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Recruiting NCT07428473

A Study of STX-1150 in Participants With Elevated Low-Density Lipoprotein Cholesterol (LDL-C)

Phase I Interventional Elevated LDL-C and High Cholesterol

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: STX-1150.
Who it may be relevant to
Registry conditions: Elevated LDL-C and High Cholesterol. Basic parameters: 18 years — 70 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Australia, New Zealand
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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)

Overview

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).

Interventions

  • Drug STX-1150
    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.

Primary outcome measures

  • 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]
Secondary outcome measures (7)
  • 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]

Eligibility criteria

Inclusion criteria

  • 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

Exclusion criteria

  • 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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

Australia · 1 center
  • Monash Health/ Victorian Heart Hospital (VHH) — Clayton
New Zealand · 1 center
  • New Zealand Clinical Research (NZCR) — Christchurch

Identifiers

NCT: NCT07428473 · STX-1150-01

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗