A Study of AZD4063 in PLN R14del Dilated Cardiomyopathy

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

A Study of AZD4063 in PLN R14del Dilated Cardiomyopathy (NCT07241104) is a Phase 1 interventional studying Dilated Cardiomyopathy, sponsored by AstraZeneca. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

About This Trial

What Stage of Research Is This?

Phase 1 trials test a new treatment for the first time in humans, focusing on safety, dosing, and how the body processes the drug. For Dilated Cardiomyopathy, a Phase 1 study typically enrolls a small number of participants — often healthy volunteers or patients who have exhausted standard treatment options. Phase 1 results determine whether a treatment moves into larger Phase 2 efficacy studies.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

With a target enrollment of 31 participants, this is a small study — typical of early-phase research, rare-disease trials, or pilot studies designed to generate preliminary signal before a larger study is launched.

Who May Be Eligible (Plain English)

Who May Qualify: - 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. Who Should NOT Join This 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 weakened immune system 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) ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language

Inclusion Criteria: * 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. Exclusion Criteria: * 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: 1. Alanine Transaminase \>2 upper normal limit (ULN) 2. Aspartate Transaminase \>2 ULN 3. Total bilirubin \> 2 x ULN 4. Estimated GFR \< 30 mL/min/1.73 m2 5. Hemoglobin \<10g/dL * Any vital sign values with the following deviations at Screening 1. Systolic blood pressure \> 160 mmHg 2. Diastolic blood pressure \> 100 mmHg 3. Pulse rate \> 100 beats per minute * 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 * Participation in another clinical study with a study intervention administered in the last 3 months * Participants with a known hypersensitivity to AZD4063 * Participants who are part of a gene therapy trial

Treatments Being Tested

DRUG

AZD4063

AZD4063 will be administered in the SAD and MAD part of the study as solution for injection via subcutaneous route of administration.

Locations (4)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

Research Site

Amsterdam, Netherlands

Research Site

Groningen, Netherlands

Research Site

Rotterdam, Netherlands

Research Site

Utrecht, Netherlands

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT07241104), the sponsor (AstraZeneca), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT07241104 clinical trial studying?

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. The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT07241104?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT07241104?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT07241104. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT07241104. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.