminase, alanine transaminase, and total bilirubin, unless elevation in total bilirubin is due to Gilbert’s syndrome based on central laboratory evaluation
- •11. Adequate renal function demonstrated by estimated glomerular filtration rate =45 mL/min/1.73 m2 (calculated by a central laboratory using the Modification of Diet in Renal Disease formula)
- •12. Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must be willing to use 1 highly effective method of contraception throughout study participation and for 28 days after last dose administration
- •13. Males who agree to use appropriate means of contraception throughout study participation until 28 days after last dose administration
- •14. Patient, or patient’s legal representative, is able and willing to provide written informed consent and the patient is willing to comply with the study requirements
- •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 12
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Untreated hypo- or hyperthyroidism
- •2. Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed before the first dose of study drug administration
- •3. Active infection with hepatitis B or hepatitis C (based on serology)
- •4. Known human immunodeficiency virus infection
- •5. New York Heart Association (NYHA) classification of >2
- •6. Known leptomeningeal amyloidosis
- •7. Other known causes of sensorimotor or autonomic neuropathy (eg, autoimmune disease)
- •8. Known type I diabetes
- •9. Type II diabetes mellitus for =5 years from the time of informed consent
- •10. Vitamin B12 levels below the lower limit of normal
- •11. Known history of alcohol abuse within the last 2 years from the time of informed consent
- •12. Received an investigational agent or device within 30 days of anticipated study drug administration or 5 half-lives of the study drug, whichever is longer
- •13. Currently taking diflunisal, tafamidis, doxycycline, or tauroursodeoxycholic acid; if previously on any of these agents, must have completed a 14-day washout before start of study drug administration in this study
- •14. Malignancy within the last 2 years from the time of informed consent, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated
- •15. History of allergic reaction to an oligonucleotide or N-acetylgalactosamine (GalNAc)
- •16. History of intolerance to subcutaneous (SC) injection
- •17. Other medical conditions or comorbidities which, in the opinion of the Investigator, would interfere with study compliance or data interpretation
研究者
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