NCT07241104

Brief Summary

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
31

participants targeted

Target at P25-P50 for phase_1

Timeline
19mo left

Started Dec 2025

Typical duration for phase_1

Geographic Reach
1 country

4 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress22%
Dec 2025Nov 2027

First Submitted

Initial submission to the registry

November 17, 2025

Completed
4 days until next milestone

First Posted

Study publicly available on registry

November 21, 2025

Completed
10 days until next milestone

Study Start

First participant enrolled

December 1, 2025

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 22, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 22, 2027

Last Updated

March 31, 2026

Status Verified

March 1, 2026

Enrollment Period

2 years

First QC Date

November 17, 2025

Last Update Submit

March 30, 2026

Conditions

Dilated Cardiomyopathy

Keywords

Single Ascending DoseMultiple Ascending DosePhospholamban

Outcome Measures

Primary Outcomes (1)

  • Number of participants with adverse events (AEs)

    The safety and tolerability of AZD4063 following the SC administration of single and repeated doses in participants with PLN R14del dilated cardiomyopathy

    Cohort 1 (SAD): From Day 1 to Day 109, Cohorts 2 and 3 (SAD) From Day 1 to Day 99; For Cohorts 1,2 and 3 (MAD): Day 1 to Day 155

Secondary Outcomes (7)

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

    Cohort 1 (SAD): Up to Day 95, Cohorts 2 and 3 (SAD): Up to Day 85; Cohorts 1,2,3 (MAD): Up to Day 141

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

    Cohort 1 (SAD): Up to Day 95, Cohorts 2 and 3 (SAD): Up to Day 85; Cohorts 1,2,3 (MAD): Up to Day 141

  • Maximum plasma drug concentration (Cmax)

    Cohort 1 (SAD): Up to Day 95, Cohorts 2 and 3 (SAD): Up to Day 85; Cohorts 1,2,3 (MAD): Up to Day 141

  • Renal clearance (CLR)

    Cohort 1 (SAD): Up to Day 95, Cohorts 2 and 3 (SAD): Up to Day 85; Cohorts 1,2,3 (MAD): Up to Day 141

  • Cumulative amount of analyte excreted (Ae)

    Cohort 1 (SAD): Up to Day 95, Cohorts 2 and 3 (SAD): Up to Day 85; Cohorts 1,2,3 (MAD): Up to Day 141

  • +2 more secondary outcomes

Study Arms (10)

Cohort 1 (SAD): Dose 1 of AZD4063

EXPERIMENTAL

Participants will receive Dose 1 of AZD4063 via SC injection in Cohort 1 of SAD.

Drug: AZD4063

Cohort 2 (SAD): Dose 2 of AZD4063

EXPERIMENTAL

Participants will receive Dose 2 of AZD4063 via SC injection in Cohort 2 of SAD.

Drug: AZD4063

Cohort 3 (SAD): Dose 3 of AZD4063

EXPERIMENTAL

Participants will receive Dose 3 of AZD4063 via SC injection in Cohort 3 of SAD.

Drug: AZD4063

Cohort 1 (MAD): Dose 4 of AZD4063

EXPERIMENTAL

Participants will receive Dose 4 of AZD4063 via SC injection in Cohort 1 of MAD.

Drug: AZD4063

Cohort 2 (MAD): Dose 5 of AZD4063

EXPERIMENTAL

Participants will receive Dose 5 of AZD4063 via SC injection in Cohort 2 of MAD.

Drug: AZD4063

Cohort 3 (MAD): Dose 6 of AZD4063

EXPERIMENTAL

Participants will receive Dose 6 of AZD4063 via SC injection in Cohort 3 of MAD.

Drug: AZD4063

Optional Cohort 1 (SAD): Dose 7 of AZD4063

EXPERIMENTAL

Participants 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.

Drug: AZD4063

Optional Cohort 2 (SAD): Dose 8 of AZD4063

EXPERIMENTAL

Participants 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.

Drug: AZD4063

Optional Cohort 1 (MAD): Dose 9 of AZD4063

EXPERIMENTAL

Participants will receive Dose 9 of AZD4063 via SC injection in the optional cohort of the study. This additional cohort will be added depending on the findings.

Drug: AZD4063

Optional Cohort 2 (MAD): Dose 10 of AZD4063

EXPERIMENTAL

Participants will receive Dose 10 of AZD4063 via SC injection in the optional cohort of the study. This additional cohort will be added depending on the findings.

Drug: AZD4063

Interventions

AZD4063DRUG

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

Cohort 1 (MAD): Dose 4 of AZD4063Cohort 1 (SAD): Dose 1 of AZD4063Cohort 2 (MAD): Dose 5 of AZD4063Cohort 2 (SAD): Dose 2 of AZD4063Cohort 3 (MAD): Dose 6 of AZD4063Cohort 3 (SAD): Dose 3 of AZD4063Optional Cohort 1 (MAD): Dose 9 of AZD4063Optional Cohort 1 (SAD): Dose 7 of AZD4063Optional Cohort 2 (MAD): Dose 10 of AZD4063Optional Cohort 2 (SAD): Dose 8 of AZD4063

Eligibility Criteria

Age18 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

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
  • +19 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Research Site

Amsterdam, 1081 HV, Netherlands

NOT YET RECRUITING

Research Site

Groningen, 9713 GZ, Netherlands

RECRUITING

Research Site

Rotterdam, 3015 GD, Netherlands

RECRUITING

Research Site

Utrecht, 3584 CX, Netherlands

RECRUITING

MeSH Terms

Conditions

Cardiomyopathy, Dilated

Condition Hierarchy (Ancestors)

CardiomegalyHeart DiseasesCardiovascular DiseasesCardiomyopathiesLaminopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

AstraZeneca Clinical Study Information Center

CONTACT

1-877-240-9479information.center@astrazeneca.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 17, 2025

First Posted

November 21, 2025

Study Start

December 1, 2025

Primary Completion (Estimated)

November 22, 2027

Study Completion (Estimated)

November 22, 2027

Last Updated

March 31, 2026

Record last verified: 2026-03

Data Sharing

IPD Sharing
Will share

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.

Time Frame
AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA Pharma Data Sharing Principles. For details of our timelines, please refer to our disclosure commitment at: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
Access Criteria
When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.
More information

Locations

NL(4)