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
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.
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.
Single group
CTX112
Biological/VaccineCTX112 is a CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components
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.
To assess the pharmacodynamics response to CTX112 in adults with neurological autoimmune disorders
Number of B cells in blood over time.
To assess the pharmacokinetics (PK) of CTX112 in adults with neurological autoimmune disorders.
Levels of CTX112 in blood over time.
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
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
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