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Набор скоро начнётся NCT07353398

Early-phase Study of ART002g1 Injection in HeFH: Safety, Tolerability and Preliminary Efficacy

Ранняя фаза I С лечением Heterozygous Familial Hypercholesterolemia

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: ART002g1 Injection.
Кому может быть актуально
Состояния в реестре: Heterozygous Familial Hypercholesterolemia. 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
Китай
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

Early-phase Clinical Study on Safety, Tolerability and Preliminary Efficacy of ART002g1 Injection in the Treatment of Heterozygous Familial Hypercholesterolemia

Обзор

This study is an open-label, single ascending dose (SAD) study designed to evaluate the safety and tolerability of ART002g1 in patients with heterozygous familial hypercholesterolemia (HeFH) who require further reduction in low-density lipoprotein cholesterol (LDL-C). ART002g1 uses base editing technology, which is designed to interfere with the expression of the PCSK9 gene in the liver, thereby reducing the circulating levels of PCSK9 and LDL-C. The primary objectives of this study are to determine the safety and pharmacodynamic (PD) profiles of ART002g1 in this patient population.

Вмешательства

  • Препарат ART002g1 Injection
    Intravenous (IV) infusion

Первичные конечные точки

  • Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) [Срок оценки: As of Week 48 (W48) post-administration of ART002g1 for Injection]
Вторичные конечные точки (8)
  • Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: Tmax [Срок оценки: As of Week 2 (W2) post-administration of ART002g1 for Injection]
  • Pharmacodynamic (PD) Assessments: Serum PCSK9 protein [Срок оценки: As of Week 48 (W48) post-administration of ART002g1 for Injection]
  • Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: Cmax [Срок оценки: As of Week 2 (W2) post-administration of ART002g1 for Injection]
  • Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: AUC [Срок оценки: As of Week 2 (W2) post-administration of ART002g1 for Injection]
  • Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: t½ [Срок оценки: As of Week 2 (W2) post-administration of ART002g1 for Injection]
  • Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: CL [Срок оценки: As of Week 2 (W2) post-administration of ART002g1 for Injection]
  • Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1:Vss [Срок оценки: As of Week 2 (W2) post-administration of ART002g1 for Injection]
  • Pharmacodynamic (PD) Assessments: Serum LDL-C [Срок оценки: As of Week 48 (W48) post-administration of ART002g1 for Injection]

Критерии участия

Subjects must meet all the following criteria to be eligible for enrollment:

  • Male or female, aged 18 to 70 years (inclusive) at the time of signing the Informed Consent Form (ICF);
  • Body weight between 45 and 90 kg (inclusive) at screening;
  • Definite diagnosis of heterozygous familial hypercholesterolemia (HeFH), meeting either of the following two criteria (1) or (2):

（1) HeFH diagnosed to be caused by mutations in the LDLR, APOB, or PCSK9 gene;

(2) Meeting 2 out of the 3 following criteria for adults per the Dutch Lipid Clinical Network (DLCN) criteria:

  • Serum LDL-C ≥ 4.7 mmol/L without prior lipid-lowering treatment;
  • Cutaneous or tendinous xanthomas, or arcus cornealis (in subjects < 45 years old);
  • Presence of FH or early-onset atherosclerotic cardiovascular disease (ASCVD) in first-degree relatives.

Subjects must not be enrolled if they meet any one or more of the following exclusion criteria:

  • Diagnosis of compound heterozygous FH, double heterozygous FH, or homozygous FH (HoFH);
  • Positive for hepatitis B surface antigen (HBsAg), or positive for hepatitis B core antibody (HBcAb) with peripheral blood HBV DNA titer ≥ 1 × 10² copies/L; positive for hepatitis C virus (HCV) antibody with positive peripheral blood HCV RNA; positive for human immunodeficiency virus (HIV) antibody;
  • Any unstable systemic disease, including but not limited to: unstable angina; cerebrovascular accident or transient ischemic attack (within 6 months prior to screening); myocardial infarction (within 6 months prior to screening); history of heart failure (NYHA Class II-IV); severe arrhythmia requiring pharmacotherapy; liver, kidney, or metabolic diseases; or other unstable systemic diseases as determined by the investigator;
  • History of percutaneous transluminal coronary angioplasty (PTCA), percutaneous coronary intervention (PCI), or coronary artery bypass grafting (CABG) within 6 months prior to the first dose; or documented severe coronary artery stenosis as confirmed by coronary CT or coronary angiography within 90 days prior to randomization.

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение
Нерандомизированное
Модель
Последовательный дизайн
Маскирование
Открытое
Основная цель
Лечение

Центры проведения

Китай · 1 центр
  • Shanghai General Hospital — Шанхай

Идентификаторы

NCT: NCT07353398 · IIT-ATMP-2026001

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗