NCT07000136

A Phase III Study to Assess the Effect of AZD0780 on LDL-C in Patients With HeFH

A Phase III, Randomised, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Effect of AZD0780 on Low-Density Lipoprotein Cholesterol in Patients With Heterozygous Familial Hypercholesterolaemia

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 455 (actual)
• Sponsor: AstraZeneca · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a study to evaluate the efficacy and safety of AZD0780 in adults with HeFH and elevated LDL-C, either with clinical ASCVD and LDL-C levels of 55 mg/dL or higher or without clinical ASCVD and LDL-C levels of 70 mg/dL or higher. AZD0780 is a small molecule that reduces the amount of LDL-C in the blood. Placebo will be used for comparison, and neither the participants nor the Investigators will know who is receiving the AZD0780 medication and who is receiving the placebo until the end of study. The total length of the study for an individual participant will be up to approximately 56 weeks, including a screening period of up to 14 days, treatment with AZD0780 or placebo for 52 weeks, and a safety follow-up period of 10 days.

Detailed description

This is a randomised, double-blind, placebo-controlled, parallel-group Phase III study to evaluate the effect on the reduction of LDL-C and the safety and tolerability of AZD0780 versus placebo, administered as xx mg once daily orally, on top of a high intensity lipid-lowering regimen. The target population is adults ≥ 18 years of age with HeFH either with ASCVD and LDL-C ≥ 55 mg/dL, or without clinical ASCVD and LDL-C ≥ 70 mg/dL. The study will be conducted at approximately 170 centres in approximately 25 countries. The screening period is up to 14 days (and may be conditionally extended), starts at the date of signed informed consent, and ends on the day before the randomisation visit. Participants will be randomised in a 2:1 ratio to either AZD0780 or placebo for a treatment period of 52 weeks and a 10-day safety follow-up. Those randomised to the AZD0780 group will receive AZD0780 xx mg orally once daily during the treatment period, while those in the placebo group will receive matching placebo. The study will include approximately 405 randomised participants. An independent data monitoring committee will, on a regular basis, review accumulating data from the study, evaluate adverse effects of the IMP, and make recommendations regarding whether to halt or modify the study.

Conditions

• Heterozygous Familial Hypercholesterolaemia

Interventions

Type	Name	Description
DRUG	AZD0780	Participants will receive daily oral dose of AZD0780
DRUG	Placebo	Participants will receive daily oral dose of placebo

Timeline

• Start date: 2025-06-10
• Primary completion: 2027-01-04
• Completion: 2027-01-04
• First posted: 2025-06-02
• Last updated: 2026-01-16

Locations

135 sites across 24 countries: United States, Argentina, Australia, Brazil, Bulgaria, Canada, Chile, Czechia, Denmark, Finland, France, Germany, Hungary, Japan, Netherlands, New Zealand, Norway, Slovakia, South Korea, Spain, Sweden, Taiwan, Turkey (Türkiye), Vietnam

Regulatory

• FDA-regulated drug study