A Phase III Study to Assess the Effect of AZD0780 on LDL-C in Patients With HeFH
NCT ID: NCT07000136
Last Updated: 2026-01-16
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
455 participants
INTERVENTIONAL
2025-06-10
2027-01-04
Brief Summary
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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.
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Detailed Description
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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
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
TRIPLE
Study Groups
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AZD0780
Participants will receive daily oral dose of AZD0780
AZD0780
Participants will receive daily oral dose of AZD0780
Placebo
Participants will receive daily oral dose of placebo
Placebo
Participants will receive daily oral dose of placebo
Interventions
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AZD0780
Participants will receive daily oral dose of AZD0780
Placebo
Participants will receive daily oral dose of placebo
Eligibility Criteria
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Inclusion Criteria
* 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
* 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.
18 Years
ALL
No
Sponsors
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AstraZeneca
INDUSTRY
Responsible Party
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Locations
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Research Site
Garden Grove, California, United States
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San Diego, California, United States
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Hialeah, Florida, United States
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Orlando, Florida, United States
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Peachtree Corners, Georgia, United States
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Chicago, Illinois, United States
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Hammond, Louisiana, United States
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Las Vegas, Nevada, United States
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New York, New York, United States
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Morganton, North Carolina, United States
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Cincinnati, Ohio, United States
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Lima, Ohio, United States
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Chattanooga, Tennessee, United States
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Houston, Texas, United States
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Humble, Texas, United States
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Kingwood, Texas, United States
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McAllen, Texas, United States
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Mesquite, Texas, United States
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Redmond, Washington, United States
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CABA, , Argentina
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Ciudad Autonoma de Bs As, , Argentina
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Ciudad de Buenos Aires, , Argentina
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Ciudad de Buenos Aires, , Argentina
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Mar del Plata, , Argentina
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Rosario, , Argentina
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Rosario, , Argentina
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Melbourne, , Australia
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Campinas, , Brazil
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São Caetano do Sul, , Brazil
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São Paulo, , Brazil
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São Paulo, , Brazil
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São Paulo, , Brazil
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Plovdiv, , Bulgaria
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Sofia, , Bulgaria
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Sofia, , Bulgaria
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Vancouver, British Columbia, Canada
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London, Ontario, Canada
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Chicoutimi, Quebec, Canada
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Montreal, Quebec, Canada
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Québec, Quebec, Canada
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Trois-Rivières, Quebec, Canada
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Santiago, , Chile
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Valdivia, , Chile
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Hradec Králové, , Czechia
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Prague, , Czechia
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Prague, , Czechia
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Uherské Hradiště, , Czechia
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Aarhus N, , Denmark
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Copenhagen, , Denmark
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Esbjerg, , Denmark
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Herlev, , Denmark
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Herning, , Denmark
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Hvidovre, , Denmark
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Viborg, , Denmark
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Helsinki, , Finland
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Kuopio, , Finland
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Tampere, , Finland
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Turku, , Finland
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Bron, , France
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Dijon, , France
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Marseille, , France
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Paris, , France
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Saint-Herblain, , France
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Berlin, , Germany
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Dresden, , Germany
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Leipzig, , Germany
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Budapest, , Hungary
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Budapest, , Hungary
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Debrecen, , Hungary
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Nyíregyháza, , Hungary
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Székesfehérvár, , Hungary
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Chūōku, , Japan
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Fukushima, , Japan
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Hamamatsu, , Japan
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Ichikawa-shi, , Japan
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Kanazawa, , Japan
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Kanazawa, , Japan
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Kishiwada-shi, , Japan
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Kita-gun, , Japan
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Komatsu-shi, , Japan
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Minatoku, , Japan
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Minatoku, , Japan
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Okayama, , Japan
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Okayama, , Japan
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Osaka, , Japan
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Suita-shi, , Japan
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Takatsuki-shi, , Japan
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Tamanashi, , Japan
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Urasoe-Shi, , Japan
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Yokohama, , Japan
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Amsterdam, , Netherlands
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Roosendaal, , Netherlands
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Rotterdam, , Netherlands
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Utrecht, , Netherlands
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Christchurch, , New Zealand
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Grafton, , New Zealand
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Bodø, , Norway
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Grålum, , Norway
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Oslo, , Norway
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Oslo, , Norway
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Trondheim, , Norway
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Bratislava, , Slovakia
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Bratislava, , Slovakia
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Košice, , Slovakia
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Nitra, , Slovakia
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Žilina, , Slovakia
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Gyeonggi-do, , South Korea
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Seoul, , South Korea
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Seoul, , South Korea
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Seoul, , South Korea
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A Coruña, , Spain
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Barcelona, , Spain
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Córdoba, , Spain
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Reus, , Spain
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Seville, , Spain
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Gothenburg, , Sweden
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Linköping, , Sweden
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Lund, , Sweden
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Stockholm, , Sweden
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Stockholm, , Sweden
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Uppsala, , Sweden
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Västerås, , Sweden
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Kaohsiung City, , Taiwan
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Taichung, , Taiwan
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Taipei, , Taiwan
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Taipei, , Taiwan
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Adana, , Turkey (Türkiye)
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Afyonkarahisar, , Turkey (Türkiye)
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Ankara, , Turkey (Türkiye)
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Izmir, , Turkey (Türkiye)
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İzmit, , Turkey (Türkiye)
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Hanoi, , Vietnam
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Ho Chi Minh City, , Vietnam
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Ho Chi Minh City, , Vietnam
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Hồ Chí Minh, , Vietnam
Countries
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Other Identifiers
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2025-520520-17
Identifier Type: REGISTRY
Identifier Source: secondary_id
D7960C00013
Identifier Type: -
Identifier Source: org_study_id
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