Find Dilated Cardiomyopathy (DCM) Clinical Trials Near You
A Phase I First-in-human Study to Investigate Safety, Tolerability, and Pharmacokinetics of AZD4063 in Adults With Phospholamban R14del Dilated Cardiomyopathy
Status: Recruiting
Location: See all (4) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 80
Healthy Volunteers: f
View:
• 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
• Participants on stable medical therapy for at least 6 weeks prior to Screening and during the Screening period, with no significant improvement in heart failure
• Participants with implantable cardioverter-defibrillator (ICD) or Cardiac resynchronization therapy device (CRT-D)
• Participants with Body mass index (BMI) within the range 18-35 kg/m2
• Females of childbearing potential must not be lactating, and if heterosexually active must agree to use an approved method of highly effective contraception
• All females must have a negative pregnancy test at the Screening Visit.
Locations
Other Locations
Netherlands
Research Site
NOT_YET_RECRUITING
Amsterdam
Research Site
RECRUITING
Groningen
Research Site
RECRUITING
Rotterdam
Research Site
NOT_YET_RECRUITING
Utrecht
Contact Information
Primary
AstraZeneca Clinical Study Information Center
information.center@astrazeneca.com
1-877-240-9479
Time Frame
Start Date:2025-12-01
Estimated Completion Date:2027-11-22
Participants
Target number of participants:31
Treatments
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.