Идёт набор NCT07428473

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

Фаза I С лечением Elevated LDL-C and High Cholesterol

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают: В протоколе указаны: STX-1150.
Кому может быть актуально: Состояния в реестре: Elevated LDL-C and High Cholesterol. Базовые параметры: 18 лет — 70 лет · Все.
Что важно проверить: Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится: Австралия, Новая Зеландия
Следующий шаг: Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Официальное название

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)

Обзор

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.

Подробное описание

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).

Вмешательства

Препарат 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.

Первичные конечные точки

Incidence and Severity of Treatment-Emergent Adverse Events (TEAEs) [Срок оценки: Up to Week 52 from administration of STX-1150]
Incidence and Severity of Serious Adverse Events (SAEs) [Срок оценки: Up to Week 52 from administration of STX-1150]
Incidence and Severity of Adverse Events of Special Interest (AESI) [Срок оценки: Up to Week 52 from administration of STX-1150]

Вторичные конечные точки (7)

Incidence of dose-limiting toxicities (DLTs) [Срок оценки: Within 14 days from administration of STX-1150]
Percent Change from Baseline in Plasma PCSK9 Concentration [Срок оценки: Up to Week 52]
Percent Change from Baseline in LDL-C [Срок оценки: Up to 52 weeks]
Plasma Concentrations of STX-1150 Lipid Components [Срок оценки: Up to 52 weeks]
Number of Participants with Treatment-Induced Immunogenicity [Срок оценки: Up to 52 Weeks]
Absolute Change from Baseline in LDL-C [Срок оценки: Up to 52 weeks]
The Absolute Change from Baseline in Plasma PCSK9 Concentration [Срок оценки: Up to Week 52]

Критерии участия

Критерии включения

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

Критерии исключения

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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет