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

NCT06561009 | Not Yet Recruiting Phase 1 DMC

• 1 other identifier: IRB2024-YX-220-01
• Study Type: interventional
• Target: 20
• Locations: 0 countries
• Sites: N/A

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

Outcome Measures

Primary Outcomes (2)

• Incidence of dose-limiting toxicity (DLT)

  Incidence of dose-limiting toxicity (DLT) will be evaluated within the first 28 days following BAFFR CART cells infusion.

  Up to 28 days

• Incidence and severity of adverse events

  Adverse events will be evaluated following the chemotherapy preparative regimen and infusion of BAFFR CART cells within the first 28 days.

  Up to 28 days

Secondary Outcomes (8)

• Proportion of BAFFR CART cells in peripheral blood

  Up to 1 years

• Annualized relapse rate (ARR)

  Up to 1 years

• Changes of B cell levels in bone marrow and peripheral blood

  Up to 1 years

• Changes of AQP4 antibody titers

  Up to 1 years

• Changes of cytokine in peripheral blood

  Up to 1 years

Study Arms (1)

Participant Group

EXPERIMENTAL

BAFFR CART cells

Drug: Anti-BAFFR CART

Interventions

Anti-BAFFR CART DRUG

Anti-BAFFR CART

Participant Group

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Male or female subjects aged 18-60 years;
• Patients must be diagnosed as AQP4-IgG-positive NMOSD;
• At least one immunosuppressant has been used for over a year with poorly controlled symptoms;
• 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.
• 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.
• Subjects must provide written informed consent before the study begins and comply with the requirements of the study protocol.

You may not qualify if:

• Subjects have received B cell deletion treatment within 6 months before screening;
• Chronic and active hepatitis B (HBV), hepatitis C (HCV), Human Immunodeficiency Virus (HIV) infection, CMV or syphilis infections concurrently.
• Subjects with Papovaviruses infection.
• Subjects have received live attenuated vaccine vaccination within 8 weeks before screening; or plan to receive live vaccine vaccination within 8 weeks after treatment;
• History of psychoactive drug abuse and failed to withdraw, or have a history of psychiatric disorders.
• Pregnant or lactating women.
• Subjects with severe heart, liver, kidney or bone marrow function disorder.
• Allergic constitution or a history of severe allergies.
• Subjects with conditions adjudicated by the investigator as unsuitable for lymphodepletion or cell infusion.

Sponsors & Collaborators

• Tianjin Medical University General Hospital: lead

MeSH Terms

Conditions

Neuromyelitis Optica

Condition Hierarchy (Ancestors)

Myelitis, Transverse; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Demyelinating Diseases; Eye Diseases; Autoimmune Diseases; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Department of Neurology

Study Record Dates

• First Submitted: July 23, 2024
• First Posted: August 19, 2024
• Study Start: December 1, 2025
• Primary Completion (Estimated): October 1, 2027
• Study Completion (Estimated): October 1, 2028
• Last Updated: May 30, 2025

Record last verified: 2025-05