EUCTR2012-001831-30-IT进行中（未招募）1 期
A Phase 2/3 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy - ISIS 420915-CS2
Isis Pharmaceuticals, Inc.0 个研究点目标入组 195 人开始时间: 2013年6月19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
- 195
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.Stage 1 and Stage 2 FAP patients with the following:
- •a.NIS score >15 and <85
- •b.Ability to walk unaided or with the use of no more than one stick/cane
- •c.Documented transthyretin variant by genotyping
- •d.Documented amyloid deposit by biopsy
- •2.Willingness to take vitamin A supplements
- •3.Aged 18 to 75 years old at the time of informed consent
- •4.Satisfy the following:
- •a.Females: Non-pregnant and non-lactating; surgically sterile, post-menopausal, abstinent, or if engaged in sexual relations of child-bearing potential, patient is using an acceptable contraceptive method for 4 weeks prior to, during, and for 3 months after the last dose of Study Drug
- •b.Males: Surgically sterile, abstinent, or if engaged in sexual relations of child-bearing potential, patient is utilizing an acceptable contraceptive method during and for 3 months after the last dose of Study Drug
- •5.Must have given written informed consent (signed and dated) and any authorizations required by local law and be able to comply with all 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 165
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 30
排除标准
- •1. Unwillingness to comply with study procedures, including follow-up, as specified by this protocol, or unwillingness to cooperate fully with the Investigator
- •2. Screening laboratory results as follows, or any other clinically significant abnormalities in screening laboratory values that would render a patient unsuitable for inclusion:
- •a.ALT/AST >1.9 x ULN
- •b.Bilirubin =1.5 x ULN (patients with bilirubin =1.5 x ULN may be allowed on study following discussion with the Study Medical Monitor if indirect bilirubin only is elevated,ALT/AST is not greater than the ULN and genetic testing confirming Gilbert’s disease)
- •c.Platelets <100 x 109/L
- •d.Persistently positive (2 out of 3 consecutive tests =1+) for protein on urine dipstick. In the event of a positive test eligibility may be confirmed by a quantitative total urine protein measurement of <1.0 g/24 hours
- •e. Persistently positive (2 out of 3 consecutive tests = trace positive) for blood on urine dipstick. In the event of a positive test eligibility may be confirmed with urine microscopy showing <5 red blood cells per high power field
- •f. TSH values outside normal range
- •3. Retinol level at screen < LLN
- •4. QTcF >470 msec (average of triplicates). QTcF values >470 msec should be confirmed by Sponsor central ECG reader. Patients with a left bundle branch block or pacemaker will not be eligible if QTcF >500 msec (QTcF values >500 msec should be confirmed by Sponsor entral ECG reader)
- •5. Uncontrolled hypertension (blood pressure >160/100)
- •6. Positive test result for human immunodeficiency virus (HIV), hepatitis B or hepatitis C
- •7. Karnofsky performance status =50
- •8. Renal insufficiency as defined by estimated creatinine clearance calculated according to the formula of CKD-EPI <45 mL/min/1.73 m2 at screen. If the calculated creatinine clearance is thought to be artificially low, a 24-hour urine creatinine clearance can be completed with prior Sponsor approval
- •9. Known type 1 or type 2 diabetes mellitus
- •10. Other causes of sensorimotor or autonomic neuropathy (e.g., autoimmune disease)
- •11. Treatment with another investigational drug, biological agent, or device within three-months of screening, or five half-lives of study agent, whichever is longer
- •12. If previously treated with Vyndaqel® must have discontinued treatment for 2 weeks prior to Study Day 1. If previously treated with Diflunisal, must have discontinued treatment for 3 days prior to Study Day 1
- •13. Previous treatment with any oligonucleotide or siRNA within 12 months of screening
- •14. Prior liver transplant or anticipated liver transplant within 1 year of screening
- •15. New York Heart Association (NYHA) functional classification of =3
- •16. Acute coronary syndrome or major surgery within 3 months of screening
- •17. Known Primary Amyloidosis
- •18. Known Leptomeningeal Amyloidosis
- •19. Anticipated survival less than 2 years
- •20. Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed prior to Study Day 1
- •21. Malignancy within 5 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated
- •22. Have any other conditions, which, in the opinion of the Investigator would make the patient unsuitable for inclusion, or could interfere with the patient participating in or completing the study
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