A Study of AZD4063 in PLN R14del Dilated Cardiomyopathy

Recruiting now · Phase 1

Conditions studied: Dilated Cardiomyopathy

In brief

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

Key facts

Study ID

NCT07241104

Run by

AstraZeneca

People needed

31

Starts

2025-12-01

Expected to finish

2027-11-22

Last updated by the study team

2026-07-16

Who can join

Age: 18 and older, up to 80. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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 females must have a negative pregnancy test at the Screening Visit.

You may not qualify if…

• 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
• Congenital long QT syndrome
• QTcF < 350 ms
• 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
• Participants with sustained ventricular arrhythmia requiring treatment and considered clinically not stable by the Investigator
• 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)
• Routinely scheduled outpatient intravenous infusions for heart failure
• Uncontrolled hypertension
• Significant primary valvular disease
• Congenital heart disease
• Left ventricular wall thickness of > 13 mm or with any relative with hypertrophic cardiomyopathy (HCM)
• Recent acute presentation of myocarditis
• Restrictive or peripartum cardiomyopathy; infiltrative disorders (sarcoidosis)
• Alcohol consumption in excess
• 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
• Any vital sign values with the following deviations at Screening
• Systolic blood pressure > 160 mmHg

Where it is running

• Research Site — Amsterdam, Netherlands (enrolling)
• Research Site — Groningen, Netherlands (enrolling)
• Research Site — Rotterdam, Netherlands (enrolling)
• Research Site — Utrecht, Netherlands (enrolling)

Full record on ClinicalTrials.gov

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