A Phase 1/2 interventional study of CTX112 in Progressive Multiple Sclerosis (PMS), Neuromyelitis Optica and Myelin Oligodendrocyte Glycoprotein Antibody-associated Disease, sponsored by CRISPR Therapeutics. Not yet recruiting. Open to participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2026-10-22.
Sponsored by CRISPR Therapeutics · Phase 1/2, Interventional, and Treatment
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
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.
244 studies on the registry are indexed under Multiple Sclerosis, Chronic Progressive; 53 are open to participants now.
This study's planned enrollment of 220 is above the median of 54 across 189 interventional studies indexed under Multiple Sclerosis, Chronic Progressive.
Browse Multiple Sclerosis, Chronic Progressive studies →CRISPR Therapeutics is the lead sponsor of 4 studies on the registry; 3 are open to participants now.
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Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Relapsed and refractory
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Administered by IV infusion following lymphodepleting chemotherapy
Biological: CTX112
CTX112 is a CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components
Also known as: Zugocabtagene geleucel, zugo-cel
To evaluate the safety of CTX112 in adult participants with neurological autoimmue disorders, including PMS, relapsing NMOSD, relapsing MOGAD, refractory AIE, and refractory SPS.
Incidence of dose-limiting toxicities.
Time frame: From CTX112 infusion up to 28 days post infusion
To assess the pharmacodynamics response to CTX112 in adults with neurological autoimmune disorders
Number of B cells in blood over time.
Time frame: From CTX112 infusion up to 24 months post-infusion
To assess the pharmacokinetics (PK) of CTX112 in adults with neurological autoimmune disorders.
Levels of CTX112 in blood over time.
Time frame: From CTX112 infusion up to 24 months post-infusion.
To assess the preliminary efficacy of CTX112 in adults with PMS.
For participants with PMS, outcomes include but are not limited to, change from baseline in Expanded Disability Status Scale (EDSS) score
Time frame: From CTX112 infusion up to Month 6, Month 12, Month 18, and Month 24
To assess the preliminary efficacy of CTX112 in adults with NMOSD.
For participants with NMOSD, outcomes include but are not limited to, annualized rate of relapses
Time frame: From CTX112 infusion up to Month 6, Month 12, Month 18, and Month 24
To assess the preliminary efficacy of CTX112 in adults with MOGAD
For participants with MOGAD, outcomes include but are not limited to annualized rate of relapses
Time frame: From CTX112 infusion up to Month 6, Month 12, Month 18, and Month 24
To assess the preliminary efficacy of CTX112 in adults with AIE
For participants with AIE, outcomes include but are not limited to, change from baseline in the Modified Rankin Score (mRS) score
Time frame: From CTX112 infusion up to Month 6, Month 12, Month 18, and Month 24
To assess the preliminary efficacy of CTX112 in adults with SPS
For participants with SPS, outcomes include but are not limited to, change from baseline in Modified Rankin Score(mRS) score
Time frame: From CTX112 infusion up to Month 6, Month 12, Month 18, and Month 24
No study locations are listed for this record.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is not yet recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Allogeneic CRISPR-Cas9 Engineered Anti-CD19 CAR T-cells CTX112
Multiple Sclerosis, Chronic Progressive →
CRISPR Therapeutics