Familial Hypercholesterolemia - HomozygousPhase 3ORION-13NCT04659863
Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia
A Phase 3 study testing Inclisiran in people with Familial Hypercholesterolemia - Homozygous, run by Novartis Pharmaceuticals at 11 sites in 8 countries.
Trial summary
This was a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDL-C). Read more about this study
- Who is it for?
- Ages 12 to 17 years
- Conditions the study is for
- Familial Hypercholesterolemia - Homozygous
- What is being tested?
- Inclisiran
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 13 participants
- Where
- 11 sitesin 8 countries
- Study dates
- 16 Feb 2021to 18 Nov 2024
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Homozygous Familial Hypercholesterolemia (HoFH) diagnosed by genetic confirmation
- Fasting LDL-C >130 mg/dL (3.4 mmol/L) at screening
- On maximally tolerated dose of statin (investigator's discretion) with or without other lipid-lowering therapy; stable for ≥ 30 days before screening
- Male or female participants >=12 to <18 years of age at screening
Who cannot join
- Documented evidence of a null (negative) mutation in both LDLR alleles
- Heterozygous familial hypercholesterolemia (HeFH)
- Active liver disease
- Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
- Previous treatment with monoclonal antibodies directed towards PCSK9 (within 90 days of screening)
- Treatment with mipomersen or lomitapide (within 5 months of screening)
- Recent and/or planned use of other investigational medicinal products or devices
Trial locations and contacts
11 sites in 8 countries.
11 sites in 8 countries
Greece2 sites
Metropolitan Hospital
Athens, 18547
University General Hospital of Ioannina
Ioannina, GR, 455 00
Lebanon2 sites
American University of Beirut Medical Center
Beirut, 1107 2020
Hotel Dieu de France Hospital
El Achrafiyé, 166830
Turkey (Türkiye)2 sites
Novartis Investigative Site
Istanbul, TUR, 34098
Novartis Investigative Site
Izmir, 35100
United States1 site
Excel Medical Clinical Trials LLC
Boca Raton, Florida, 33434
About this study
Official title: Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Inclisiran in Adolescents (12 to Less Than 18 Years) With Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol (ORION-13)
This was a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDL-C).
Read the detailed description
This was a two-part (double-blind, inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2]) multicenter study in adolescents (aged 12 to < 18 years) with HoFH and elevated LDL-C (> 130 mg/dL; 3.4 mmol/L) on stable, individualized, optimal standard of care (SoC) background lipid-lowering therapy (including maximally tolerated statin treatment, at the Investigator's discretion) to evaluate the safety, tolerability, and efficacy of inclisiran in this pediatric patient population.
Following an approximately 4-week screening/run-in period, the study had 2 sequential parts as follows:
Part 1/Year 1: 12 months double-blind, parallel group period, in which participants were randomized in a 2:1 ratio to receive either inclisiran sodium 300 mg subcutaneous (s.c.) or placebo. The primary endpoint was assessed at Day 330.
Part 2/Year 2: 12 months single arm, open-label follow-up period, with all participants receiving inclisiran sodium 300 mg s.c.
- Being tested
- Inclisiran, Placebo
- Keywords
- Homozygous familial hypercholesterolemia (HoFH), LDL-cholesterol (LDL-C), adolescents, pediatric, small interfering ribonucleic acid (siRNA)
- Medical subject headings
- Homozygous Familial Hypercholesterolemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
InclisiranDrug
Also called KJX839
Inclisiran sodium 300 mg (equivalent to 284 mg inclisiran) in 1.5 mL solution for subcutaneous injection
PlaceboDrug
Also called Saline solution
Sterile normal saline (0.9% sodium chloride in water for injection) for subcutaneous injection
| Study group | Role | What they receive |
|---|---|---|
| Part 1 - Inclisiran | Experimental | Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270) Receives: Drug: Inclisiran |
| Part 1 - Placebo | Placebo comparator | Placebo sc injection (given at Day 1, 90 and 270) Receives: Drug: Placebo |
| Part 2 - Inclisiran (Total) | Experimental | Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360. Receives: Drug: Inclisiran |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage Change in LDL-C From Baseline to Day 330 (Part 1/Year 1)
Percentage change in low-density lipoprotein cholesterol (LDL-C) from baseline to Day 330 (Year 1)
Measured: Baseline and Day 330
Secondary outcomes (21)
Time-adjusted Percent Change in LDL-C From Baseline After Day 90 and up to Day 330 (Part 1/Year 1)
Time-adjusted percent change in LDL-C (after Day 90 and up to Day 330), calculated as the average of percent changes from baseline to Days 150, 270 and 330
Measured: Baseline, after Day 90 up to Day 330
Percent Change in LDL-C From Baseline up to Day 720
Percentage change in LDL-C from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Absolute Change in LDL-C From Baseline up to Day 720
Absolute change in LDL-C from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Percent Change in Apo B From Baseline up to Day 720
Percentage change in apolipoprotein B (Apo B) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Absolute Change in Apo B From Baseline up to Day 720
Absolute change in apolipoprotein B (Apo B) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Percent Change in Lp(a) From Baseline up to Day 720
Percentage change in lipoprotein (a) [Lp(a)] from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Absolute Change in Lp(a) From Baseline up to Day 720
Absolute change in lipoprotein (a) [Lp(a)] from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Percent Change in Non-HDL-C From Baseline up to Day 720
Percentage change in non-high density lipoprotein cholesterol (non-HDL-C) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Absolute Change in Non-HDL-C From Baseline up to Day 720
Absolute change in non-high density lipoprotein cholesterol (non-HDL-C) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Percent Change in Total Cholesterol From Baseline up to Day 720
Percentage change in total cholesterol from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Absolute Change in Total Cholesterol From Baseline up to Day 720
Absolute change in total cholesterol from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Percent Change in Triglycerides From Baseline up to Day 720
Percentage change in triglycerides from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Absolute Change in Triglycerides From Baseline up to Day 720
Absolute change in triglycerides from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Percent Change in HDL-C From Baseline up to Day 720
Percentage change in high density lipoprotein cholesterol (HDL-C) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Absolute Change in HDL-C From Baseline up to Day 720
Absolute change in high density lipoprotein cholesterol (HDL-C) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Percent Change in VLDL-C From Baseline up to Day 720
Percentage change in very low density lipoprotein cholesterol (VLDL-C) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Absolut Change in VLDL-C From Baseline up to Day 720
Absolute change in very low density lipoprotein cholesterol (VLDL-C) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Percent Change in Apo A1 From Baseline up to Day 720
Percentage change in apolipoprotein A1 (Apo A1) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Absolute Change in Apo A1 From Baseline up to Day 720
Absolute change in apolipoprotein A1 (Apo A1) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Percent Change in PCSK9 From Baseline up to Day 720
Percentage change in proprotein convertase subtilisin/kexin type 9 (PCSK9) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Absolut Change in PCSK9 From Baseline up to Day 720
Absolute change in proprotein convertase subtilisin/kexin type 9 (PCSK9) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2020
- Study start16 Feb 2021
- Primary completion30 Oct 2023
- Study completion18 Nov 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Familial Hypercholesterolemia - HomozygousStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Parallel (Year 1) to single-group (Year 2)
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04659863
- Sponsor's protocol ID
- CKJX839C12302
- Other IDs
- 2020-002755-38
- Acronym
- ORION-13
- Status last verified
- Dec 2025
- First submitted
- 2 Dec 2020
- Last updated
- 13 Jan 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Publications
Wiegman A, Peterson AL, Hegele RA, Bruckert E, Schweizer A, Lesogor A, Wang Y, Defesche J. Efficacy and Safety of Inclisiran in Adolescents With Genetically Confirmed Homozygous Familial Hypercholesterolemia: Results From the Double-Blind, Placebo-Controlled Part of the ORION-13 Randomized Trial. Circulation. 2025 Jun 24;151(25):1758-1766. doi: 10.1161/CIRCULATIONAHA.124.073233. Epub 2025 May 20.
PubMed 40391436Reijman MD, Schweizer A, Peterson ALH, Bruckert E, Stratz C, Defesche JC, Hegele RA, Wiegman A. Rationale and design of two trials assessing the efficacy, safety, and tolerability of inclisiran in adolescents with homozygous and heterozygous familial hypercholesterolaemia. Eur J Prev Cardiol. 2022 Jul 20;29(9):1361-1368. doi: 10.1093/eurjpc/zwac025.
PubMed 35175352Warden BA, Duell PB. Inclisiran: A Novel Agent for Lowering Apolipoprotein B-containing Lipoproteins. J Cardiovasc Pharmacol. 2021 Aug 1;78(2):e157-e174. doi: 10.1097/FJC.0000000000001053.
PubMed 33990512
Study at a glance
From the registry record, last updated by the sponsor on 13 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Inclisiran in Adolescents (12 to Less Than 18 Years) With Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol (ORION-13)
- ClinicalTrials.gov ID
- NCT04659863
- Acronym / sponsor ID
- ORION-13 / CKJX839C12302
- Keywords
- Homozygous familial hypercholesterolemia (HoFH), LDL-cholesterol (LDL-C), adolescents, pediatric, small interfering ribonucleic acid (siRNA)
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 16 Feb 2021 (actual)
- Primary completion
- 30 Oct 2023 (actual)
- Study completion
- 18 Nov 2024 (actual)
- First posted
- 9 Dec 2020 (actual)
- Results
- Posted 31 May 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This was a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDL-C).
Detailed description
This was a two-part (double-blind, inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2]) multicenter study in adolescents (aged 12 to < 18 years) with HoFH and elevated LDL-C (> 130 mg/dL; 3.4 mmol/L) on stable, individualized, optimal standard of care (SoC) background lipid-lowering therapy (including maximally tolerated statin treatment, at the Investigator's discretion) to evaluate the safety, tolerability, and efficacy of inclisiran in this pediatric patient population.
Following an approximately 4-week screening/run-in period, the study had 2 sequential parts as follows:
Part 1/Year 1: 12 months double-blind, parallel group period, in which participants were randomized in a 2:1 ratio to receive either inclisiran sodium 300 mg subcutaneous (s.c.) or placebo. The primary endpoint was assessed at Day 330.
Part 2/Year 2: 12 months single arm, open-label follow-up period, with all participants receiving inclisiran sodium 300 mg s.c.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
- Model description
- Parallel (Year 1) to single-group (Year 2)
| Arm | Type | Receives |
|---|---|---|
| Part 1 - Inclisiran | Experimental | Drug: InclisiranInclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270) |
| Part 1 - Placebo | Placebo comparator | Drug: PlaceboPlacebo sc injection (given at Day 1, 90 and 270) |
| Part 2 - Inclisiran (Total) | Experimental | Drug: InclisiranInclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360. |
Who may qualify
- Ages
- 12 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Homozygous Familial Hypercholesterolemia (HoFH) diagnosed by genetic confirmation
- Fasting LDL-C >130 mg/dL (3.4 mmol/L) at screening
- On maximally tolerated dose of statin (investigator's discretion) with or without other lipid-lowering therapy; stable for ≥ 30 days before screening
- Male or female participants >=12 to <18 years of age at screening
Key exclusion criteria (7 listed)
- Documented evidence of a null (negative) mutation in both LDLR alleles
- Heterozygous familial hypercholesterolemia (HeFH)
- Active liver disease
- Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
- Previous treatment with monoclonal antibodies directed towards PCSK9 (within 90 days of screening)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| InclisiranAlso: KJX839Inclisiran sodium 300 mg (equivalent to 284 mg inclisiran) in 1.5 mL solution for subcutaneous injection | Drug | Part 1 - Inclisiran, Part 2 - Inclisiran (Total) |
| PlaceboAlso: Saline solutionSterile normal saline (0.9% sodium chloride in water for injection) for subcutaneous injection | Drug | Part 1 - Placebo |
Primary outcome
Percentage Change in LDL-C From Baseline to Day 330 (Part 1/Year 1)
Time frame: Baseline and Day 330
Percentage change in low-density lipoprotein cholesterol (LDL-C) from baseline to Day 330 (Year 1)
21 secondary outcomes
Time-adjusted Percent Change in LDL-C From Baseline After Day 90 and up to Day 330 (Part 1/Year 1)
Time frame: Baseline, after Day 90 up to Day 330
Percent Change in LDL-C From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Absolute Change in LDL-C From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Percent Change in Apo B From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Absolute Change in Apo B From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Percent Change in Lp(a) From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Absolute Change in Lp(a) From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Percent Change in Non-HDL-C From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Absolute Change in Non-HDL-C From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Percent Change in Total Cholesterol From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Absolute Change in Total Cholesterol From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Percent Change in Triglycerides From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Absolute Change in Triglycerides From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Percent Change in HDL-C From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Absolute Change in HDL-C From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Percent Change in VLDL-C From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Absolut Change in VLDL-C From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Percent Change in Apo A1 From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Absolute Change in Apo A1 From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Percent Change in PCSK9 From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Absolut Change in PCSK9 From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jan 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40391436Wiegman A, Peterson AL, Hegele RA, Bruckert E, Schweizer A, Lesogor A, Wang Y, Defesche J. Efficacy and Safety of Inclisiran in Adolescents With Genetically Confirmed Homozygous Familial Hypercholesterolemia: Results From the Double-Blind, Placebo-Controlled Part of the ORION-13 Randomized Trial. Circulation. 2025 Jun 24;151(25):1758-1766. doi: 10.1161/CIRCULATIONAHA.124.073233. Epub 2025 May 20.
- Derived from this study: PubMed 35175352Reijman MD, Schweizer A, Peterson ALH, Bruckert E, Stratz C, Defesche JC, Hegele RA, Wiegman A. Rationale and design of two trials assessing the efficacy, safety, and tolerability of inclisiran in adolescents with homozygous and heterozygous familial hypercholesterolaemia. Eur J Prev Cardiol. 2022 Jul 20;29(9):1361-1368. doi: 10.1093/eurjpc/zwac025.
- Derived from this study: PubMed 33990512Warden BA, Duell PB. Inclisiran: A Novel Agent for Lowering Apolipoprotein B-containing Lipoproteins. J Cardiovasc Pharmacol. 2021 Aug 1;78(2):e157-e174. doi: 10.1097/FJC.0000000000001053.
- A Plain Language Trial Summary is available on www.novctrd.comLink provided by the sponsor.
- A Pediatric Plain Language Trial Summary is available on www.novctrd.comLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
11 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Inclisiran in people with Familial Hypercholesterolemia - Homozygous, run by Novartis Pharmaceuticals at 11 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in Greece, Lebanon, Turkey (Türkiye), United States, Canada and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 31 May 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage Change in LDL-C From Baseline to Day 330 (Part 1/Year 1) (time frame: Baseline and Day 330). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04659863, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Jan 2026.