Actively Recruiting
A Phase I First-in-human Study to Investigate Safety, Tolerability, and Pharmacokinetics of AZD4063 in Adults With Phospholamban R14del Dilated Cardiomyopathy
Led by AstraZeneca · Updated on 2026-03-31
31
Participants Needed
4
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating AZD4063 in adults with phospholamban (PLN) R14del dilated cardiomyopathy to assess its safety, tolerability, and how the body processes the drug after single and multiple doses. This Phase 1, first-in-human study includes various dose levels to better understand how AZD4063 works in this specific heart condition. The study has two main parts: single ascending dose (SAD) and multiple ascending dose (MAD) cohorts. Participants in SAD receive one subcutaneous injection of AZD4063 at increasing doses across three groups, followed by a follow-up period. After reviewing SAD data, the MAD part begins, involving multiple subcutaneous doses across three groups with follow-up. Optional cohorts may be added depending on emerging safety and pharmacokinetic data. Participants will undergo screening before each part and receive injections as assigned to their cohort. Researchers will monitor for adverse events and measure drug levels in the blood over several weeks. They will also assess heart function and molecular markers to understand how the drug affects the condition. The study includes close safety monitoring throughout, with total observation periods ranging up to 155 days depending on the cohort.
CONDITIONS
Brief Title
A Study of AZD4063 in PLN R14del Dilated Cardiomyopathy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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 will not qualify if you...
- 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
- 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 are part of a gene therapy trial
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to approximately 20 weeks depending on cohort
Participants receive one or multiple doses of AZD4063 by subcutaneous injection depending on their assigned cohort. This includes single ascending dose (SAD) cohorts, multiple ascending dose (MAD) cohorts, and optional cohorts added based on emerging data.
Multiple visits for dosing and assessments according to cohort assignment
Duration - Up to Day 155 depending on cohort
Participants are monitored for safety, tolerability, and pharmacokinetics after completing treatment.
Several follow-up visits for safety and pharmacokinetic assessments
Trial Site Locations
Total: 4 locations
1
Research Site
Amsterdam, Netherlands, 1081 HV
Not Yet Recruiting
2
Research Site
Groningen, Netherlands, 9713 GZ
Actively Recruiting
3
Research Site
Rotterdam, Netherlands, 3015 GD
Actively Recruiting
4
Research Site
Utrecht, Netherlands, 3584 CX
Actively Recruiting
Research Team
A
AstraZeneca Clinical Study Information Center
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
10
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