Last updated 11 days ago
A Phase III Study to Assess the Effect of AZD0780 on LDL-C in Patients With HeFH
405 patients around the world
Available in Chile, Brazil, Argentina, United States
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 maximally tolerated
lipid-lowering regimen including maximally tolerated statin therapy. 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.
AstraZeneca
405Patients around the world
This study is for people with
Hypercholesterolemia
Familial hypercholesterolemia
Requirements for the patient
From 18 Years
All Gender
Medical requirements
- ≥ 18 years of age at the time of signing the ICF.Diagnosis of HeFH by genetic confirmation or a definite clinical diagnosis, ie, a score > x using the Dutch Lipid Network or equivalent as per internationally accepted diagnostic algorithms.Fasting serum by central laboratory at screening as follows: LDL-C ≥ 55 mg/dL (≥ 1.4 mmol/L) in participants with HeFH and clinical ASCVD or ≥ 70 mg/dL (≥ 1.8 mmol/L) in HeFH without clinical ASCVD.Participants should be receiving a maximally tolerated lipid lowering regimen including a maximally tolerated statin.Participants must achieve a stable dose (> 28 days) of lipid lowering therapies before screening.Participants who are judged by the treating physician not to tolerate high intensity statins may be included if treated with a low- or moderate intensity statin dose.Participants not receiving any statins must have documented intolerable side effects to at least 2 different statins, including one at the lowest standard dose or on a chronic medication that would prohibit the use of a statin.
- Homozygous familial hypercholesterolaemia, LDL apheresis or plasma apheresis within 12 months prior to screening, or any other underlying known disease or condition that may interfere with interpretation of the clinical study results as judged by the Investigator.Any of the following laboratory values at screening.Calculated eGFR < 15 mL/min/1.73 m2.AST or ALT > 3 × ULN.TBL > 2 × ULN except for patients with Gilberts syndrome, where TBL 3 × ULN is acceptable provided direct bilirubin < 1.5 × ULN.Fasting triglycerides ≥ 400 mg/dL (≥ 4.52 mmol/L).Creatine kinase > 5 × ULN.Urine albumin-to-creatinine ratio ≥ 500 mg/g.Uncontrolled type 2 diabetes mellitus defined as HbA1C ≥ 9.5% at screening.Inadequately treated hypothyroidism defined as TSH > 1.5 ULN at screening or participants whose thyroid replacement therapy was initiated or modified within the last 3 months prior to screening.Use of mipomersen or lomitapide within 12 months prior to screening or planned use during the study.Use of gemfibrozil within 1 week prior to screening or planned use during the study.Use of PCSK-9 inhibitors: evolocumab/alirocumab within 12 weeks of the screening visit or planned use during the study or inclisiran within 18 months of the screening visit or planned use during the study.Any other approved PCSK-9 inhibitor use within 5 half lives prior to the screening visit or planned use during the study.
StudyAZURE-HeFH
SponsorAstraZeneca
Study typeInterventional
Conditions
Familial hypercholesterolemia
Requirements
From 18 YearsAll Gender
Unique study IDclinicaltrials.gov
NCT07000136
