Набор скоро начнётся NCT07169175

A Phase Ⅰ/Ⅱa Study of SNUG01 in Adult Subjects With ALS

Фаза I / Фаза II С лечением Amyotrophic Lateral Sclerosis (ALS)

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: SNUG01.
Кому может быть актуально: Состояния в реестре: Amyotrophic Lateral Sclerosis (ALS). Базовые параметры: 18 лет — 80 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, 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

A Multicenter, Open-label, Single-arm, Dose Escalation and Expansion, Phase I/IIa Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of SNUG01 in Adult Subjects With Amyotrophic Lateral Sclerosis (ALS)

Обзор

The goal of this clinical trial is to evaluate the safety, tolerability and preliminary efficacy of SNUG01 in in adult subjects with Amyotrophic Lateral Sclerosis (ALS).

Подробное описание

This is a 52-week, multicenter, open-label, single-arm, dose escalation and expansion Phase Ⅰ/Ⅱa study of SNUG01 in adults with ALS. Safety will be the primary focus, with secondary emphasis on immunogenicity, PK and preliminary clinical efficacy of SNUG01.

Вмешательства

Препарат SNUG01
AAV (adeno-associated virus) Gene therapy

Первичные конечные точки

The safety and tolerability of SNUG01 administered by intrathecal. [Срок оценки: up to 1 year after administration]
To recommend the optimal expansion dose of SNUG01 that demonstrates acceptable safety with maximum preliminary efficacy administered by IT. [Срок оценки: up to 1 years]

Вторичные конечные точки (3)

Immunogenicity of SNUG01 [Срок оценки: up to 1 years]
PK of SNUG01 (Biodistribution and Viral Shedding) [Срок оценки: up to 1 years]
Preliminary Clinical Efficacy of SNUG01 [Срок оценки: up to 1 years]

Критерии участия

Key Inclusion Criteria\*\*:

Subjects who are able to provide written informed consent form (ICF).
Subjects who are males or females must be ≥ 18 years and ≤ 80 years of age at the screening visit.
Subjects who have clinically definite ALS, clinically probable ALS, or clinically probable-laboratory supported ALS as specified in the revised version of the El Escorial World Federation of Neurology criteria.
Subjects must have an ALS disease duration (from first symptom onset to the screening visit) ≤ 2 years.
Subjects with a body mass index (BMI) ≥ 19 kg/m2 at the screening visit.
Subjects whose percent-predicted Forced Vital Capacity (%FVC) is ≥ 70% or percent-predicted Slow Vital Capacity (%SVC) is ≥ 60%, adjusted for sex, age, and height at the screening visit.
The ALSFRS-R score ≥ 30 during the screening period, and the three respiratory scores (dyspnea, upright respiration, and respiratory insufficiency) must be full marks.
Key Exclusion Criteria\*\*:

Serum Anti-AAV9 neutralizing antibody titer ≥ 1:100.
Current or previous exposure to gene therapy, stem cell products, and solid organ transplantation.
Subjects who have implanted or are estimated to require a diaphragmatic pacing system during the study period.
Any thromboembolic event, such as deep vein thrombosis, pulmonary arteriovenous embolism, and jugular vein embolism, has occurred within 6 months before the administration.
Suffering from autoimmune diseases or ongoing immune-related therapy, except intranasal, inhalation, ocular, topical, intra-articular corticosteroid therapy or corticosteroid physiological replacement therapy.
Active or chronic uncontrolled infection within 4 weeks before the administration, deemed unacceptable in the discretion of the investigator.
Evidence of human immunodeficiency virus (HIV) and treponema pallidum (TP) infection, as documented by the treatment for HIV or TP, or by HIV or TP antibodies positivity at the screening visit.
Has a positive serum pregnancy test at screening (females of childbearing potential only), a positive urine or serum pregnancy test at baseline (Day -1. females of childbearing potential only), or is nursing.

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

Здоровые добровольцы: Нет