Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT07000136




Registration number
NCT07000136
Ethics application status
Date submitted
23/05/2025
Date registered
2/06/2025
Date last updated
23/07/2025

Titles & IDs
Public title
A Phase III Study to Assess the Effect of AZD0780 on LDL-C in Patients With HeFH
Scientific 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
Secondary ID [1] 0 0
2025-520520-17
Secondary ID [2] 0 0
D7960C00013
Universal Trial Number (UTN)
Trial acronym
AZURE-HeFH
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Heterozygous Familial Hypercholesterolaemia 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - AZD0780
Treatment: Drugs - Placebo

Experimental: AZD0780 - Participants will receive daily oral dose of AZD0780

Placebo comparator: Placebo - Participants will receive daily oral dose of placebo


Treatment: Drugs: AZD0780
Participants will receive daily oral dose of AZD0780

Treatment: Drugs: Placebo
Participants will receive daily oral dose of placebo
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Relative change in LDL-C from baseline to 12 weeks
Timepoint [1] 0 0
Baseline - 12 weeks
Secondary outcome [1] 0 0
Relative change in LDL-C from baseline to 12 weeks
Timepoint [1] 0 0
Baseline - 12 weeks
Secondary outcome [2] 0 0
Indicator for LDL-C < 70 mg/dL (< 1.8 mmol/L) at 12 weeks
Timepoint [2] 0 0
Baseline - 12 weeks
Secondary outcome [3] 0 0
Indicator for LDL-C < 55 mg/dL (< 1.4 mmol/L) at 12 weeks
Timepoint [3] 0 0
Baseline - 12 weeks
Secondary outcome [4] 0 0
Relative change in LDL-C from baseline to 28 weeks
Timepoint [4] 0 0
Baseline - 28 weeks
Secondary outcome [5] 0 0
Relative change in LDL-C from baseline to 52 weeks
Timepoint [5] 0 0
Baseline - 52 weeks
Secondary outcome [6] 0 0
Relative change in Apo B from baseline to 12 weeks
Timepoint [6] 0 0
Baseline - 12 weeks
Secondary outcome [7] 0 0
Relative change in non-HDL-C from baseline to 12 weeks
Timepoint [7] 0 0
Baseline - 12 weeks
Secondary outcome [8] 0 0
Relative change in total cholesterol from baseline to 12 weeks
Timepoint [8] 0 0
Baseline - 12 weeks
Secondary outcome [9] 0 0
Relative change in Lp(a) from baseline to 12 weeks
Timepoint [9] 0 0
Baseline - 12 weeks

Eligibility
Key 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 be receiving a maximally tolerated lipid lowering regimen including a maximally tolerated statin. Ezetimibe is further strongly recommended.

1. Participants must achieve a stable dose (> 28 days) of lipid lowering therapies before screening.
2. Participants who are judged by the treating physician not to tolerate high intensity statins (according to guidelines, typically, atorvastatin = 40 mg once daily or rosuvastatin = 20 mg once daily) may be included if treated with a low- or moderate intensity statin dose.
3. 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 (according to the prescribing information for the statin in question).
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
10/06/2025
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
6/04/2027
Actual
Sample size
Target
405
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Research Site - Melbourne
Recruitment hospital [2] 0 0
Research Site - Woolloongabba
Recruitment postcode(s) [1] 0 0
3004 - Melbourne
Recruitment postcode(s) [2] 0 0
4102 - Woolloongabba
Recruitment outside Australia
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United States of America
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California
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Izmit
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Vietnam
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Hanoi
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Ho Chi Minh City
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Vietnam
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H? Chí Minh

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
AstraZeneca
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
AstraZeneca Clinical Study Information Center
Address 0 0
Country 0 0
Phone 0 0
1-877-240-9479
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment:

https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure

"Yes", indicates that AZ are accepting requests for IPD, but this does not mean all requests will be approved.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP)
When will data be available (start and end dates)?
AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA/PhRMA Data-Sharing Principles. For details of our timelines, please refer to our disclosure commitment at:

https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure
Available to whom?
When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. A Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://vivli.org/


What supporting documents are/will be available?

No Supporting Document Provided


Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT07000136