Набор скоро начнётся NCT07732400

A Study of VV-14303 for the Treatment of Metabolic Dysfunction-associated Steatohepatitis (MASH)

Фаза I / Фаза II С лечением Metabolic Dysfunction-associated Steatohepatitis (MASH)

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: VV-14303.
Кому может быть актуально: Состояния в реестре: Metabolic Dysfunction-associated Steatohepatitis (MASH). Базовые параметры: 18 лет — 75 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: США, 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 1/2, First-in-Human, Multi-Arm, Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK), and Efficacy of VV-14303, an Adeno-associated Virus Vector-mediated Fibroblast Growth Factor 21 (FGF21) Gene Therapy, in Adults With Metabolic Dysfunction-associated Steatohepatitis (MASH)

Обзор

A Study of VV-14303 for the Treatment of Metabolic dysfunction-associated steatohepatitis (MASH)

Подробное описание

A Phase 1/2, First-in-Human, Multi-Arm, Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of VV-14303, an Adeno-associated Virus Vector-mediated Fibroblast Growth Factor 21 (FGF21) Gene Therapy, in Adults with Metabolic dysfunction associated steatohepatitis (MASH) (the RESTORE Study)

Вмешательства

Генная терапия VV-14303
will be administered via ultrasound guided intramuscular injections

Первичные конечные точки

Incidence and severity of adverse events, abnormal clinical laboratory values, abnormal physical exams, abnormal vital signs, abnormal ECGs, and abnormal imaging [Срок оценки: 52 Weeks]
Number of participants with improvement in overall metabolic health, as assessed by changes in serum biomarker levels [Срок оценки: 6 weeks]
Changes in liver fat content as assessed by Magnetic Resonance Proton Density Fat Fraction (MRI-PDFF) as assessed by FibroScan® [Срок оценки: 26 Weeks]
Change in liver fat content as assessed by controlled attenuation parameter (CAP) as assessed by FibroScan® [Срок оценки: 26 weeks]

Вторичные конечные точки (7)

Efficacy associated with VV-14303 in participants with MASH [Срок оценки: Week 26 and 52]
Efficacy associated with VV-14303 in participants with MASH [Срок оценки: Week 26 and 52]
Efficacy associated with VV-14303 in participants with MASH [Срок оценки: 52 Weeks]
Efficacy associated with VV-14303 in participants with MASH [Срок оценки: 52 Weeks]
Part 1: Efficacy associated with VV-14303 in participants with MASH [Срок оценки: 26 Weeks]
Part 1: Efficacy associated with VV-14303 in participants with MASH [Срок оценки: 26 Weeks]
Concentration of adeno-associated virus (AAV) vector-mediated transgene product in serum [Срок оценки: 52 Weeks]

Критерии участия

Критерии включения

Participant is capable of providing signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol
Must be 18 to 75 years of age (inclusive) at Screening
Body Mass Index (BMI) of 25 to <40 kg/m2 (inclusive)
Male participants must agree to use a highly effective contraception during the Treatment Period and at least 12 months after administration of VV-14303. Female participants must not be a woman of child-bearing potential (WOCBP)
Biopsy-confirmed MASH
Previous history or presence of ≥2 of the following metabolic risk factors: obesity (BMI ≥25 kg/m²), hypertension (blood pressure \[BP\] ≥140/90 mmHg or on antihypertensive medication), dyslipidemia (triglycerides ≥150 mg/dL or high-density lipoprotein cholesterol \[HDL-C\] <40 mg/dL in men/<50 mg/dL in women or on lipid-lowering therapy), type 2 diabetes mellitus
Must be willing to refrain from the donation of blood, plasma, platelets, eggs, or sperm during the 12-month post-treatment follow-up period

Критерии исключения

Presence of alternate and/or additional liver disease etiologies at Screening, including but not limited to chronic viral hepatitis, autoimmune hepatitis
Use of treatments for metabolic syndrome management, including oral antidiabetic drugs (OADs) (e.g., metformin), incretin mimetics (GLP-1 receptor agonists or GLP-1/gastric inhibitory polypeptide \[GIP\] agonists) or other glucose-lowering agents that has not been stable for at least 6 months prior to Screening
Use of Resmetirom that has not been stable for at least 6 months prior to Screening visit
Any medical, cognitive, or psychiatric condition that, in the opinion of the Investigator, could contraindicate the use of the investigational drug, make consistent study assessment and follow-up over the 12-month Post-Treatment Follow-up Period unlikely, or would make the participant an unsafe study candidate.
Type 1 diabetes, or poorly controlled type 2 diabetes (HbA1c > 8.0% at Screening)
History of major trauma to the muscle(s) intended for IM injection meeting any of the following criteria:

Within 6 months prior to Screening, or
At any timepoint prior to Screening with continued neurologic or musculoskeletal symptoms
Prior participation in any systemic experimental treatment or receiving any other systemic investigational treatment including within 6 weeks or 5 half-lives of the active ingredient (whichever is longer) prior to the start of Screening
Any vaccination or planned vaccination 30 days prior to dosing, or planned vaccination 8 weeks post dosing
Previously received AAV or adenoviral therapy or participation in any previous gene therapy trial

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

Здоровые добровольцы: Нет