Safety and Efficacy of BAFFR CART for Relapsed/ Refractory Neuromyelitis Optica Spectrum Disorder

NCT ID: NCT06561009

Last Updated: 2025-05-30

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-12-01
• Study Completion Date: 2028-10-01

Brief Summary

This is an open-label, single-arm, dose-escalation study in up to 20 participants with relapsed/refractory Neuromyelitis Optica Spectrum Disorders (NMOSD). The aim is to evaluate the safety and efficacy of the treatment with BAFFR CART.

Conditions

Neuromyelitis Optica Spectrum Disorder

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Participant Group

BAFFR CART cells

Group Type: EXPERIMENTAL

Anti-BAFFR CART

Intervention Type: DRUG

Anti-BAFFR CART

Interventions

Anti-BAFFR CART

Anti-BAFFR CART

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Male or female subjects aged 18-60 years;

2. Patients must be diagnosed as AQP4-IgG-positive NMOSD;

3. At least one immunosuppressant has been used for over a year with poorly controlled symptoms;

4. Clinical evidence of at least two relapses in the last 12 months or three relapses in the last 24 months and one relapse in the preceding 12 months before screening.

5. Subjects and their partners must be willing to use effective and reliable methods of contraception, devices or medicines, within one year before BAFFR CART cells infusion.

6. Subjects must provide written informed consent before the study begins and comply with the requirements of the study protocol.

Exclusion Criteria

1. Subjects have received B cell deletion treatment within 6 months before screening;

2. Chronic and active hepatitis B (HBV), hepatitis C (HCV), Human Immunodeficiency Virus (HIV) infection, CMV or syphilis infections concurrently.

3. Subjects with Papovaviruses infection.

4. Subjects have received live attenuated vaccine vaccination within 8 weeks before screening; or plan to receive live vaccine vaccination within 8 weeks after treatment;

5. History of psychoactive drug abuse and failed to withdraw, or have a history of psychiatric disorders.

6. Pregnant or lactating women.

7. Subjects with severe heart, liver, kidney or bone marrow function disorder.

8. Allergic constitution or a history of severe allergies.

9. Subjects with conditions adjudicated by the investigator as unsuitable for lymphodepletion or cell infusion.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 60 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Tianjin Medical University General Hospital

OTHER

Sponsor Role: lead

Responsible Party

Qiang Liu

Department of Neurology

Responsibility Role: PRINCIPAL_INVESTIGATOR

Other Identifiers

IRB2024-YX-220-01

Identifier Type: -

Identifier Source: org_study_id