A 2-Part, Phase 2/3 Study to Assess the Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia Part A - Open-label, Single-arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia Part B - Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Safety, Tolerability and Efficacy of AMG 145 in Subjects - TESLA - 20110233

Amgen0 个研究点目标入组 3 人开始时间: 待定最近更新: 2024年4月29日

适应症

试验速览

暂无简介。

•Males and females, * 12 to * 80 years of age with a diagnosis of homozygous familial
•hypercholesterolemia by genetic confirmation or a clinical diagnosis based on a history of an
•untreated LDL cholesterol concentration greater than 500 mg/dL (13 mmol/L) together with either
•xanthoma before 10 years of age or evidence of heterozygous familial hypercholesterolemia in
•both parents.
•For enrollment, subjects have to be stable on a low-fat diet and their pre-existing, lipid-lowering
•therapies (such as statins, cholesterol-absorption inhibitors, bile-acid sequestrants, nicotinic acid,
•or combinations thereof) for at least 4 weeks, with fasting central lab LDL cholesterol
•concentration >130 mg/dL (3.4 mmol/L), central lab triglyceride concentration < 400 mg/dL
•(4.5 mmol/L), and bodyweight of 40 kg or greater at screening. Patients are should not change
•their background lipid-lowering drug during the trial.

•LDL or plasma apheresis within 8 weeks prior to enrollment; use of
•Mipomersen within 5 months of screening; New York Heart Failure Association (NYHA) class III
•or IV or last known left ventricular ejection fraction < 30%; cardiac arrhythmia within past
•3 months that is not controlled by medication; myocardial infarction, unstable angina,
•percutaneous coronary intervention (PCI), coronary artery bypass graft (CABG) or stroke within
•3 months of enrollment; planned cardiac surgery or revascularization; systolic blood pressure
•(SBP) > 180 mmHg or diastolic BP (DBP) > 110 mmHg; requiring statin up-titration within
•4 weeks of screening; estimated glomerular filtration rate (eGFR) < 30 ml/min/1.73m2; persistent
•aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 3x ULN, creatine kinase
•(CK) > 5x ULN without a known source; known major active infection, or major hematologic,
•renal, metabolic, gastrointestinal or endocrine dysfunction; deep vein thrombosis or pulmonary
•embolism within 3 months prior to enrollment.

发起方