Clinical trial NCT06561009: Safety and Efficacy of BAFFR CART for Relapsed/ Refractory Neuromyelitis Optica Spectrum Disorder
Official title: Safety and Efficacy of BAFFR CART for Relapsed/ Refractory Neuromyelitis Optica Spectrum Disorder
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE1
PHASE2
Conditions: Neuromyelitis Optica Spectrum Disorder
Lead sponsor: Tianjin Medical University General Hospital
Sponsor class: OTHER
First posted: 2024-08-19
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Incidence of dose-limiting toxicity (DLT)
timeFrame: Up to 28 days
measure: Incidence and severity of adverse events
timeFrame: Up to 28 days
Secondary outcomes:
measure: Proportion of BAFFR CART cells in peripheral blood
timeFrame: Up to 1 years
measure: Annualized relapse rate (ARR)
timeFrame: Up to 1 years
measure: Changes of B cell levels in bone marrow and peripheral blood
timeFrame: Up to 1 years
measure: Changes of AQP4 antibody titers
timeFrame: Up to 1 years
measure: Changes of cytokine in peripheral blood
timeFrame: Up to 1 years
measure: Changes of Expanded Disability Status Scale (EDSS) score
timeFrame: Up to 1 years
measure: Changes of visual acuity
timeFrame: Up to 1 years
measure: Accumulated total active MRI lesions
timeFrame: Up to 1 years
Other outcomes:
Locations:
Study officials:
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