ClinicalTrials.gov record NCT07804004

A Safety and Efficacy Study Evaluating CTX112 in Subjects With Refractory Neurologic Autoimmune Disease

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 5, 2026

A Phase 1/2 Dose Evaluation Trial of the Safety and Preliminary Efficacy of Anti-CD19 Allogeneic CRISPR-Cas9-Engineered T Cells (CTX112) in Adult Participants With Refractory Neurologic Autoimmune Disease.

About this page. This page shows the study's official ClinicalTrials.gov record ↗, with added help to make it easier to understand. We explain medical terms, highlight key details in plain language, and suggest questions you may want to ask the study team.

The record is periodically copied from ClinicalTrials.gov and may lag behind changes. AI summaries are checked by software, not a clinician.Read moreShow less

The official record is written by the study sponsor and published on ClinicalTrials.gov. It may not always reflect the latest changes. Before making any decisions, please confirm important information with the study team and your own healthcare team.

Where you see "AI summary", AI was used to create a plain-language explanation and software checked it against the source. It has not been reviewed by a clinician, so it may contain mistakes or leave out important details.

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.

Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.

PhasePhase 1/2A study that combines safety and dose-finding (phase 1) with a first look at whether the treatment has an effect (phase 2).

Age18 years old to 70 years old

SexAll

AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 1/2, single-arm, open-label trial evaluates CTX112 in adults with several refractory neurologic autoimmune diseases, including PMS, NMOSD, MOGAD, autoimmune encephalitis, and stiff‑person syndrome. Participants receive lymphodepleting chemotherapy followed by an IV infusion of CTX112 and are followed long term. The study may enroll up to 220 participants and includes follow-up of interventions for up to 5 years.

What it is studying. The study is testing the safety, appropriate dosing, and early signs of effect of CTX112 (allogeneic, CRISPR‑Cas9‑engineered anti‑CD19 CAR‑T cells) in people with listed neurologic autoimmune diseases. It also measures how the cells behave in the blood and changes in B‑cell counts and disease outcomes over time.

What the researchers hope to learn. Scientific goals are to evaluate safety (including dose‑limiting toxicities within 28 days), to assess pharmacodynamics (B‑cell counts) and pharmacokinetics (levels of CTX112 in blood), and to gather preliminary efficacy data for each condition (for example, EDSS changes for PMS, relapse rates for NMOSD and MOGAD, and mRS changes for AIE and SPS) over follow‑up visits up to 24 months or longer as specified.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

This is a single-arm, open-label, multicenter, ascending dose Phase 1/2 trial evaluating the safety and preliminary efficacy of CTX112 in adult participants with neurological autoimmune diseases (AIDs), including Progressive Multiple Sclerosis, relapsing Neuromyelitis Optica Spectrum Disorder, relapsing Myelin Oligodendrocyte Glycoprotein Antiody-Associated Disease, refractory AutoImmune Encephalitis, and refractory Stiff Person Syndrome

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Biological: CTX112

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

Age ≥18 years and <70 years at screening.
Body weight >40 kg.
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.
Adequate hematologic, renal, liver, cardiac and pulmonary function.
Participants must agree to use acceptable methods of contraception.
Willing and able to comply with scheduled visits, treatment plan, laboratory tests, contraceptive guidelines, and other trial procedures.
Confirmed diagnosis of PMS, relapsing NMOSD, relapsing MOGAD, refractory AIE, and refractory SPS

Exclusion criteria

Prior treatment with any gene therapy or genetically modified cell therapy.
Prior solid organ (e.g., heart, liver, kidney, lung) transplant or hematopoietic cell transplant.
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.
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.
Presence or history of certain bacterial, viral or fungal infection.
Malignancy in the last 5 years (with the exception of cancers deemed to be low likelihood for recurrence).
History or current diagnosis that requires uninterrupted, ongoing anticoagulation.
Pregnant or lactating.
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.
History of suicide attempts within 3 years prior to screening.
Any contraindications to lumbar puncture or MRI scans

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Show the detailed description from the registry

This trial will evaluate the safety and preliminary efficacy of CTX112 in adult participants with Progressive Multiple Sclerosis (PMS), relapsing Neuromyelitis Optica Spectrum Disorder (NMOSD), relapsing Myelin Oligodendrocyte Glycoprotein Antiody-Associated Disease (MOGAD), refractory AutoImmune Encephalitis (AIE), and refractory Stiff Person Syndrome (SPS). PMS, NMOSD, MOGAD, AIE, and SPS are neurological AIDs where B cells play a significant role. Compartmentalized central nervous system (CNS) inflammation can also occur, leaving a proportion of patients unresponsive to biologics, including clusters of differentiation 19 and 20 (CD19 and CD20)-monoclonal antibodies (mAbs). Both anti-CD19 and anti-CD20 mAbs penetrate the blood-brain barrier (BBB) poorly, limiting the degree of B-cell depletion attainable within the CNS compartment. No curative treatment exists for these conditions, and despite the best available therapies a substantial proportion of patients continue to experience relapses and/or accruing disability and suffer from low quality of life combined with high morbidity and mortality. Current options focus largely on treating or preventing relapses, typically with peripherally acting agents that require repeated parenteral administration. This underscores an urgent need for novel therapies able to cross the BBB and target the CNS B-cell compartment, thereby potentially eliciting a deep immune reset and durable remission in patients who have failed multiple lines of standard therapy. Preliminary evidence suggests that chimeric antigen receptor (CAR) T cells may offer long-lasting benefits, with extended treatment-free remissions and a manageable safety and tolerability profile.CTX112 is a CD19 directed CAR-T cell immunotherapy comprised of allogeneic T cells prepared for the treatment of refractory autoimmune diseases. The cells are collected from healthy adult volunteer donors and are genetically modified ex vivo using CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats/ CRISPR-associated protein 9) gene editing components (single guide RNA and Cas9 nuclease). As with autologous CD19-targeting CAR T cell therapy, CTX112 has the potential to generate clinical responses in patients with PMS, relapsing NMOSD, relapsing MOGAD, refractory AIE and SPS following a one-time treatment, but with the benefit of immediate availability and without the need for the patient to undergo apheresis for collection of T cells. This trial may enroll up to 220 participants. The total duration of trial intervention for each participant is up to 5 years.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants receive lymphodepleting chemotherapy followed by a single intravenous (IV) infusion of CTX112, a CRISPR‑Cas9–engineered anti‑CD19 T‑cell product. They will have blood tests over time to measure B cells and levels of CTX112 and will undergo clinical assessments, including condition‑specific outcome measures (for example EDSS for PMS, relapse counts for NMOSD/MOGAD, and mRS for AIE and SPS). The registry does not describe the study procedures in detail; its eligibility criteria exclude people who have a contraindication to a lumbar puncture (spinal tap) or an MRI scan. Participants will have safety assessments and other monitoring. Some outcomes are tracked up to 24 months after infusion, and the total duration of trial intervention for each participant can be up to 5 years.

How the study is structured. This is an interventional, single‑group, open‑label Phase 1/2 trial with ascending doses. After lymphodepleting chemotherapy, participants receive a single IV infusion of CTX112 (allogeneic CD19‑directed CAR‑T cells) and are monitored for safety, blood levels of CTX112, B‑cell counts, and clinical outcomes over months to years. There is no masking and no comparison (placebo) group.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
The registry says recruitment has not started. When is it expected to open, and can I register interest now?
No sites are listed on the registry yet. Where will the study take place?
Which of the eligibility criteria would need to be checked with tests before I could join?

More on stiff person syndrome

This trial is also listed on this site under autoimmune encephalitis.

Clinical trials8 trials listed for stiff person syndromeEvery trial the registry lists for stiff person syndrome, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in stiff person syndrome researchLatest monthly update: September 2026. Includes: Tube removal spasms. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start: Sep 15, 2026 (estimated)
Primary completion: Dec 30, 2029 (estimated)
Study completion: Dec 30, 2029 (estimated)
Target enrollment: 220 participants (estimated)
Last updated on registry: Sep 9, 2026
Data verified: Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor: CRISPR Therapeutics

Participating sites

Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins.

Terms used on this page · explanations by MedicalUpdates.ai

single-arm: All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
open-label: No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
ascending dose: Groups of participants receive increasing doses of a treatment, one group after another, to find a dose that can be given safely.
refractory: A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
at screening: The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
written informed consent: The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
assent: A child's or dependent adult's agreement to take part, given alongside the formal consent of a parent or legal representative.
Prior treatment: Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
contraindications: A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
lumbar puncture: A procedure in which a needle is placed between the bones of the lower back to collect spinal fluid or to give a treatment.
MRI: A scan that uses magnets and radio waves, not X-rays, to make detailed pictures of the inside of the body. Some implants and metal objects mean a person cannot have one.
Inclusion Criteria: The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria: The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
standard therapy: Treatment generally considered appropriate for a condition in a particular clinical setting. It can vary by patient, place and over time. In many studies the new treatment is added to, or compared with, standard of care.
chimeric antigen receptor: A treatment in which a person's own immune cells (T cells) are collected, modified in a laboratory to recognise a target, and returned to the body.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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