EUCTR2006-002792-41-DE进行中（未招募）不适用
Safety and Efficacy of Orally Administered Fx-1006A in Patients with Familial Amyloid Polyneuropathy (FAP): A Phase II/III, Randomised, Double-Blind, Placebo-Controlled Study - Not applicable
FoldRx Pharmaceuticals Limited0 个研究点目标入组 120 人开始时间: 2007年6月20日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 120
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
- •Inclusion Criteria for All Patients:
- •-Patient is aged =18 through 75 years.
- •-If female, patient is post-menopausal, surgically sterilized, or willing to use two acceptable methods of birth control (i.e., a hormonal contraceptive, intra-uterine device, diaphragm with spermicide, condom with spermicide) throughout the study and for 3 months from the end of the study. (A condom alone is not considered an acceptable method of birth control.) If male with a female partner of childbearing potential, willing to use two acceptable methods of birth control for the duration of the study. For both females and males, acceptable birth control must be used for at least 3 months after the last dose of study medication.
- •-Patient is, in the opinion of the investigator, willing and able to comply with the study medication regimen and all other study requirements.
- •Inclusion Criteria for Patients with Positive Biopsy
- •-Patient has amyloid documented by biopsy (in accordance with institutional site standard of care)
- •-Patient has documented V30M TTR mutation (procedure detailed in a Laboratory Manual).
- •-Patient has peripheral and/or autonomic neuropathy with a Karnofsky Performance Status =50.
- •Inclusion Criteria for Patients with Negative or No Biopsy
- •-Patient has documented V30M TTR mutation (procedure detailed in a Laboratory Manual).
- •-Patient has a clear family history with first degree relative with a diagnosis of FAP with progressive, sensorimotor polyneuropathy and V30M TTR mutation.
- •-Patient has peripheral and/or autonomic neuropathy with a Karnofsky Performance Status = 50
- •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
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Exclusion Criteria for All Patients
- •-Chronic use of non-protocol approved non-steroidal anti-inflammatory drugs (NSAIDs), defined as greater than 3-4 times/month. The following NSAID are allowed: acetylsalicylic acid, etodolac, ibuprofen, indomethicin, ketoprofen, nabumetone, naproxen, nimesulide, piroxicam, and sulindac.
- •-Patient has primary amyloidosis.
- •-If female, patient is pregnant or breast feeding.
- •-Patient has received prior liver transplantation.
- •-Patient has no recordable sensory threshold for vibration perception in both feet, as measured by CASE IV.
- •-Patients with positive results for hepatitis B surface antigen (HBsAg), anti-hepatitis C virus (HCV), and/or human immunodeficiency virus (HIV).
- •-Patient has renal insufficiency (creatinine clearance < 30 ml/min).
- •-Patient has liver function test abnormalities: alanine transaminases (ALT) and/or aspartate transaminases (AST) >2 times upper limit of normal (ULN) that in the medical judgment of the investigator are due to reduced liver function or active liver disease.
- •-Patient has a New York Heart Association (NYHA) Functional Classification =III
- •-Patient has a co-morbidity anticipated to limit survival to less than 18 months.
- •-Patient received an investigational drug/device and/or participated in another clinical investigational study within 60 days before Baseline.
- •Exclusion Criterion for Patients with Positive Biopsy
- •-Patient has other causes of sensorimotor neuropathy (B12 deficiency, Diabetes Mellitus, HIV treated with retroviral medications, thyroid disorders, alcohol abuse, and chronic inflammatory diseases).
- •Exclusion Criterion for Patients with Negative or No Biopsy
- •-Patient has other cause(s) of sensorimotor neuropathy, such as:
- •Vitamin B12 deficiency confirmed at Screening
- •Diabetes Mellitus confirmed by elevated HbA1c at Screening
- •Chemotherapeutic or other associated chronic drug therapy
- •Heavy metal or environmental toxin exposure
- •HIV confirmed by positive HIV antibody at Screening
- •Thyroid disorders, confirmed by abnormal thyroid function at Screening
- •Alcohol abuse confirmed by medical history
研究者
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