Can a One-Time gene shot calm inflammation in ALS?
NCT ID NCT06704347
First seen Jul 29, 2026 · Last updated Jul 30, 2026 · Updated 1 time
Summary
This early-stage trial is testing whether a single injection of XT-150, a gene therapy that produces an anti-inflammatory protein, is safe for people with ALS. Eight adults with ALS will receive one of two doses and be followed for six months. The goal is to check for side effects and see how the body processes the therapy, not yet to measure whether it slows the disease.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- a single injection of XT-150, a piece of DNA that produces an anti-inflammatory protein called interleukin-10
- What this could lead to
- If safe, this could pave the way for a new approach to slow nerve damage in ALS by reducing inflammation in the nervous system.
- What could go wrong
- This is a very early, small safety study with only 8 participants, so it cannot prove whether the treatment works. Gene therapies can also cause unexpected immune reactions.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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About 8 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Mar 2027
An estimate. Start dates often move.
- Expected to finish
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Jun 2028
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 80 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
4 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Barrow Neurological Institute (St. Joseph's)
Phoenix, Arizona, 85013, United States
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Henry Ford Health
Detroit, Michigan, 48202, United States
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Johns Hopkins University
Baltimore, Maryland, 21205, United States
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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