Universal CAR-T Cells in Patients with Refractory Autoimmune Diseases of the Nervous System.

Capital Medical University

Status and phase: Not yet enrolling; Early Phase 1

Conditions: Multiple Sclerosis; Myasthenia Gravis, Generalized; Chronic Inflammatory Demyelinating Polyradiculoneuropathy; Neuromyelitis Optica Spectrum Disorders

Treatments: Drug: Universal BCMA CAR-T; Drug: Universal BCMA CAR-T; Universal CD19 CAR-T; Drug: Universal CD19 CAR-T

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT06485232

2024-BRL-302-02

Details and patient eligibility

About

This is an open label, single-site, dose-escalation study in up to 25 participants with refractory autoimmune diseases of nervous system. This study aims to evaluate the safety and efficacy of the treatment with universal BCMA and CD19 CART.

Enrollment: 25 estimated patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Aged 18-75 years (for MS patients, 18-55 years); both genders eligible.
• Subjects with refractory neurological autoimmune diseases who have failed standard treatment or lack effective treatment, Including neuromyelitis optica spectrum disorders(NMOSD), generalized myasthenia gravis(gMG), chronic inflammatory demyelinating Polyradiculoneuropathy(CIDP) and multiple sclerosis(MS).
• Anticipated survival of ≥ 12 weeks as judged by the researcher.
• Agrees to use double barrier methods, condoms, oral or injectable contraceptives, or intrauterine devices during the study period and for one year after taking the study medication.
• Provides written informed consent.

Exclusion criteria

• History of solid organ transplantation.
• Malignant tumor within the last two years.
• Positive for Hepatitis B surface antigen (HBsAg) or Hepatitis B core antibody (HBcAb), with peripheral blood Hepatitis B virus (HBV) DNA detected as positive; positive for Hepatitis C virus antibodies, with peripheral blood Hepatitis C virus RNA detected as positive; positive for Human Immunodeficiency Virus (HIV) antibodies; positive for Cytomegalovirus (CMV) DNA; positive for syphilis.
• Primary immunodeficiency (congenital or acquired).
• Severe cardiac disease.
• History of psychiatric disorders or history of psychotropic drug abuse, with no history of withdrawal.
• Allergic constitution or a history of severe allergies.
• Pregnant or breastfeeding women.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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25 participants in 3 patient groups

BCMA CAR-T Group

Experimental group

Description:

Universal BCMA CAR-T

Treatment:

Drug: Universal BCMA CAR-T

CD19 CAR-T Group

Experimental group

Description:

Universal CD19 CAR-T

Treatment:

Drug: Universal CD19 CAR-T

BCMA CAR-T + CD19 CAR-T Group

Experimental group

Description:

Universal BCMA CAR-T; Universal CD19 CAR-T

Treatment:

Drug: Universal BCMA CAR-T; Universal CD19 CAR-T