A Study of AZD4063 in PLN R14del Dilated Cardiomyopathy

A vizsgálat adatai

Ki végzi

AstraZeneca

Azonosító

NCT07241104

A regiszteradat frissítve

2026. szeptember 4.

Eredeti leírás

The purpose of the study is to assess the safety, tolerability and the pharmacokinetics (PK) of AZD4063 after single dose administration in participants with phospholamban (PLN) R14del dilated cardiomyopathy.A teljes eredeti leírásKevesebb

A Phase I First-in-human Study to Investigate Safety, Tolerability, and Pharmacokinetics of AZD4063 in Adults With Phospholamban R14del Dilated Cardiomyopathy

The purpose of the study is to assess the safety, tolerability and the pharmacokinetics (PK) of AZD4063 after single dose administration in participants with phospholamban (PLN) R14del dilated cardiomyopathy.

Bevonási feltételek: Participants must be 18 to 80 years of age inclusive, at the time of Screening; Participants with pre-existing positive screening for R14 del PLN mutation; Participants with screening Left ventricular eject fraction ≤ 45% as assessed by echocardiography; Participants with New York Heart Association (NYHA) function class I-III; Participants on stable medical therapy for at least 6 weeks prior to Screening and during the Screening period, with no significant improvement in heart failure; Participants with implantable cardioverter-defibrillator (ICD) or Cardiac resynchronization therapy device (CRT-D); Participants with Body mass index (BMI) within the range 18-35 kg/m2; Females of childbearing potential must not be lactating, and if heterosexually active must agree to use an approved method of highly effective contraception; All women of childbearing potential must have a negative pregnancy test at the Screening Visit.

Kizáró feltételek: History of subendocardial Late Gadolinium Enhancement (LGE) suggestive of previous myocardial infarction and/or significant coronary artery disease (50% > stenosis in one major epicardial coronary artery or need for previous percutaneous coronary intervention or coronary artery bypass grafting); Participants who have previously received AZD4063 as part of this study; Participants who are part of a gene therapy trial; Participants with positive hepatitis C antibody, hepatitis B virus surface antigen or hepatitis B virus core antibody; Known to have tested positive for Human immunodeficiency virus (HIV); Any known genetic mutation associated with hereditary electrical or structural disease; Participants with sustained ventricular arrhythmia requiring treatment and considered clinically not stable by the Investigator; Routinely scheduled outpatient intravenous infusions for heart failure; Uncontrolled hypertension; Significant primary valvular disease; Congenital heart disease; Restrictive or peripartum cardiomyopathy; infiltrative disorders (sarcoidosis); Toxin exposure, systemic disease known to cause Dilated Cardiomyopathy (DCM); History of severe allergy/hypersensitivity or ongoing clinically important allergy/hypersensitivity; Any history of cardiotoxic drug exposure with documented cardiomyopathy; Noncardiac condition that limits expected lifespan to less than 1 year; Participants with a known hypersensitivity to AZD4063; Congenital long QT syndrome; Known Short QT syndrome (SQTS) or family history of SQTS; Catecholaminergic polymorphic ventricular tachycardia (CPVT as calcium ion channelopathy) and recent hospitalization for heart failure or significant ventricular arrhythmia within 3 months; Any laboratory values with the following deviations:; Alanine Transaminase >2 upper normal limit (ULN); Aspartate Transaminase >2 ULN; Total bilirubin > 2 x ULN; Estimated GFR < 30 mL/min/1.73 m2; Hemoglobin <10g/dL; QTcF < 350 ms; Left ventricular wall thickness of > 13 mm or with any relative with hypertrophic cardiomyopathy (HCM); Recent acute presentation of myocarditis; Alcohol consumption in excess; Any vital sign values with the following deviations at Screening; Systolic blood pressure > 160 mmHg; Diastolic blood pressure > 100 mmHg; Pulse rate > 100 beats per minute; Participation in another clinical study with a study intervention administered in the last 3 months.

ClinicalTrials.gov NCT07241104