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New treatment option for Dilated Cardiomyopathy
Official title A Study of AZD4063 in PLN R14del Dilated Cardiomyopathy
ClinicalTrials.gov ID: NCT07241104
What this study is testing
What is AZD4063?
AZD4063 is an investigational medicine, given as an injection under the skin, being studied as a potential treatment for dilated cardiomyopathy.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 80
You may be able to join 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...
You likely can't join 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
See the full eligibility 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.
- 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...
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Amsterdam, Netherlands
- Groningen, Netherlands
- Rotterdam, Netherlands
- Utrecht, Netherlands
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 80 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Amsterdam, Netherlands; Groningen, Netherlands; Rotterdam, Netherlands; Utrecht, Netherlands. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.