Health TrialsbyHealthPanel

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 dose administration in participants with phospholamban (PLN) R14del dilated cardiomyopathy.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

    Drug

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

Treatment groups

23 Participants
are divided into 8 treatment groups

8

Treatment groups

See 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 4 (SAD): Dose 4 of AZD4063Experimental treatment 1 intervention
Group E: Optional Cohort 1 (SAD): Dose 5 of AZD4063Experimental treatment 1 intervention
Group F: Optional Cohort 2 (SAD): Dose 6 of AZD4063Experimental treatment 1 intervention
Group G: Optional Cohort 3 (SAD): Dose 7 of AZD4063Experimental treatment 1 intervention
Group H: Optional Cohort 4 (SAD): Dose 8 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 doses in participants with PLN R14del dilated cardiomyopathy will be evaluated.

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

Secondary outcomes

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.

Time frame
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.

Time frame
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.

Time frame
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.

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

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

AstraZeneca

Lead sponsor

Source ClinicalTrials.gov
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