EUCTR2014-003835-20-DE进行中（未招募）1 期
A Phase 3 Multicenter, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ALN TTRSC in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 200
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. Are male or female of 18 to 90 years of age (inclusive).
- •2. Have a documented TTR mutation.
- •3. Amyloid deposits in cardiac or non-cardiac tissue confirmed by Congo Red (or equivalent) staining or technetium (99mTc) scintigraphy (99mTc-3,3-diphosphono-1,2- propanodicarboxylic acid [DPD-Tc] or 99mTc-yrophosphate [PYP-Tc]) with Grade 2 or 3 cardiac uptake, centrally confirmed.
- •4. If patient has monoclonal gammopathy, TTR amyloidosis needs to be confirmed through TTR protein identification by immunohistochemistry or mass spectrometry.
- •5. Have a medical history of heart failure (HF) with at least 1 prior hospitalization for HF, which may include hospitalization for arrhythmia or pacemaker placement, OR clinical evidence of HF (as evidenced by one or more of the following: elevated jugular venous pressure, peripheral edema, shortness of breath or signs of pulmonary congestion on x-ray or auscultation) that either requires/required treatment with diuretics or is/was associated with an N terminal prohormone of B-type natriuretic peptide (NT-proBNP) >400 ng/L or B-type natriuretic peptide (BNP) >100 ng/L.
- •6. Have evidence of cardiac involvement by Screening/Baseline echocardiogram including an end-diastolic intraventricular septum thickness of =12 mm. For patients with an end-diastolic intraventricular septum thickness of <12mm, an endomyocardialbiopsy showing amyloid deposition is required.
- •7. Can walk =150 meters on a 6-minute walk test (6-MWT).
- •8. Have a Karnofsky performance status of =50%.
- •9. Symptoms of HF optimally managed with no CV hospitalizations within 4 weeks prior to consent or during Screening/Baseline.
- •10. Have adequate liver function, demonstrated by an AST and ALT = 2.0×ULN, albumin >3 g/dL (>4.35 µmol/L), and total bilirubin <2.0 mg/dL (34.2 µmol/L), unless elevation in total bilirubin is due to Gilbert's Syndrome.
- •11. No active infection with hepatitis B (HBV) or hepatitis C (HCV) by serology.
- •12. Women of child-bearing potential must have a negative pregnancy test, cannot be breastfeeding, and use 1 highly effective method of contraception in combination with a barrier method throughout study participation, and for 28 days after last dose of study drug.
- •13. Males with partners of child-bearing potential, must agree to use a condom, accompanied with spermicidal foam, gel, film, cream, or suppository, except in countries where spermicide is not available for use in combination with condom, throughout study participation and for 28 days after the last dose of study drug; males must also abstain from sperm donation after the first dose of study drug through study participation and for 28 days after the last dose of study drug.
- •14. Must be willing and able to comply with protocol-required visit schedule and visit requirements and provide informed consent or have a legal guardian who can provide informed consent.
- •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 100
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 100
排除标准
- •1. Has estimated Glomerular Filtration Rate (eGFR) <30 mL/min/1.73m2 (using the Modification of Diet in Renal Disease [MDRD] formula)
- •2. Has known primary amyloidosis (AL), leptomeningeal amyloidosis, non- FAC hereditary cardiomyopathy, hypertensive cardiomyopathy, or cardiomyopathy due to valvular heart disease
- •3. Has non-amyloid diseases affecting exercise testing (e.g., severe chronic obstructive lung disease, severe arthritis, peripheral vascular disease affecting ambulation).
- •4. Has uncontrolled hypertension
- •5. Has uncontrolled ischemic heart disease
- •6. Has uncontrolled clinically significant cardiac arrhythmia
- •7. Had acute coronary syndrome within the past 3 months
- •8. Has a Polyneuropathy Disability score >2
- •9. Has untreated hypo- or hyperthyroidism
- •10. Has a New York Heart Association (NYHA) classification of IV
- •11. Has known or suspected systemic bacterial, viral, parasitic, or fungal infection
- •12. Has known human immunodeficiency virus (HIV) infection
- •13. Current, heavy alcohol use, defined as regular consumption of greater than 2 to 3 units/day for women and 3 to 4 units/day for men (a unit of alcohol equals 1 glass of wine [125 mL], 1 measure of spirits, or ½ pint of beer), or a known history of alcohol abuse within the past 2 years.
- •14. Has received an investigational agent or device within 30 days of anticipated study drug administration or 5 half-lives of the investigational drug, whichever is longer
- •15. Is currently taking diflunisal, tafamidis, doxycycline, or tauroursodeoxycholic acid; if previously on any of these agents, must have completed a 14-day wash-out prior to start of study drug administration in this study
- •16. Had metastatic cancer within the past 5 years
- •17. History of allergic reaction to an oligonucleotide or Nacetylgalactosamine (GalNAc).
- •18. Has a history of intolerance to SC injection
- •19. Has other medical conditions or comorbidities which, in the opinion of the Investigator, would interfere with study compliance or data interpretation
- •20. Has had a heart or liver transplant, or is being considered for a transplant during the study period.
- •21. Known history of clinically significant chronic liver disease in the
- •opinion of the Investigator.
研究者
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