Elevated LDL-C and High CholesterolPhase 1STX-1150-01NCT07428473
A Study of STX-1150 in Participants With Elevated Low-Density Lipoprotein Cholesterol (LDL-C)
A Phase 1 study testing STX-1150 in people with Elevated LDL-C and High Cholesterol, run by Monash University at 3 sites in 2 countries.
Trial summary
STX-1150 is an investigational therapy designed to lower LDL-C by silencing a gene called PCSK9 in the liver. STX-1150 does not edit or permanently change the gene. STX-1150 comprises an mRNA and guide RNA (gRNA) delivered via lipid nanoparticles (LNP) for intravenous infusion. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Elevated LDL-C and High Cholesterol
- What is being tested?
- STX-1150
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 64 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 30 Jun 2026to 30 Dec 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Elevated serum LDL-C with or without LDL-C lowering medication
- Willing and able to give informed consent before initiation of any study-related procedures and willing to comply with all required study procedures
Who cannot join
- Patients with history of an ASCVD event </= 6 months.
- Any uncontrolled or serious disease, or any medical or surgical condition that may interfere with participation
- Diagnosis of familial hypercholesterolemia
- Active or history of liver disease
- Previous treatment with PCSK9-inhibitor or other prior treatment within a specified timeframe
- Clinically significant abnormal laboratory values
Trial locations and contacts
3 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
3 sites in 2 countries
New Zealand2 sites, 2 recruiting
- Recruiting
New Zealand Clinical Research (NZCR)
Christchurch, Christchurch, 8011
Thinish Sundararaj, 64 3 372 9477 ext. 7279, scroll.christchurch@nzcr.co.nz
- Recruiting
Aotearoa Clinical Trials/Middlemore Hospital
Papatoetoe, Auckland, 2025
Shalini Guleria, +64 (0) 21 476 972, shalini.guleria@aotearoatrials.nz
Australia1 site, 1 recruiting
- Recruiting
Monash Health/ Victorian Heart Hospital (VHH)
Clayton, Victoria, 3168
Debra O'Leary, PhD, +61 3 7511 1252, Debra.OLeary@monashhealth.org
About this study
Official title: A Phase 1 Open-Label Single Ascending Dose (Part 1) and Single or Multi-Dose Expansion (Part 2) Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of STX-1150 in Participants With Elevated Low-Density Lipoprotein Cholesterol (LDL-C)
STX-1150 is an investigational therapy designed to lower LDL-C by silencing a gene called PCSK9 in the liver. STX-1150 does not edit or permanently change the gene. STX-1150 comprises an mRNA and guide RNA (gRNA) delivered via lipid nanoparticles (LNP) for intravenous infusion. The mRNA produces a protein that switches off the PCSK9 gene expression without altering the DNA sequence. This process leverages natural mechanisms that regulate gene activity.
The study will enroll up to 64 participants with elevated LDL-C across sites in Australia and New Zealand. The follow-up period will be up to 1- year post-treatment.
Read the detailed description
STX-1150 is an investigational product designed to epigenetically silence the PCSK9 gene. It comprises an mRNA and a guide RNA (gRNA) delivered in a lipid nanoparticle (LNP) for intravenous (IV) infusion.
STX-1150 epigenetically silences the expression of the PCSK9 gene in the liver, thereby reducing circulating PCSK9 and LDL-C levels. The active components, an mRNA and a gRNA are encapsulated in lipid nanoparticles (LNPs) for targeted hepatic delivery. The gRNA precisely guides the complex to a specific locus within the PCSK9 gene promoter.
By reducing PCSK9 expression, STX-1150 prevents the degradation of LDL receptors (LDL-R), leading to increased LDL-R levels on hepatocytes and enhanced clearance of LDL-C from the bloodstream. This targeted and durable epigenetic silencing represents a promising therapeutic strategy for long-term LDL-C reduction, particularly benefiting patients with elevated LDL-C or a high risk for Atherosclerotic Cardiovascular Disease (ASCVD).
- Conditions
- Elevated LDL-C and High Cholesterol
- Being tested
- STX-1150
- Keywords
- LDL-C, PCSK9, Cardiovascular Disease, ASCVD, CMD, Genomic medicine, Gene Therapy, Atherosclerosis, Cholesterol, Heart Attack, Hypertension, Hypercholesterolemia, CVM, Cardiometabolic Disease, Epigenetic, CRISPR
- Medical subject headings
- Hypercholesterolemia, Cardiovascular Diseases, Atherosclerosis, Myocardial Infarction, Hypertension
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
STX-1150Drug
Drug: STX-1150 is an investigational product designed to epigenetically silence the PCSK9 gene. Epigenome modulation offers a way to silence genes without changing their underlying DNA sequence.
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 | Experimental | Part 1: An open-label, single ascending dose will serve to identify the Dose-Limiting Toxicities (DLTs) and Optimal Biological Dose (OBD) of STX-1150.
Part 2: Following Part 1, an open-label, single or multi-dose expansion of the OBD cohort will be conducted to further characterize the effect of STX-1150 and obtain additional safety data at the OBD. Receives: Drug: STX-1150 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and Severity of Treatment-Emergent Adverse Events (TEAEs)
Measured: Up to Week 52 from administration of STX-1150
Incidence and Severity of Serious Adverse Events (SAEs)
Measured: Up to Week 52 from administration of STX-1150
Incidence and Severity of Adverse Events of Special Interest (AESI)
Measured: Up to Week 52 from administration of STX-1150
Secondary outcomes (7)
Incidence of dose-limiting toxicities (DLTs)
Measured: Within 14 days from administration of STX-1150
Percent Change from Baseline in Plasma PCSK9 Concentration
Measured: Up to Week 52
Percent Change from Baseline in LDL-C
Measured: Up to 52 weeks
Plasma Concentrations of STX-1150 Lipid Components
Measured: Up to 52 weeks
Number of Participants with Treatment-Induced Immunogenicity
Measured: Up to 52 Weeks
Absolute Change from Baseline in LDL-C
Measured: Up to 52 weeks
The Absolute Change from Baseline in Plasma PCSK9 Concentration
Measured: Up to Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Feb 2026
- Study start30 Jun 2026
- Primary completion30 Dec 2028 (estimated)
- Study completion30 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Elevated LDL-C and High CholesterolStudies of the same condition, open and under way.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This Phase 1 study is designed to characterise safety, tolerability, pharmacokinetics, and pharmacodynamics of STX-1150. Part 1: An open-label, single ascending dose will serve to identify the Dose Limiting Toxicities (DLTs) and the Optimal Biological Dose (OBD) of STX-1150. Part 2: Following Part 1, an open-label, single or multi-dose expansion of the OBD cohort will be conducted to further characterise the effect of STX-1150.
- Masking
- None (open label)
- Enrolment
- 64 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Monash University
- Sponsor type
- University, hospital or other
- Collaborators
- Scribe Therapeutics Inc.
- Responsible party
- Principal investigator, Stephen Nicholls, Monash University
- Investigators
- Stephen Nicholls, MBBS, FRACP, PhD, Study chair, VHI
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07428473
- Sponsor's protocol ID
- STX-1150-01
- Acronym
- STX-1150-01
- Status last verified
- Sep 2026
- First submitted
- 1 Feb 2026
- Last updated
- 21 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Open-Label Single Ascending Dose (Part 1) and Single or Multi-Dose Expansion (Part 2) Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of STX-1150 in Participants With Elevated Low-Density Lipoprotein Cholesterol (LDL-C)
- ClinicalTrials.gov ID
- NCT07428473
- Acronym / sponsor ID
- STX-1150-01 / STX-1150-01
- Conditions
- Elevated LDL-C and High Cholesterol
- Keywords
- LDL-C, PCSK9, Cardiovascular Disease, ASCVD, CMD, Genomic medicine, Gene Therapy, Atherosclerosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 64 participants (estimated)
- Lead sponsor
- Monash UniversityUniversity, hospital or other
- Collaborators
- Scribe Therapeutics Inc.
- Study start
- 30 Jun 2026 (actual)
- Primary completion
- 30 Dec 2028 (estimated)
- Study completion
- 30 Dec 2028 (estimated)
- First posted
- 23 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
STX-1150 is an investigational therapy designed to lower LDL-C by silencing a gene called PCSK9 in the liver. STX-1150 does not edit or permanently change the gene. STX-1150 comprises an mRNA and guide RNA (gRNA) delivered via lipid nanoparticles (LNP) for intravenous infusion. The mRNA produces a protein that switches off the PCSK9 gene expression without altering the DNA sequence. This process leverages natural mechanisms that regulate gene activity.
The study will enroll up to 64 participants with elevated LDL-C across sites in Australia and New Zealand. The follow-up period will be up to 1- year post-treatment.
Detailed description
STX-1150 is an investigational product designed to epigenetically silence the PCSK9 gene. It comprises an mRNA and a guide RNA (gRNA) delivered in a lipid nanoparticle (LNP) for intravenous (IV) infusion.
STX-1150 epigenetically silences the expression of the PCSK9 gene in the liver, thereby reducing circulating PCSK9 and LDL-C levels. The active components, an mRNA and a gRNA are encapsulated in lipid nanoparticles (LNPs) for targeted hepatic delivery. The gRNA precisely guides the complex to a specific locus within the PCSK9 gene promoter.
By reducing PCSK9 expression, STX-1150 prevents the degradation of LDL receptors (LDL-R), leading to increased LDL-R levels on hepatocytes and enhanced clearance of LDL-C from the bloodstream. This targeted and durable epigenetic silencing represents a promising therapeutic strategy for long-term LDL-C reduction, particularly benefiting patients with elevated LDL-C or a high risk for Atherosclerotic Cardiovascular Disease (ASCVD).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This Phase 1 study is designed to characterise safety, tolerability, pharmacokinetics, and pharmacodynamics of STX-1150. Part 1: An open-label, single ascending dose will serve to identify the Dose Limiting Toxicities (DLTs) and the Optimal Biological Dose (OBD) of STX-1150. Part 2: Following Part 1, an open-label, single or multi-dose expansion of the OBD cohort will be conducted to further characterise the effect of STX-1150.
| Arm | Type | Receives |
|---|---|---|
| Arm 1 | Experimental | Drug: STX-1150Part 1: An open-label, single ascending dose will serve to identify the Dose-Limiting Toxicities (DLTs) and Optimal Biological Dose (OBD) of STX-1150. Part 2: Following Part 1, an open-label, single or multi-dose expansion of the OBD cohort will be conducted to further characterize the effect of STX-1150 and obtain additional safety data at the OBD. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Elevated serum LDL-C with or without LDL-C lowering medication
- Willing and able to give informed consent before initiation of any study-related procedures and willing to comply with all required study procedures
Key exclusion criteria (6 listed)
- Patients with history of an ASCVD event </= 6 months.
- Any uncontrolled or serious disease, or any medical or surgical condition that may interfere with participation
- Diagnosis of familial hypercholesterolemia
- Active or history of liver disease
- Previous treatment with PCSK9-inhibitor or other prior treatment within a specified timeframe
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 |
|---|---|---|
| STX-1150Drug: STX-1150 is an investigational product designed to epigenetically silence the PCSK9 gene. Epigenome modulation offers a way to silence genes without changing their underlying DNA sequence. | Drug | Arm 1 |
Primary outcome
Incidence and Severity of Treatment-Emergent Adverse Events (TEAEs)
Time frame: Up to Week 52 from administration of STX-1150
Incidence and Severity of Serious Adverse Events (SAEs)
Time frame: Up to Week 52 from administration of STX-1150
Incidence and Severity of Adverse Events of Special Interest (AESI)
Time frame: Up to Week 52 from administration of STX-1150
7 secondary outcomes
Incidence of dose-limiting toxicities (DLTs)
Time frame: Within 14 days from administration of STX-1150
Percent Change from Baseline in Plasma PCSK9 Concentration
Time frame: Up to Week 52
Percent Change from Baseline in LDL-C
Time frame: Up to 52 weeks
Plasma Concentrations of STX-1150 Lipid Components
Time frame: Up to 52 weeks
Number of Participants with Treatment-Induced Immunogenicity
Time frame: Up to 52 Weeks
Absolute Change from Baseline in LDL-C
Time frame: Up to 52 weeks
The Absolute Change from Baseline in Plasma PCSK9 Concentration
Time frame: Up to Week 52
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Sep 2026.
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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.
Stephen Nicholls, MBBS, FRACP, PhD study chair
VHI
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing STX-1150 in people with Elevated LDL-C and High Cholesterol, run by Monash University at 3 sites in 2 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 2 inclusion and 6 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: looking for participants now, as of the sponsor's last update on 21 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in New Zealand, Australia. 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. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Domenic Sacca (+61 423245187, domenic.sacca@monash.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and Severity of Treatment-Emergent Adverse Events (TEAEs) (time frame: Up to Week 52 from administration of STX-1150). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 21 Sep 2026.