safety study of xt-150 in participants with als

Conditions: Amyotrophic Lateral Sclerosis (ALS)

Summary: 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

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.

Eligibility criteria:
key inclusion criteria: * 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 key exclusion criteria: * 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