A Phase 3, Prospective, Multicenter, Open Label, 2-Part Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of ALXN2060 in Japanese Participants with Symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM).

Ishioka Tatsunari0 个研究点目标入组 22 人开始时间: 2020年12月4日最近更新: 2024年4月1日

适应症

试验速览

暂无简介。

•1. Established diagnosis of ATTR-CM with either wild-type TTR or a variant TTR genotype .
•2. History of heart failure evidenced by at least one prior hospitalization for heart failure or clinical evidence of heart failure without prior heart failure hospitalization manifested by signs or symptoms of volume overload or elevated pressures or heart failure symptoms that required or requires ongoing treatment with a diuretics.
•3. New York Heart Association Class I-III symptoms due to ATTR-CM.
•4. On stable doses of cardiovascular medical therapy.
•5. Completed >=150 meters on the 6MWT on 2 tests prior to Day 1.
•6. Left ventricular (LV) wall (interventricular septum or LV posterior wall) thickness >=12 mm
•7. Biomarkers of myocardial wall stress: N-terminal pro-brain-type natriuretic peptide (NT-proBNP)300 pg/m

•1. Acute myocardial infarction, acute coronary syndrome or coronary revascularization, or experienced stroke or transient ischemic attack within 90 days prior to screening.
•2. Hemodynamic instability at screening.
•3. Likely to undergo heart transplantation within a year of screening.
•4. Current treatment with marketed drug products and other investigational agents for the treatment of ATTR-CM.
•5. Current treatment with calcium channel blockers with conduction system effects (for example, verapamil, diltiazem). The use of dihydropyridine calcium channel blockers is allowed.
•6. Confirmed diagnosis of light-chain (AL) amyloidosis.
•7. Biomarkers of myocardial wall stress: NT-ProBNP >= 8,500 pg/mL.
•8. Measure of kidney function, estimated glomerular filtration rate by Modification of Diet in Renal Disease formula < 30 mL/minute/1.73 meters squared.

发起方

Ishioka Tatsunari