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

NCT07804004 | Not Yet Recruiting Phase 1 FDA Drug

• 1 other identifier: CRSP-AID-502
• Study Type: interventional
• Target: 220
• Locations: 0 countries
• Sites: N/A

Brief Summary

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

Conditions

Progressive Multiple Sclerosis (PMS); Neuromyelitis Optica; Myelin Oligodendrocyte Glycoprotein Antibody-associated Disease; Autoimmune Encephalitis; Stiff-Person Syndrome

Keywords

CD19; CTX112; Zugocabtagene geleucel; zugo-cel; CAR-T; PMS; Relapsing NMOSD; Relapsing MOGAD; Refractory AIE; Refractory SPS

Outcome Measures

Primary Outcomes (1)

• 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.

  From CTX112 infusion up to 28 days post infusion

Secondary Outcomes (7)

• To assess the pharmacodynamics response to CTX112 in adults with neurological autoimmune disorders

  From CTX112 infusion up to 24 months post-infusion

• To assess the pharmacokinetics (PK) of CTX112 in adults with neurological autoimmune disorders.

  From CTX112 infusion up to 24 months post-infusion.

• To assess the preliminary efficacy of CTX112 in adults with PMS.

  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.

  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

  From CTX112 infusion up to Month 6, Month 12, Month 18, and Month 24

Study Arms (1)

CTX112

EXPERIMENTAL

Administered by IV infusion following lymphodepleting chemotherapy

Biological: CTX112

Interventions

CTX112 BIOLOGICAL

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

CTX112

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• CRISPR Therapeutics: lead

MeSH Terms

Conditions

Multiple Sclerosis, Chronic Progressive; Neuromyelitis Optica; Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease; Autoimmune Diseases of the Nervous System; Stiff-Person Syndrome

Condition Hierarchy (Ancestors)

Multiple Sclerosis; Demyelinating Autoimmune Diseases, CNS; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Myelitis, Transverse; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Eye Diseases; Spinal Cord Diseases; Central Nervous System Diseases; Neuromuscular Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 12, 2026
• First Posted: September 4, 2026
• Study Start: September 15, 2026
• Primary Completion (Estimated): December 30, 2029
• Study Completion (Estimated): December 30, 2029
• Last Updated: September 9, 2026

Record last verified: 2026-09