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Dilated CardiomyopathyPhase 1PULSENCT07241104

A Study of AZD4063 in PLN R14del Dilated Cardiomyopathy

A Phase 1 study testing AZD4063 in people with Dilated Cardiomyopathy, run by AstraZeneca at 4 sites in Netherlands.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

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 more about this study

Who is it for?
Ages 18 to 80 years
Conditions the study is for
Dilated Cardiomyopathy
What is being tested?
AZD4063
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
23 participants
Where
4 sitesNetherlands
Study dates
1 Dec 2025to 22 Nov 2027
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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)
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  • 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.

Who cannot join

  • 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
Show all 35Show fewer
  • 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
  • Diastolic blood pressure > 100 mmHg
  • 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

Trial locations and contacts

4 sites in 1 country. Contact the study team or a site near you to ask about taking part.

AstraZeneca Clinical Study Information Center contact

1-877-240-9479

information.center@astrazeneca.com

Netherlands4 sites, 4 recruiting

  • Research Site

    Amsterdam, 1081 HV

    Recruiting

  • Research Site

    Groningen, 9713 GZ

    Recruiting

  • Research Site

    Rotterdam, 3015 GD

    Recruiting

  • Research Site

    Utrecht, 3584 CX

    Recruiting

Other trials in Netherlands

About this study

Official title: 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.

Read the detailed description

This is a Phase 1, first in human, unblinded, ascending dose study which will consist of a SAD (single ascending dose) part and an Optional part.

The SAD part of the study will assess the single doses of AZD4063 across 4 cohorts. It will consist of:

  • A Screening period
  • A Treatment period: The participants will receive a single dose of AZD4063 by subcutaneous (SC) injection
  • A Follow-up period

Optional cohorts may be added based on emerging safety, PK and PD data of the preceding cohorts.

Being tested
AZD4063
Keywords
Single Ascending Dose, Phospholamban
Medical subject headings
Cardiomyopathy, Dilated

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AZD4063Drug

    AZD4063 will be administered as solution for injection via subcutaneous route of administration.

Study groups in NCT07241104
Study groupRoleWhat they receive
Cohort 1 (SAD): Dose 1 of AZD4063ExperimentalParticipants will receive Dose 1 of AZD4063 via SC injection in Cohort 1 of SAD.
Receives: Drug: AZD4063
Cohort 2 (SAD): Dose 2 of AZD4063ExperimentalParticipants will receive Dose 2 of AZD4063 via SC injection in Cohort 2 of SAD.
Receives: Drug: AZD4063
Cohort 3 (SAD): Dose 3 of AZD4063ExperimentalParticipants will receive Dose 3 of AZD4063 via SC injection in Cohort 3 of SAD.
Receives: Drug: AZD4063
Cohort 4 (SAD): Dose 4 of AZD4063ExperimentalParticipants will receive Dose 4 of AZD4063 via SC injection in Cohort 4 of SAD.
Receives: Drug: AZD4063
Optional Cohort 1 (SAD): Dose 5 of AZD4063ExperimentalParticipants will receive Dose 5 of AZD4063 via SC injection in the optional cohort of the study. This additional cohort will be added depending on the findings.
Receives: Drug: AZD4063
Optional Cohort 2 (SAD): Dose 6 of AZD4063ExperimentalParticipants will receive Dose 6 of AZD4063 via SC injection in the optional cohort of the study. This additional cohort will be added depending on the findings.
Receives: Drug: AZD4063
Optional Cohort 3 (SAD): Dose 7 of AZD4063ExperimentalParticipants will receive Dose 7 of AZD4063 via SC injection in the optional cohort of the study. This additional cohort will be added depending on the findings.
Receives: Drug: AZD4063
Optional Cohort 4 (SAD): Dose 8 of AZD4063ExperimentalParticipants will receive Dose 8 of AZD4063 via SC injection in the optional cohort of the study. This additional cohort will be added depending on the findings.
Receives: Drug: AZD4063

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of participants with adverse events (AEs)

    The safety and tolerability of AZD4063 following the SC administration of single doses in participants with PLN R14del dilated cardiomyopathy will be evaluated.

    Measured: Cohorts 1 and 2: Day 1 to 80; Cohorts 3 and 4: Day 1 to 210

Secondary outcomes (7)

  1. Area under plasma concentration-time curve from 0 to infinity (AUCinf)

    The AUCinf of single ascending doses of AZD4063 following SC administration will be evaluated.

    Measured: Cohorts 1 and 2: Day 1 to 80; Cohorts 3 and 4: Day 1 to 210

  2. Area under the plasma concentration-curve from 0 to the last quantifiable concentration (AUClast)

    The AUClast of single doses of AZD4063 following SC administration will be evaluated.

    Measured: Cohorts 1 and 2: Day 1 to 80; Cohorts 3 and 4: Day 1 to 210

  3. Maximum plasma drug concentration (Cmax)

    The Cmax of single doses of AZD4063 following SC administration will be evaluated.

    Measured: Cohorts 1 and 2: Day 1 to 80; Cohorts 3 and 4: Day 1 to 210

  4. Renal clearance (CLR)

    The CLR of the single doses of AZD4063 following SC administration will be evaluated.

    Measured: Cohorts 1 and 2: Day 1 to 94; Cohorts 3 and 4: Day 1 to Follow up (Day 224)

  5. Cumulative amount of analyte excreted (Ae)

    The Ae of single doses of AZD4063 following SC administration will be evaluated.

    Measured: Cohorts 1 and 2: Day 1 to 94; Cohorts 3 and 4: Day 1 to Follow up (Day 224)

  6. Fraction of dose excreted unchanged in urine (Fe)

    The Fe of single doses of AZD4063 following SC administration will be evaluated.

    Measured: Cohorts 1 and 2: Day 1 to 94; Cohorts 3 and 4: Day 1 to Follow up (Day 224)

  7. Change in endomyocardial biopsy PLN messenger ribonucleic acid (mRNA levels) from baseline to estimated peak knockdown and estimated return-to-baseline

    The effects of single ascending doses of AZD4063 on knockdown of PLN mRNA will be evaluated.

    Measured: Cohorts 1 and 2: Day 1 to 94; Cohorts 3 and 4: Day 1 to Follow up (Day 224)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted21 Nov 2025
  2. Study start1 Dec 2025
  3. Primary completion22 Nov 2027 (estimated)
  4. Study completion22 Nov 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
23 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07241104
Sponsor's protocol ID
D8340C00001
Acronym
PULSE
Status last verified
Aug 2026
First submitted
17 Nov 2025
Last updated
4 Sep 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. "Yes", indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing AZD4063 in people with Dilated Cardiomyopathy, run by AstraZeneca at 4 sites in Netherlands.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 35 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 4 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact AstraZeneca Clinical Study Information Center (1-877-240-9479, information.center@astrazeneca.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of participants with adverse events (AEs) (time frame: Cohorts 1 and 2: Day 1 to 80; Cohorts 3 and 4: Day 1 to 210). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07241104.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Sep 2026.

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