Amyotrophic Lateral Sclerosis (ALS)
Conditions
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
Detailed description
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.
Interventions
BIOLOGICALXT-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).
Sponsors
Xalud Therapeutics, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 | Up to Day 180 | Vital signs include temperature, heart rate, respiratory rate and blood pressure |
| Number of participants with clinically significant laboratory abnormalities | Up to Day 180 | Clinical laboratory assessments include hematology, chemistry, coagulation, lipids, C-reactive protein, erythrocyte sedimentation rate and CSF total protein, cell count and glucose |
Countries
United States
Contacts
CONTACTDirector of Medical Information
Outcome results
None listed