Safety Study of XT-150 in Participants With ALS

NCT06704347 | Not Yet Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: XT-150-1-0402
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 4

Brief 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

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Outcome Measures

Primary Outcomes (4)

• Number of participants reporting adverse events (AEs), Serious Adverse Events (SAEs) and AEs of Special Interest (AESIs)

  Up to Day 180

• Number of participants with clinically significant physical examination findings

  Up to Day 180

• Number of participants with clinically significant vital sign abnormalities

  Vital signs include temperature, heart rate, respiratory rate and blood pressure

  Up to Day 180

• Number of participants with clinically significant laboratory abnormalities

  Clinical laboratory assessments include hematology, chemistry, coagulation, lipids, C-reactive protein, erythrocyte sedimentation rate and CSF total protein, cell count and glucose

  Up to Day 180

Study Arms (2)

Cohort 1: 1.5 mg XT-150

EXPERIMENTAL

Participants will be administered 1.5 mg of XT-150 once by intrathecal injection on Day 1. An intrathecal injection is an injection using a thin needle inserted into the spinal canal at the base of your spinal cord performed by your study doctor.

Biological: XT-150

Cohort 2: 4.5 mg XT-150

EXPERIMENTAL

Participants will be administered 1.5 mg of XT-150 once by intrathecal injection on Day 1. An intrathecal injection is an injection using a thin needle inserted into the spinal canal at the base of your spinal cord performed by your study doctor.

Biological: XT-150

Interventions

XT-150 BIOLOGICAL

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

Cohort 1: 1.5 mg XT-150; Cohort 2: 4.5 mg XT-150

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Xalud Therapeutics, Inc.: lead

Study Sites (4)

Barrow Neurological Institute (St. Joseph's)

Phoenix, Arizona, 85013, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21205, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Henry Ford Health

Detroit, Michigan, 48202, United States

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Director of Medical Information

CONTACT

212-301-6673; medical.information@xaludthera.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 20, 2024
• First Posted: November 26, 2024
• Study Start: November 1, 2025
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): March 1, 2027
• Last Updated: April 6, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)