Exploratory Clinical Study on the Safety and Efficacy of Targeted BCMA Autologous CART Cell Injection in Subjects With Recurrent/Refractory Light Chain Amyloidosis

NCT06983951 | Recruiting

• 1 other identifier: BRYY-IIT-LCYJ-2024-025
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

This study is a multicenter, open label, fixed dose exploratory clinical trial with an expected enrollment of 30 subjects. The main objective is to evaluate the safety of targeted BCMA autologous CART cell injection in the treatment of recurrent or refractory light chain amyloidosis in subjects, preliminarily verify the effectiveness of targeted BCMA autologous CART cell injection in the treatment of recurrent or refractory light chain amyloidosis in subjects, and explore the pharmacokinetic, pharmacodynamic, and immunogenic characteristics of targeted BCMA autologous CART cell injection after reinfusion.

Conditions

Light Chain Amyloidosis

Outcome Measures

Primary Outcomes (2)

• Frequency Of AES, SAES

  0 day to 24 months after treatment

• Dose Limiting Toxicity

  0~28 day after treatment

Study Arms (1)

BCMA CART

EXPERIMENTAL

Genetic: BCMA CART

Interventions

BCMA CART GENETIC

The cell product used in this study is a targeted BCMA autologous CART cell injection. Research drug name: Targeted BCMA Autologous CART Cell Injection Drug code: BJBR-BCMA-006 Administration method: single intravenous injection

BCMA CART

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may not qualify if:

• Participants must personally sign the informed consent form approved by the ethics committee in writing before the start of the study;
• The age of the subject is ≥ 18 years old;
• Diagnosed with light chain amyloidosis through pathological examination;
• Subjects with recurrent/refractory light chain amyloidosis who have previously received second-line or higher treatment;
• dFLC \> 50mg/L
• Expected survival period ≥ 12 weeks;
• ECOG score ≤ 2 points;
• Female subjects with fertility should agree to take effective contraceptive measures from the date of signing the informed consent form until 365 days after reinfusion. Effective contraceptive measures are defined as abstinence or using contraceptive methods with an annual failure rate of less than 1% as specified in the plan;
• Prior to enrollment, participants must have appropriate organ function and meet all of the following test results:
• Absolute neutrophil count ≥ 1.0 × 109/L \[allowed to use granulocyte colony-stimulating factor (G-CSF) support\]; 9.2 Platelet count ≥ 50 × 109/L; 9.3 Hemoglobin ≥ 8 g/dl; 9.4 Bilirubin value ≤ 1.5 x upper limit of normal (ULN); 9.5 ALT or AST ≤ 2.5 times the upper limit of normal (ULN) (with liver involvement ≤ 5 times the upper limit of normal); 9.6 Mayo 2004 Stage I-IIIa participants; 9.7 Stable coagulation function: INR ≤ 1.5, APTT ≤ 1.2 x upper limit of normal (ULN); Basic blood oxygen saturation is greater than 92% in indoor natural air environment.
• Subjects who meet any of the following criteria will be excluded:
• Subjects who have received the following previous treatments:
• Individuals who have received gene therapy prior to enrollment; 1.2 Subjects who received live vaccines within 4 weeks prior to enrollment; 1.3 Received other intervention clinical drug treatments within 12 weeks prior to single collection;
• Patients with central involvement or complete intestinal obstruction;
• Patients with moderate to severe pleural and peritoneal effusion who are difficult to control with conventional treatment and require continuous catheterization and drainage;

Sponsors & Collaborators

• Beijing Boren Hospital: lead

Study Sites (1)

Beijing Gobroad Brond Hospital

Beijing, Beijing Municipality, 100070, China

RECRUITING

MeSH Terms

Conditions

Immunoglobulin Light-chain Amyloidosis

Condition Hierarchy (Ancestors)

Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Amyloidosis; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases; Paraproteinemias

Study Officials

• yajing zhang, MD/PhD

  Beijing Gaobo Boren Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

yajing zhang, MD/PhD

CONTACT

+8601083605002(716); zhangyj3@gobroadhealthcare.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 19, 2025
• First Posted: May 21, 2025
• Study Start: February 20, 2025
• Primary Completion (Estimated): September 30, 2027
• Study Completion (Estimated): September 30, 2027
• Last Updated: May 21, 2025

Record last verified: 2025-02

Locations

CN (1)