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).
Study 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
Eligibility
Sex
ALL
Min age
18 Years
Max age
80 Years
Healthy volunteers
No
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
Primary outcome measure(s)
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
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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