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Clinical trial Recruiting at 4 sites Phase 1 Interventional Ages 18 to 80

AZD4063 for PLN R14del dilated cardiomyopathy.

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

Run by AstraZeneca · NCT07241104 · Data updated 2026-11-12

This study will test whether AZD4063 is safe and how the body processes it in adults with PLN R14del dilated cardiomyopathy.

Quick facts

PhasePhase 1
Study typeInterventional
Ages18 Years to 80 Years
SexAll
Enrollment23 (estimated)
SponsorAstraZeneca
Industry-sponsored
Locations4 sites (Amsterdam and 3 other locations)
StatusRecruiting
StartedDecember 1, 2025
Main results expectedNovember 22, 2027
Study endsNovember 22, 2027
Registry record updatedSeptember 4, 2026
Trial IDNCT07241104 on ClinicalTrials.gov

Is this trial a fit for you?

Likely a good fit

Adults aged 18–80 with a confirmed PLN R14del mutation, left ventricular ejection fraction ≤45%, NYHA class I–III, stable heart-failure medications, and BMI 18–35 kg/m2 are ideal candidates.

Probably not a fit

People without the PLN R14del mutation, those with active hepatitis C, pregnant or lactating individuals, or those outside the age or BMI ranges are unlikely to benefit or be eligible.

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 1, first-in-human, open-label ascending dose study will give AZD4063 by subcutaneous injection to adults with the PLN R14del mutation and dilated cardiomyopathy. The single ascending dose (SAD) portion includes three cohorts that receive a single dose with safety, pharmacokinetic (PK), and pharmacodynamic (PD) monitoring, and the multiple ascending dose (MAD) portion will begin after review of SAD safety, PK, and PD data. Optional cohorts may be added based on emerging safety, PK, and PD results. Participants will be screened, treated at one of the Dutch research sites, and followed for safety, tolerability, PK, and PD endpoints.

Why it matters

Potential benefit: If successful, AZD4063 could lead to a targeted therapy that improves heart muscle function or slows disease progression in people with PLN R14del cardiomyopathy.

How similar studies have gone: This is the first-in-human study of AZD4063, so human data are lacking and comparable human studies targeting PLN R14del are limited.

Who can join: the full rules

Show full inclusion / exclusion criteria 
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 women of childbearing potential 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 have previously received AZD4063 as part of this study
* Participants who are part of a gene therapy trial

Where it's running

  • Research Site
    Amsterdam, Netherlands     Recruiting
  • Research Site
    Groningen, Netherlands     Recruiting
  • Research Site
    Rotterdam, Netherlands     Recruiting
  • Research Site
    Utrecht, Netherlands     Recruiting

Study contacts

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

How to take part

  1. Review the eligibility criteria above with your treating physician.
  2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
  3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.
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Last reviewed 2026-11-14 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.