Ignite Creation Date:
2025-12-24 @ 7:56 PM
Ignite Modification Date:
2026-03-06 @ 8:51 AM
Study NCT ID:
NCT07241104
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-11-21
First Post:
2025-11-17
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Study of AZD4063 in PLN R14del Dilated Cardiomyopathy
Sponsor:
AstraZeneca
Organization:
Study Overview
Official Title:
A Phase I First-in-human Study to Investigate Safety, Tolerability, and Pharmacokinetics of AZD4063 in Adults With Phospholamban R14del Dilated Cardiomyopathy
Status:
NOT_YET_RECRUITING
Status Verified Date:
2025-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
PULSE
Brief 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.
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.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: