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A Study to Find Out How Safe and Effective AZD0780 Is for Adults with High 'Bad' Cholesterol from an Inherited Condition
EU CTIS ID: 2025-520520-17-00
What this study is testing
To compare the effect of treatment with AZD0780 versus placebo on LDL-C at 12 weeks
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- ≥ 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 maximum 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.
You likely can't join if
- 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 (CKD-EPI formula; Delgado et al 2022, Inker et al 2021) - 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 > 5X ULN - Urine albumin to creatinine ratio ≥ 500mg/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
See the full eligibility criteria
Who can join
- ≥ 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 maximum 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.
What rules you out
- 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 (CKD-EPI formula; Delgado et al 2022, Inker et al 2021) - 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 > 5X ULN - Urine albumin to creatinine ratio ≥ 500mg/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.
The study team makes the final eligibility decision.
Where it's taking place
- Chile
- Vietnam
- Taiwan
- Australia
- United States
- Argentina
- New Zealand
- South Africa
- Canada
- Japan
- Turkey
- Korea, Republic of
- Brazil
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Chile; Vietnam; Taiwan; Australia; United States; Argentina and 7 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.