Testing a new gene therapy injection for early-stage ALS

Plain-English translation of NCT07287397 on ClinicalTrials.gov ↗ · Source last updated September 2026 · Translation generated September 2026 · How we translate trials

Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.

What is this trial?

This trial is testing a new gene therapy called VTx-002, which is given as a single injection directly into the fluid around the spinal cord. The study involves 12 people with early-stage ALS and will carefully monitor whether the medication is safe and how well it is tolerated over 5 years. Researchers will track participants closely during the first year and then continue follow-up visits every 6 months for the remaining time.

ALS is a serious disease that affects nerve cells and causes progressive muscle weakness, and there are currently very few treatment options. This trial is exploring whether this new gene therapy approach might help slow or modify the disease in people who have been diagnosed recently.

Do you qualify?

You likely qualify if…

you have been diagnosed with ALS within the past 18 months
you are at least 18 years old
your lung function (measured by a breathing test) is at least 80% of normal
you do not have ALS caused by specific gene mutations (FUS or SOD1)
you have a reliable caregiver or partner who can support you during the study
you are willing to visit the study center and complete all required visits and tests over 5 years

You likely don't qualify if…

you have other serious brain or nervous system diseases besides ALS
you already have a breathing tube or feeding tube in place
you have serious health conditions that would make corticosteroid medication unsafe for you (such as uncontrolled high blood pressure, severe osteoporosis, or uncontrolled diabetes)
you have had other serious health problems in the past 6 months, such as heart disease, uncontrolled diabetes, liver or kidney disease, or cancer
you are taking blood thinning medications and cannot safely stop them before the injection procedure

What participation looks like

If you join this study, you will receive a single injection of the medication delivered directly into the fluid around your spinal cord under general anesthesia. During year 1, you will have 12 visits to the study center (including at least one overnight stay after the injection) plus 4 remote visits by phone or video. From years 2 through 5, you will have visits every 6 months, either in person or by phone/video. Overall, you will complete up to 20 study visits over the 5-year period, which will include breathing tests, neurological exams, and blood work to monitor your safety and how you respond to the treatment.

AI-generated summary from trial data · Sep 3, 2026 · Not medical advice

Trial locations(11 sites)

United States

St Joseph's Hospital and medical Center - Barrow Neurological Institute, Phoenix, Arizona

University of California San Diego Medical Center, San Diego, California

Mayo Clinic in Florida, Jacksonville, Florida

University of Miami School of Science, Miami, Florida

Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, Boston, Massachusetts