Clinical Research Directory

Browse clinical research sites, groups, and studies.

Sites Groups Studies

Back to Studies

ACTIVE NOT RECRUITING

NCT07000136

PHASE3

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

Sponsor: AstraZeneca

View on ClinicalTrials.gov

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.

Official title: 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

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

INTERVENTIONAL

Enrollment

455

Start Date

2025-06-10

Completion Date

2027-01-04

Last Updated

2026-01-16

Healthy Volunteers

Conditions

Heterozygous Familial Hypercholesterolaemia

Interventions

DRUG

AZD0780

Participants will receive daily oral dose of AZD0780

DRUG

Placebo

Participants will receive daily oral dose of placebo

Inclusion criteria: •≥ 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 \[Nordestgaard et al 2013\] or equivalent as per internationally accepted diagnostic algorithms (AHA \[Gidding et al 2015\], US MEDPED \[Williams et al 1993\], Simon Broome \[Scientific Steering Committee on behalf of the Simon Broome Register Group 1991\], or Japanese Atherosclerosis Society Guidelines \[Okamura et al 2024\]) * 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. Clinical ASCVD is defined as MI, stable or unstable angina, coronary or other arterial revascularisation, ischaemic stroke, or peripheral artery disease. * Participants should receive a background lipid lowering regimen anticipated to achieve at least a \~50% reduction in LDL-C. Except in cases of intolerance, the regimen should include a high intensity statin therapy or lower intensity statin therapy in combination with an oral agent with proven outcome benefit (eg, ezetimibe and/or bempedoic acid). Thus, the background lipid-lowering therapy must consist of one of the following: \- A high intensity LDL lowering regimen (i) A high intensity statin regimen, as defined by country specific guidelines - Oral combination therapy with ezetimibe and/or bempedoic acid is strongly recommended OR: (ii) A lower intensity statin regimen in combination with ezetimibe and/or bempedoic acid : OR: \- A maximally tolerated statin regimen - Oral combination therapy with ezetimibe and/or bempedoic acid is strongly recommended. Participants must achieve a stable background lipid lowering therapy \> 28 days before screening. Exclusion criteria: * 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 (cholesterol-lowering medications) 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.

Locations (135)

Research Site

Garden Grove, California, United States

Research Site

San Diego, California, United States

Research Site

Hialeah, Florida, United States

Research Site

Orlando, Florida, United States

Research Site

Peachtree Corners, Georgia, United States

Research Site

Chicago, Illinois, United States

Research Site

Hammond, Louisiana, United States

Research Site

Las Vegas, Nevada, United States

Research Site

New York, New York, United States

Research Site

Morganton, North Carolina, United States

Research Site

Cincinnati, Ohio, United States

Research Site

Lima, Ohio, United States

Research Site

Chattanooga, Tennessee, United States

Research Site

Houston, Texas, United States

Research Site

Humble, Texas, United States

Research Site

Kingwood, Texas, United States

Research Site

McAllen, Texas, United States

Research Site

Mesquite, Texas, United States

Research Site

Redmond, Washington, United States

Research Site

CABA, Argentina

Research Site

Ciudad Autonoma de Bs As, Argentina

Research Site

Ciudad de Buenos Aires, Argentina

Research Site

Ciudad de Buenos Aires, Argentina

Research Site

Mar del Plata, Argentina

Research Site

Rosario, Argentina

Research Site

Rosario, Argentina

Research Site

Melbourne, Australia

Research Site

Campinas, Brazil

Research Site

São Caetano do Sul, Brazil

Research Site

São Paulo, Brazil

Research Site

São Paulo, Brazil

Research Site

São Paulo, Brazil

Research Site

Plovdiv, Bulgaria

Research Site

Sofia, Bulgaria

Research Site

Sofia, Bulgaria

Research Site

Vancouver, British Columbia, Canada

Research Site

London, Ontario, Canada

Research Site

Chicoutimi, Quebec, Canada

Research Site

Montreal, Quebec, Canada

Research Site

Québec, Quebec, Canada

Research Site

Trois-Rivières, Quebec, Canada

Research Site

Santiago, Chile

Research Site

Valdivia, Chile

Research Site

Hradec Králové, Czechia

Research Site

Prague, Czechia

Research Site

Prague, Czechia

Research Site

Uherské Hradiště, Czechia

Research Site

Aarhus N, Denmark

Research Site

Copenhagen, Denmark

Research Site

Esbjerg, Denmark

Research Site

Herlev, Denmark

Research Site

Herning, Denmark

Research Site

Hvidovre, Denmark

Research Site

Viborg, Denmark

Research Site

Helsinki, Finland

Research Site

Kuopio, Finland

Research Site

Tampere, Finland

Research Site

Turku, Finland

Research Site

Bron, France

Research Site

Dijon, France

Research Site

Marseille, France

Research Site

Paris, France

Research Site

Saint-Herblain, France

Research Site

Berlin, Germany

Research Site

Dresden, Germany

Research Site

Leipzig, Germany

Research Site

Budapest, Hungary

Research Site

Budapest, Hungary

Research Site

Debrecen, Hungary

Research Site

Nyíregyháza, Hungary

Research Site

Székesfehérvár, Hungary

Research Site

Chūōku, Japan

Research Site

Fukushima, Japan

Research Site

Hamamatsu, Japan

Research Site

Ichikawa-shi, Japan

Research Site

Kanazawa, Japan

Research Site

Kanazawa, Japan

Research Site

Kishiwada-shi, Japan

Research Site

Kita-gun, Japan

Research Site

Komatsu-shi, Japan

Research Site

Minatoku, Japan

Research Site

Minatoku, Japan

Research Site

Okayama, Japan

Research Site

Okayama, Japan

Research Site

Osaka, Japan

Research Site

Suita-shi, Japan

Research Site

Takatsuki-shi, Japan

Research Site

Tamanashi, Japan

Research Site

Urasoe-Shi, Japan

Research Site

Yokohama, Japan

Research Site

Amsterdam, Netherlands

Research Site

Roosendaal, Netherlands

Research Site

Rotterdam, Netherlands

Research Site

Utrecht, Netherlands

Research Site

Christchurch, New Zealand

Research Site

Grafton, New Zealand

Research Site

Bodø, Norway

Research Site

Grålum, Norway

Research Site

Oslo, Norway

Research Site

Oslo, Norway

Research Site

Trondheim, Norway

Research Site

Bratislava, Slovakia

Research Site

Bratislava, Slovakia

Research Site

Košice, Slovakia

Research Site

Nitra, Slovakia

Research Site

Žilina, Slovakia

Research Site

Gyeonggi-do, South Korea

Research Site

Seoul, South Korea

Research Site

Seoul, South Korea

Research Site

Seoul, South Korea

Research Site

A Coruña, Spain

Research Site

Barcelona, Spain

Research Site

Córdoba, Spain

Research Site

Reus, Spain

Research Site

Seville, Spain

Research Site

Gothenburg, Sweden

Research Site

Linköping, Sweden

Research Site

Lund, Sweden

Research Site

Stockholm, Sweden

Research Site

Stockholm, Sweden

Research Site

Uppsala, Sweden

Research Site

Västerås, Sweden

Research Site

Kaohsiung City, Taiwan

Research Site

Taichung, Taiwan

Research Site

Taipei, Taiwan

Research Site

Taipei, Taiwan

Research Site

Adana, Turkey (Türkiye)

Research Site

Afyonkarahisar, Turkey (Türkiye)

Research Site

Ankara, Turkey (Türkiye)

Research Site

Izmir, Turkey (Türkiye)

Research Site

İzmit, Turkey (Türkiye)

Research Site

Hanoi, Vietnam

Research Site

Ho Chi Minh City, Vietnam

Research Site

Ho Chi Minh City, Vietnam

Research Site

Hồ Chí Minh, Vietnam

Clinical Research Directory

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

Summary

Key Details

Conditions

Interventions

Eligibility Criteria

Locations (135)