Идёт набор NCT07674667

FUNCtion ALS: Aiming to Restore UNC13A Function in People Living With ALS

Фаза I / Фаза II С лечением Amyotrophic Lateral Sclerosis

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: TRCN-1023, Placebo.
Кому может быть актуально: Состояния в реестре: Amyotrophic Lateral Sclerosis. 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: Германия, Нидерланды
Следующий шаг: Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

Не всё понятно в терминах? Прочитайте наш гид для пациентов →

Официальное название

A Randomized, Double-Blind, Placebo-Controlled, Phase 1 / 2 Trial Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of TRCN-1023 Administered by Intrathecal Injections to Adult People Living With Amyotrophic Lateral Sclerosis

Обзор

The FUNCtion Amyotrophic Lateral Sclerosis (ALS) trial is a randomized, double-blind, placebo-controlled Phase 1/2 trial to evaluate the safety and tolerability of TRCN-1023 in adults living with ALS. TRCN-1023 is an investigational medicine given as a single injection into the fluid surrounding the spine (intrathecal injection). The trial will also assess how the body processes the drug and whether it shows early signs of benefit over 24 weeks.

Подробное описание

TRCN-1023 is an investigational medicine designed to restore the function of a protein called UNC13A, which becomes disrupted in most people with Amyotrophic Lateral Sclerosis (ALS) due to a breakdown in how nerve cells process genetic information. This trial is designed to evaluate the safety and tolerability of TRCN-1023 given as a single injection into the fluid surrounding the spine, and to identify the best dose to carry forward into future studies. TRCN-1023 stays in the brain and spinal cord long enough that it may only need to be administered once every 24 weeks. The trial will also assess how the body processes the drug, whether it engages its intended target, and its effects on disease progression, physical function, and quality of life.

Вмешательства

Препарат TRCN-1023
TRCN-1023 administered as an intrathecal injection.
Другое Placebo
Intrathecal injection of artificial cerebrospinal fluid (aCSF).

Первичные конечные точки

Frequency of Adverse Events [Срок оценки: 24 Weeks]

Вторичные конечные точки (5)

Plasma Concentration of TRCN-1023 [Срок оценки: Day 1 (pre-dose, 1, 2, 6, and 24 hours post-dose), Weeks 4, 10, 16, and 24]
Cerebrospinal Fluid (CSF) Concentration of TRCN-1023 [Срок оценки: Day 1 (pre-dose and 24 hours post-dose), Weeks 4, 16, and 24 (up to 4 lumbar punctures post-dose)]
Time to Peak Plasma Concentration (Tmax) [Срок оценки: Day 1 (pre-dose, 1, 2, 6, and 24 hours post-dose), Weeks 4, 10, 16, and 24]
Peak Plasma Concentration (Cmax) of TRCN-1023 [Срок оценки: Day 1 (pre-dose, 1, 2, 6, and 24 hours post-dose), Weeks 4, 10, 16, and 24]
Area Under the Plasma Concentration-Time Curve to Last Measurable Concentration (AUClast) of TRCN-1023 [Срок оценки: Day 1 (pre-dose, 1, 2, 6, and 24 hours post-dose), Weeks 4, 10, 16, and 24]

Критерии участия

Критерии включения

Adults aged 18 to 75 years
Diagnosis of ALS (clinically definite, clinically probable, or clinically probable laboratory supported)
ALS symptoms began within the past 24 months
Able to perform breathing tests: slow vital capacity (SVC) with consistent results, with breathing capacity of at least 60% of the expected value
Able and willing to meet all study requirements, including travel to the study site, brain magnetic resonance imaging (MRI) scans, lumbar punctures, and blood draws
Able and willing to use wearable sensors and complete speech assessments at home
On a stable dose of approved ALS medication for at least 4 weeks prior to screening
Capable of providing informed consent

Критерии исключения

Carries a confirmed SOD1 or FUS gene mutation
Has a tracheostomy or requires continuous assisted ventilation more than 22 hours per day during the preceding 3 months before the first Screening Visit
Has a contraindication to brain MRI (e.g., pacemaker, metal implants)
Has a contraindication to lumbar puncture or spinal injection (e.g., blood clotting disorders, certain blood thinners, signs of increased pressure in the brain)
Has significant abnormal liver, kidney, or blood test results
Is currently enrolled in another clinical trial or has received an investigational treatment within the past 4 weeks
Has previously received gene therapy, stem cell therapy, or another Antisense oligonucleotide (ASO) treatment
Has a clinically significant condition other than ALS that could interfere with study participation

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

Здоровые добровольцы: Нет