The Safety and Efficacy of Universal CAR-T Cells Targeting BCMA in the Treatment of Refractory NMOSD

Phase 1

Not yet recruiting

• Conditions: Autoimmune Disease
• Interventions: Drug: Universal BCMA-CD19 CART

• Registration Number: NCT06633042
• Lead Sponsor: Bioray Laboratories

Brief Summary

This is an open label, Multi-center，dose-escalation study in up to 18 participants with refractory NMOSD. This study aims to evaluate the safety and efficacy of universal CAR-T Cells targeting BCMA in the Treatment of refractory NMOSD.

Detailed Description

This is a multi-center, single-arm, open-label clinical study, and the sample size is set to 12-18 subjects. Based on the "3 + 3" dose escalation design principle, subjects will be divided into 3 groups from low dose to high dose in sequence (Group A; Group B; Group C).

Study Timeline

• Status Verified: 2024-09
• Study First Submitted: 2024-09-25
• Study First Submitted QC: 2024-10-07
• Study First Posted: 2024-10-09
• Last Update Submitted: 2024-11-07
• Last Update Posted: 2024-11-11
• Study Start: 2024-11-25
• Primary Completion: 2025-10-12
• Study Completion: 2025-12-12

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 18

Inclusion Criteria

• Aged 18-65 years ; both genders eligible.
• Meets the criteria for Refractory NMOSD.
• 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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
BCMA CART Group	Universal BCMA-CD19 CART	1.0-4.0×10\^6 CAR- Tcells/kg

Primary Outcome Measures

Name	Time	Method
DLT	Within28 Days After BRL-302 Infusion	The number and severity of dose-limiting toxicity (DLT) events
AEs	Up to 12 Months After BRL-302 Infusion	The total number, incidence, and severity of AEs

Secondary Outcome Measures

Name	Time	Method
Annualized relapse rate (ARR)	1,3,6,12 month after BRL-302 infusion	Number of NMOSD relapses in subjects after cell infusion divided by observation time (years)