A Study of AZD4063 in PLN R14del Dilated Cardiomyopathy

Sponsor
AstraZeneca
Study ID
NCT07241104
Phase
PHASE1
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - 80 Years
Healthy Volunteers
Not accepted

Interventions

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

Study Details

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.

Key Dates

Start date
Dec 1, 2025
Status verified
Mar 2026
Primary completion
Nov 22, 2027
Completion
Nov 22, 2027

Study Design

Enrollment
31 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort 1 (SAD): Dose 1 of AZD4063
    Participants will receive Dose 1 of AZD4063 via SC injection in Cohort 1 of SAD.
  • Experimental: Cohort 2 (SAD): Dose 2 of AZD4063
    Participants will receive Dose 2 of AZD4063 via SC injection in Cohort 2 of SAD.
  • Experimental: Cohort 3 (SAD): Dose 3 of AZD4063
    Participants will receive Dose 3 of AZD4063 via SC injection in Cohort 3 of SAD.
  • Experimental: Cohort 1 (MAD): Dose 4 of AZD4063
    Participants will receive Dose 4 of AZD4063 via SC injection in Cohort 1 of MAD.
  • Experimental: Cohort 2 (MAD): Dose 5 of AZD4063
    Participants will receive Dose 5 of AZD4063 via SC injection in Cohort 2 of MAD.
  • Experimental: Cohort 3 (MAD): Dose 6 of AZD4063
    Participants will receive Dose 6 of AZD4063 via SC injection in Cohort 3 of MAD.
  • Experimental: Optional Cohort 1 (SAD): Dose 7 of AZD4063
    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.
  • Experimental: Optional Cohort 2 (SAD): Dose 8 of AZD4063
    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.
  • Experimental: Optional Cohort 1 (MAD): Dose 9 of AZD4063
    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.
  • Experimental: Optional Cohort 2 (MAD): Dose 10 of AZD4063
    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.

Primary Outcome Measure

Number of participants with adverse events (AEs) [ 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 155 ]

Central Contacts

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