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New pill could tame inherited high cholesterol in major trial

NCT ID NCT07000136

Disease control Sponsor: AstraZeneca Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 17, 2026 · Updated 2 times

Summary

This study tests an experimental drug called AZD0780 in 473 adults with a genetic condition that causes very high cholesterol (heterozygous familial hypercholesterolemia). The drug aims to lower LDL (bad) cholesterol more than a placebo over 12 weeks, with treatment lasting up to 52 weeks. Participants must have high LDL despite current treatments, and neither they nor their doctors will know who gets the real drug.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

473 people

The number who actually took part.

Started

Jun 2025

Expected to finish

Jan 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

familial hypercholesterolemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Heterozygous familial hypercholesterolaemia

Contacts and locations

Locations

  • Research Site

    Garden Grove, California, 92844, United States

  • Research Site

    San Diego, California, 92111, United States

  • Research Site

    Miami Lakes, Florida, 33016, United States

  • Research Site

    Orlando, Florida, 32807, United States

  • Research Site

    Peachtree Corners, Georgia, 30092, United States

  • Research Site

    Chicago, Illinois, 60607, United States

  • Research Site

    Hammond, Louisiana, 70403, United States

  • Research Site

    Las Vegas, Nevada, 89119, United States

  • Research Site

    New York, New York, 10029, United States

  • Research Site

    Morganton, North Carolina, 28655, United States

  • Research Site

    Cincinnati, Ohio, 45227, United States

  • Research Site

    Lima, Ohio, 45801, United States

  • Research Site

    Chattanooga, Tennessee, 37404, United States

  • Research Site

    Houston, Texas, 77043, United States

  • Research Site

    Humble, Texas, 77338, United States

  • Research Site

    Kingwood, Texas, 77345, United States

  • Research Site

    McAllen, Texas, 78503, United States

  • Research Site

    Mesquite, Texas, 75149, United States

  • Research Site

    Redmond, Washington, 98052, United States

  • Research Site

    CABA, C1006ACC, Argentina

  • Research Site

    Ciudad Autonoma de Bs As, C1023AAB, Argentina

  • Research Site

    Ciudad de Buenos Aires, C1094AAD, Argentina

  • Research Site

    Ciudad de Buenos Aires, C1425AGC, Argentina

  • Research Site

    Mar del Plata, 7600, Argentina

  • Research Site

    Rosario, 2000, Argentina

  • Research Site

    Rosario, S2000PBJ, Argentina

  • Research Site

    Melbourne, 3004, Australia

  • Research Site

    Campinas, 13060-080, Brazil

  • Research Site

    São Caetano do Sul, 09521-160, Brazil

  • Research Site

    São Paulo, 04004-030, Brazil

  • Research Site

    São Paulo, 04012-180, Brazil

  • Research Site

    São Paulo, 05403-000, Brazil

  • Research Site

    Plovdiv, 4002, Bulgaria

  • Research Site

    Sofia, 1527, Bulgaria

  • Research Site

    Sofia, 1618, Bulgaria

  • Research Site

    Vancouver, British Columbia, V6Z2H2, Canada

  • Research Site

    London, Ontario, N6A 5A5, Canada

  • Research Site

    Chicoutimi, Quebec, G7H 7K9, Canada

  • Research Site

    Montreal, Quebec, H2W 1R7, Canada

  • Research Site

    Québec, Quebec, G1V 4W2, Canada

  • Research Site

    Trois-Rivières, Quebec, G9A 4P3, Canada

  • Research Site

    Santiago, 7770086, Chile

  • Research Site

    Valdivia, 5090000, Chile

  • Research Site

    Hradec Králové, 500 05, Czechia

  • Research Site

    Prague, 140 00, Czechia

  • Research Site

    Prague, 19800, Czechia

  • Research Site

    Uherské Hradiště, 686 01, Czechia

  • Research Site

    Aarhus N, 8200, Denmark

  • Research Site

    Copenhagen, 2400, Denmark

  • Research Site

    Esbjerg, 6700, Denmark

  • Research Site

    Herlev, 2730, Denmark

  • Research Site

    Herning, 7400, Denmark

  • Research Site

    Hvidovre, DK-2650, Denmark

  • Research Site

    Viborg, 8800, Denmark

  • Research Site

    Helsinki, 00290, Finland

  • Research Site

    Kuopio, 70210, Finland

  • Research Site

    Tampere, 33520, Finland

  • Research Site

    Turku, 20520, Finland

  • Research Site

    Bron, 69677, France

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    Dijon, 21079, France

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    Marseille, 13005, France

  • Research Site

    Paris, 75013, France

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    Saint-Herblain, 44093, France

  • Research Site

    Berlin, 10559, Germany

  • Research Site

    Dresden, 1307, Germany

  • Research Site

    Leipzig, 04103, Germany

  • Research Site

    Budapest, 1036, Hungary

  • Research Site

    Budapest, 1132, Hungary

  • Research Site

    Debrecen, 4032, Hungary

  • Research Site

    Nyíregyháza, 4400, Hungary

  • Research Site

    Székesfehérvár, 8000, Hungary

  • Research Site

    Chūōku, 103-0027, Japan

  • Research Site

    Fukushima, 960-1295, Japan

  • Research Site

    Hamamatsu, 432-8580, Japan

  • Research Site

    Ichikawa-shi, 272-8516, Japan

  • Research Site

    Kanazawa, 920-8530, Japan

  • Research Site

    Kanazawa, 920-8641, Japan

  • Research Site

    Kishiwada-shi, 596-8522, Japan

  • Research Site

    Kita-gun, 761-0793, Japan

  • Research Site

    Komatsu-shi, 923-8560, Japan

  • Research Site

    Minatoku, 108-0073, Japan

  • Research Site

    Okayama, 700-0804, Japan

  • Research Site

    Okayama, 702-8055, Japan

  • Research Site

    Osaka, 543-8922, Japan

  • Research Site

    Suita-shi, 564-8565, Japan

  • Research Site

    Takatsuki-shi, 569-8686, Japan

  • Research Site

    Tamanashi, 865-0016, Japan

  • Research Site

    Urasoe-Shi, 901-2102, Japan

  • Research Site

    Yokohama, 247-8581, Japan

  • Research Site

    Amsterdam, 1105 AZ, Netherlands

  • Research Site

    Roosendaal, 4708 AE, Netherlands

  • Research Site

    Rotterdam, 3015 GD, Netherlands

  • Research Site

    Utrecht, 3584 CX, Netherlands

  • Research Site

    Christchurch, 8011, New Zealand

  • Research Site

    Grafton, 1010, New Zealand

  • Research Site

    Bodø, 8005, Norway

  • Research Site

    Grålum, 1712, Norway

  • Research Site

    Oslo, 0369, Norway

  • Research Site

    Oslo, 0586, Norway

  • Research Site

    Trondheim, 7030, Norway

  • Research Site

    Bratislava, 83101, Slovakia

  • Research Site

    Bratislava, 83106, Slovakia

  • Research Site

    Košice, 04022, Slovakia

  • Research Site

    Nitra, 949 11, Slovakia

  • Research Site

    Žilina, 010 01, Slovakia

  • Research Site

    Gyeonggi-do, 13620, South Korea

  • Research Site

    Seoul, 03080, South Korea

  • Research Site

    Seoul, 120-752, South Korea

  • Research Site

    Seoul, 5278, South Korea

  • Research Site

    A Coruña, 15006, Spain

  • Research Site

    Barcelona, 08036, Spain

  • Research Site

    Córdoba, 14004, Spain

  • Research Site

    Reus, 43204, Spain

  • Research Site

    Seville, 41013, Spain

  • Research Site

    Gothenburg, 413 46, Sweden

  • Research Site

    Linköping, 581 85, Sweden

  • Research Site

    Lund, 22242, Sweden

  • Research Site

    Stockholm, 117 27, Sweden

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    Stockholm, 141 86, Sweden

  • Research Site

    Uppsala, 75185, Sweden

  • Research Site

    Kaohsiung City, 80756, Taiwan

  • Research Site

    Taichung, 40705, Taiwan

  • Research Site

    Taipei, 10002, Taiwan

  • Research Site

    Taipei, 11217, Taiwan

  • Research Site

    Adana, 01060, Turkey (Türkiye)

  • Research Site

    Afyonkarahisar, 03030, Turkey (Türkiye)

  • Research Site

    Ankara, 06530, Turkey (Türkiye)

  • Research Site

    Izmir, 35100, Turkey (Türkiye)

  • Research Site

    İzmit, 41380, Turkey (Türkiye)

  • Research Site

    Hanoi, 100000, Vietnam

  • Research Site

    Ho Chi Minh City, 700000, Vietnam

  • Research Site

    Ho Chi Minh City, 70000, Vietnam

  • Research Site

    Hồ Chí Minh, 9000, Vietnam