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Can a One-Time gene shot calm inflammation in ALS?

NCT ID NCT06704347

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 8 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Mar 2027

An estimate. Start dates often move.

Expected to finish

Jun 2028

An estimate. End dates often move.

Lead sponsor

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

18 to 80 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

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.

  1. The places running it

    4 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. 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

  • Barrow Neurological Institute (St. Joseph's)

    Phoenix, Arizona, 85013, United States

  • Henry Ford Health

    Detroit, Michigan, 48202, United States

  • Johns Hopkins University

    Baltimore, Maryland, 21205, United States

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

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