NCT07055724

Study of Eque-cel CAR-T Therapy in Newly Diagnosed Severe AL Amyloidosis

Exploratory Clinical Study of Fully Human BCMA Chimeric Antigen Receptor Autologous T-cell Injection (Equecabtagene Autoleucel, Eque-cel) for the Treatment of Newly Diagnosed Mayo Stage IIIb AL Amyloidosis Patients

• Status: Not Yet Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 17 (estimated)
• Sponsor: Nanjing IASO Biotechnology Co., Ltd. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The goal of this clinical trial is to learn if Equecabtagene Autoleucel(Eque-cel), a Chimeric Antigen Receptor T-cell (CAR-T) therapy, works to treat severe Light Chain (AL) Amyloidosis in newly diagnosed adults with Mayo Stage IIIb. It will also learn about the safety and effects of Eque-cel. The main questions it aims to answer are: Does Eque-cel lead to hematologic remission (achieving a very good partial response or better) in AL amyloidosis? How safe is Eque-cel for these patients, and what side effects might occur? Participants will: Undergo blood cell collection to create personalized Eque-cel therapy. Receive pre-treatment to prepare their body for the therapy (lymphodepletion). Receive a single infusion of Eque-cel. Be monitored closely for 24 weeks after infusion, followed by long-term checkups for up to 15 years.

Detailed description

This exploratory clinical trial investigates the efficacy and safety of Eque-cel , a fully human B-cell Maturation Antigen (BCMA)-targeted CAR-T cell therapy, in treating newly diagnosed Mayo Stage IIIb AL amyloidosis, a rare and severe systemic disorder characterized by abnormal plasma cell proliferation and amyloid fibril deposition leading to organ dysfunction. Current treatments, such as high-dose chemotherapy with stem cell transplantation or daratumumab-based regimens, fail to meet the urgent needs of Stage IIIb patients due to severe cardiac involvement, poor tolerability, and lack of approved standard therapies, necessitating rapid, low-cardiotoxicity interventions. Eque-cel, approved for relapsed/refractory multiple myeloma in China, has shown promise in prior studies with high hematologic response rates and rapid action, offering a potential solution for this high-risk population. This study is an interventional clinical trial focused on treating newly diagnosed Mayo Stage IIIb AL Amyloidosis patients. It is a Phase II exploratory study aimed at initially validating the efficacy and safety of Eque-cel. The study employs a single-arm, open-label design, with all participants receiving Eque-cel CAR-T cell therapy (at a dose of 1.0×10⁶ CAR-T cells/kg), without randomization or blinding, to directly assess treatment outcomes. The trial is conducted at a single center (Zhongshan Hospital, Fudan University) and plans to enroll up to 17 participants. The process includes peripheral blood mononuclear cell (PBMC) collection, optional bridging therapy, lymphodepletion preconditioning, and cell infusion, followed by a 24-week primary follow-up period and long-term monitoring for up to 15 years. The study aims to complete patient enrollment within 2 years. The primary endpoint is to evaluate the hematologic response rate post-treatment, while secondary endpoints include safety and survival-related metrics. Through innovative CAR-T therapy, this study seeks to address unmet clinical needs and provide new treatment hope for this high-risk population.

Conditions

• AL Amyloidosis

Interventions

Type	Name	Description
BIOLOGICAL	Eque-cel CAR-T Therapy	Participants in this arm receive a single infusion of Eque-cel at a dose of 1.0 x 10\^6 CAR-T cells/kg following lymphodepletion preconditioning with fludarabine and cyclophosphamide. Eque-cel is a fully human BCMA-targeted CAR-T cell therapy designed to recognize and eliminate BCMA-expressing cells.

Timeline

• Start date: 2025-07-01
• Primary completion: 2027-06-01
• Completion: 2027-06-01
• First posted: 2025-07-09
• Last updated: 2025-07-09