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

Not Yet RecruitingPhase 1, Phase 2

Sponsor: CRISPR Therapeutics

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

Interventions: CTX112

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

Eligibility overview

Sex: ALL

Age: 18 Years to 70 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria

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