Can CRISPR-Edited cells stop autoimmune attacks on the brain?

NCT ID NCT07804004

What the study statuses mean

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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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 04, 2026 · Last updated Sep 09, 2026 · Updated 1 time

Summary

This trial tests a therapy called CTX112 in adults with severe neurologic autoimmune diseases, including progressive multiple sclerosis, neuromyelitis optica, and stiff-person syndrome. CTX112 uses donor T cells edited with CRISPR to target and deplete B cells, which are thought to drive these diseases. The study aims to see if the treatment is safe and whether it can reduce disease activity. Participants receive the therapy in increasing doses, and researchers monitor their immune response and any side effects.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
CTX112, an engineered T-cell therapy made from donor cells edited with CRISPR to target and destroy faulty immune cells
What this could lead to
If it works, this could offer a one-time treatment that resets the immune system, potentially stopping or reversing diseases like progressive MS and stiff-person syndrome.
What could go wrong
This is an early-stage trial, so safety and effectiveness are unproven. The therapy may cause severe side effects, including immune reactions or infections, and may not work for all patients.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 220 people

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

Expected to start

Sep 2026

An estimate. Start dates often move.

Expected to finish

Dec 2029

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 70 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.

Inclusion Criteria: 1. Age ≥18 years and \<70 years at screening. 2. Body weight \>40 kg. 3. Participants must voluntarily sign a written informed consent and be willing and able to comply with all trial requirements. Legal authorized representative, additional assent and witness may be used as appropriate. 4. Adequate hematologic, renal, liver, cardiac and pulmonary function. 5. Participants must agree to use acceptable methods of contraception. 6. Willing and able to comply with scheduled visits, treatment plan, laboratory tests, contraceptive guidelines, and other trial procedures. 7. Confirmed diagnosis of PMS, relapsing NMOSD, relapsing MOGAD, refractory AIE, and refractory SPS Exclusion Criteria: 1. Prior treatment with any gene therapy or genetically modified cell therapy. 2. Prior solid organ (e.g., heart, liver, kidney, lung) transplant or hematopoietic cell transplant. 3. Presence of another clinically significant central nervous (CNS) pathology or another condition that in the opinion of the investigator may increase CART cell-related toxicities or confound disease assessments for the main indication. 4. Presence of other active autoimmune disease or other conditions that are likely to pose increased safety risks and/or confound disease assessments, or pose significant risk to those receiving CART cell therapy. 5. Presence or history of certain bacterial, viral or fungal infection. 6. Malignancy in the last 5 years (with the exception of cancers deemed to be low likelihood for recurrence). 7. History or current diagnosis that requires uninterrupted, ongoing anticoagulation. 8. Pregnant or lactating. 9. Presence or history of disease requiring treatment that is not compatible with the study protocol; presence or history of other conditions that are not compatible with the study protocol. 10. History of suicide attempts within 3 years prior to screening. 11. Any contraindications to lumbar puncture or MRI scans

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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

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  1. The official record

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