A Study of AZD4063 in PLN R14del Dilated Cardiomyopathy
Key facts
- Phase
- Phase 1
This trial has no sites in United States.
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Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 80 years old?
Source: NCT07241104, who can take part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- AstraZeneca
- Trial code
- NCT07241104
- Record updated
- 4 September 2026
- Original description
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.Read full original descriptionShow less
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.
Inclusion: 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.
Exclusion: 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.