Safety Study of XT-150 in Participants With ALS

Официальное название

A Phase 1, Open-Label Study Assessing the Safety, Pharmacokinetics, Pharmacodynamics, and Biomarkers of Single Ascending Dose Regimens of XT-150 for the Treatment of Amyotrophic Lateral Sclerosis (ALS)

Обзор

This is a Phase 1, open-label, multi-center safety study of XT-150 in adult participants with Amyotrophic Lateral Sclerosis (ALS). Participants providing informed consent and meeting all study eligibility criteria will be enrolled in the study and will receive a single injection of XT-150 at the Baseline visit. Follow-up visits will occur over 180 days (6 months) after the injection. 8 participants (4 participants per dose level) will be enrolled sequentially in up to 2 ascending, single dose cohorts: Cohort 1: 1.5 mg XT-150 Cohort 2: 4.5 mg XT-150

Подробное описание

The main questions this study is aiming to answer are:

* What adverse events are reported during this study? An adverse event is any "side effect" that participants have during a study. Adverse events may or may not be caused by XT-150. The study doctors will check participants' ALS and general health throughout the trial. * Does XT-150 have any effects on participants' physical examination findings, vital signs, or laboratory values?

In addition to evaluating the safety of XT-150, this study will also collect samples to provide information on the following:

* The levels of XT-150 in your body * The effect of XT-150 on cytokines including interleukin (IL)-10 * The effect of XT-150 on relevant ALS biomarkers such as Neurofilament Light (NfL)

In addition, ALS functional score, breathing capacity and strength will be collected over the course of the study.

Вмешательства

• Биопрепарат XT-150
  XT-150 is a small piece of plasmid DNA (genetic material) that produces a variant of the anti-inflammatory cytokine, Interleukin 10 (IL-10).

Первичные конечные точки

• Number of participants reporting adverse events (AEs), Serious Adverse Events (SAEs) and AEs of Special Interest (AESIs) [Срок оценки: Up to Day 180]
• Number of participants with clinically significant physical examination findings [Срок оценки: Up to Day 180]
• Number of participants with clinically significant vital sign abnormalities [Срок оценки: Up to Day 180]
• Number of participants with clinically significant laboratory abnormalities [Срок оценки: Up to Day 180]

Критерии участия

Критерии включения

• Adults between 18 and 80 years of age
• Male or female, if of childbearing potential or sexually active, strict contraception required
• Have ALS diagnosed by a doctor (specifically, sporadic or familial ALS diagnosed as clinically probable, lab-supported probable or definite ALS defined by the El Escorial criteria)
• Have had symptoms of ALS (muscle weakness) within 36 months of starting this study
• Have the ability to slowly exhale a volume of air at least 60% of what is expected for the participant's sex, height and age
• Have not received treatment for ALS or are currently on a stable dose of an approved treatment for ALS. Patients currently receiving Tofersen are not eligible.
• Able to receive the study injection intrathecally, determined by the study doctor
• Able to undergo the study procedures and adhere to the study visit schedule at the time of study entry, with an estimated life expectancy of 6 months or greater

Критерии исключения

• Have an implanted shunt to drain cerebrospinal fluid (CSF) or an implanted CNS catheter
• Have an implanted of diaphragm pacing system
• Tracheostomy
• History or current diagnosis of cardiac conditions or ECG abnormalities indicating significant risk of safety for participants in the study
• History or current diagnosis of respiratory conditions such as COPD
• History or current diagnosis of cancer, chemical meningitis, HIV, Hep B, Hep C, uncontrolled diabetes
• Presence of an autoimmune condition (for example, rheumatoid arthritis or lupus) requiring treatment or immunodeficiency
• Clinical or laboratory evidence of hepatic or renal disease/injury.
• Taking any prohibited medications
• Women who are pregnant or nursing
• Use of any investigational drugs or devices within 30 days or 5 half-lives of the study agent (whichever is longer). Exception: Observational, non-interventional clinical studies are allowed in the opinion of the study doctor.
• Any other condition that the study doctor feels could compromise the participant's safety, ability to communicate with the study staff, or the quality of the data

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

США · 4 центра

• Barrow Neurological Institute (St. Joseph's) — Phoenix
• Johns Hopkins University — Baltimore
• Massachusetts General Hospital — Boston
• Henry Ford Health — Detroit

Идентификаторы

NCT: NCT06704347 · XT-150-1-0402

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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