NCT07241104

A Study of AZD4063 in PLN R14del Dilated Cardiomyopathy

A Phase I First-in-human Study to Investigate Safety, Tolerability, and Pharmacokinetics of AZD4063 in Adults With Phospholamban R14del Dilated Cardiomyopathy

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 31 (estimated)
• Sponsor: AstraZeneca · Industry
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

This is a Phase 1, first in human, unblinded, ascending dose study which will be comprised of: 3 single ascending dose (SAD) cohorts, 3 multiple ascending dose (MAD) cohorts and optional cohorts. The SAD part of the study will assess the single doses of AZD4063 across 3 cohorts. It will consist of: * A screening period * A treatment period: The participants will receive a single dose of AZD4063 by subcutaneous (SC) injection * A follow-up period The MAD part of the study will initiate on receiving the data from SAD cohort with available safety, PK and pharmacodynamics (PD) data from all cohorts. This part of the study will consist of: * A screening period * A treatment period: The participants will receive multiple doses of AZD4063 by SC injection * A follow-up period Optional cohorts may be added based on emerging safety, PK and PD data of the SAD and MAD cohorts.

Conditions

• Dilated Cardiomyopathy

Interventions

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

Timeline

• Start date: 2025-12-01
• Primary completion: 2027-11-22
• Completion: 2027-11-22
• First posted: 2025-11-21
• Last updated: 2026-03-31

Locations

4 sites across 1 country: Netherlands