A Study of AZD4063 in PLN R14del Dilated Cardiomyopathy
ConditionDilated Cardiomyopathy
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-80
SponsorAstraZeneca
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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
Trial design
Design model
Sequential
Treatments tested in this trial
AZD4063
DrugAZD4063 will be administered in the SAD and MAD part of the study as solution for injection via subcutaneous route of administration.
Treatment groups
31 Participants
are divided into 10 treatment groups10
Treatment groupsSee each treatment group below.
Group A: Cohort 1 (SAD): Dose 1 of AZD4063Experimental treatment 1 intervention
Group B: Cohort 2 (SAD): Dose 2 of AZD4063Experimental treatment 1 intervention
Group C: Cohort 3 (SAD): Dose 3 of AZD4063Experimental treatment 1 intervention
Group D: Cohort 1 (MAD): Dose 4 of AZD4063Experimental treatment 1 intervention
Group E: Cohort 2 (MAD): Dose 5 of AZD4063Experimental treatment 1 intervention
Group F: Cohort 3 (MAD): Dose 6 of AZD4063Experimental treatment 1 intervention
Group G: Optional Cohort 1 (SAD): Dose 7 of AZD4063Experimental treatment 1 intervention
Group H: Optional Cohort 2 (SAD): Dose 8 of AZD4063Experimental treatment 1 intervention
Group I: Optional Cohort 1 (MAD): Dose 9 of AZD4063Experimental treatment 1 intervention
Group J: Optional Cohort 2 (MAD): Dose 10 of AZD4063Experimental treatment 1 intervention
Trial outcomes
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
Time frame
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 155Secondary outcomes
1
Area under plasma concentration-time curve from 0 to infinity (AUCinf)
The AUCinf of single and repeated dose of AZD4063 following SC administration will be evaluated.
Time frame
Cohort 1 (SAD): Up to Day 95, Cohorts 2 and 3 (SAD): Up to Day 85; Cohorts 1,2,3 (MAD): Up to Day 1412
Area under the plasma concentration-curve from 0 to the last quantifiable concentration (AUClast)
The AUClast of single and repeated dose of AZD4063 following SC administration will be evaluated.
Time frame
Cohort 1 (SAD): Up to Day 95, Cohorts 2 and 3 (SAD): Up to Day 85; Cohorts 1,2,3 (MAD): Up to Day 1413
Maximum plasma drug concentration (Cmax)
The Cmax of single and repeated dose of AZD4063 following SC administration will be evaluated.
Time frame
Cohort 1 (SAD): Up to Day 95, Cohorts 2 and 3 (SAD): Up to Day 85; Cohorts 1,2,3 (MAD): Up to Day 1414
Renal clearance (CLR)
The CLR of the single and repeated dose of AZD4063 following SC administration will be evaluated.
Time frame
Cohort 1 (SAD): Up to Day 95, Cohorts 2 and 3 (SAD): Up to Day 85; Cohorts 1,2,3 (MAD): Up to Day 141Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov