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

NCT07055724 | Not Yet Recruiting Phase 2

• 1 other identifier: CT103ACI003
• Study Type: interventional
• Target: 17
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

AL Amyloidosis

Keywords

Eque-cel; CAR-T; Mayo Stage IIIb

Outcome Measures

Primary Outcomes (1)

• Proportion of Patients Achieving Hematologic VGPR or Better by Day 90

  This measure assesses the effectiveness of Eque-cel CAR-T therapy by calculating the percentage of patients who achieve a Very Good Partial Response (VGPR) or better within 90 days post-infusion. Hematologic response indicates a significant reduction in AL amyloidosis blood markers, like abnormal light chains, based on international consensus guidelines. Blood samples are tested at Day 90 using lab methods such as serum free light chain (FLC) and protein electrophoresis (SPEP) to confirm VGPR, reflecting disease control in Mayo Stage IIIb patients.

  From Eque-cel infusion to Day 90 post-infusion

Secondary Outcomes (5)

• Complete Hematologic Response Rate (CHR)

  From Eque-cel infusion to 24 months

• Major Organ Deterioration-Progression-Free Survival (MOD-PFS)

  From the time of Eque-cel infusion up to 24 months

• Progression-Free Survival (PFS)

  From the time of Eque-cel infusion up to 24 months

• Organ Response Rate (OrRR)

  From the time of Eque-cel infusion up to 24 months

• Overall Survival (OS)

  From the time of Eque-cel infusion up to 24 months

Study Arms (1)

Eque-cel Arm

EXPERIMENTAL

Eque-cel : a dose of 1.0 x 10\^6 CAR-T cells/kg

Biological: Eque-cel CAR-T Therapy

Interventions

Eque-cel CAR-T Therapy BIOLOGICAL

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.

Eque-cel Arm

Eligibility Criteria

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

You may qualify if:

• Subjects aged ≥18 years, regardless of gender.
• Performance status: Eastern Cooperative Oncology Group (ECOG) performance status score ≤2.
• Clinically diagnosed with newly diagnosed AL (light chain) amyloidosis, classified as Mayo Stage IIIb per the revised 2004 Mayo Clinic staging system at screening, with serum N-terminal pro-brain natriuretic peptide (NT-proBNP) \>8500 ng/L and serum cardiac troponin T (cTnT) \>0.035 μg/L or cardiac troponin I (cTnI) \>0.01 g/L.
• Presence of measurable hematologic disease at screening, defined as at least one of the following:
• Difference in free light chains (dFLC) \>4 mg/dL, or
• Involved free light chain (iFLC) \>4 mg/dL with abnormal kappa/lambda (κ/λ) ratio, or
• Serum protein electrophoresis (SPEP) M protein \>0.5 g/dL.
• Histopathological diagnosis of amyloidosis based on polarized light microscopy of Congo red-stained tissue specimens showing green birefringence, with confirmation of AL-derived amyloid deposits by at least one of the following methods:
• Immunohistochemistry/immunofluorescence,
• Mass spectrometry,
• Electron microscopy for characteristic appearance/immunoelectron microscopy.
• Presence of clonal plasma cells in bone marrow, detectable by light microscopy or flow cytometry.
• Organ involvement: At least one organ affected (kidney, heart, liver, nervous system, gastrointestinal tract, lung, soft tissue) as per consensus guidelines.
• Expected survival time ≥12 weeks.
• Subjects must have adequate organ function, meeting all of the following laboratory criteria prior to enrollment:

You may not qualify if:

• Subjects who have received any of the following treatments prior to enrollment:
• Gene therapy before enrollment;
• Live vaccine within 4 weeks before enrollment;
• Other interventional clinical trial drugs within 12 weeks before leukapheresis.
• Positive for hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) with detectable hepatitis B virus (HBV) DNA in peripheral blood; positive for hepatitis C virus (HCV) antibody with detectable HCV RNA in peripheral blood; positive for human immunodeficiency virus (HIV) antibody; positive for cytomegalovirus (CMV) DNA; positive for syphilis testing.
• Use of therapeutic doses of corticosteroids (defined as prednisone or equivalent \>20 mg/day) within 7 days before screening, though physiological replacement, topical, and inhaled steroids are permitted.
• Presence of uncontrolled active infection within 7 days before peripheral blood mononuclear cell (PBMC) collection (excluding genitourinary system infections and upper respiratory tract infections \<CTCAE Grade 2).
• Receipt of any systemic therapy for AL amyloidosis within 14 days before PBMC collection.
• Undergoing major surgery within 14 days before PBMC collection, or planning surgery within 2 weeks after study treatment (subjects planning local anesthesia surgery are eligible to participate).
• Unresolved non-hematologic toxicity from prior therapy to baseline or ≤Grade 1 (per NCI-CTCAE v5.0, excluding alopecia and Grade 2 peripheral neuropathy).
• Known life-threatening allergic reaction, hypersensitivity, or intolerance to cellular products or their components.
• Subjects with suspected or confirmed symptoms of central nervous system (CNS) involvement by plasma cell tumors.
• Bone marrow plasma cells \>30% with clinical symptoms of multiple myeloma accompanied by osteolytic bone lesions.
• Severe active cardiovascular disease with significant clinical symptoms despite treatment, including but not limited to unstable angina, myocardial infarction, and severe arrhythmia (excluding those with implanted implantable cardioverter-defibrillator \[ICD\] or pacemaker).
• Subjects with bleeding or severe thrombosis symptoms as judged by the investigator, or with genetic/acquired bleeding and severe thrombosis conditions (including hemophilia, coagulopathy, thrombocytopenia, splenomegaly), or currently receiving thrombolytic or anticoagulant therapy.

Sponsors & Collaborators

• Nanjing IASO Biotechnology Co., Ltd.: lead

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

• Liu peng, Professor

  Fudan University

  STUDY CHAIR

Central Study Contacts

Liu Peng

CONTACT

86-021-64041990; liu.peng@zs-hospital.sh.cn

Li Jing

CONTACT

86-021-64041990

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 26, 2025
• First Posted: July 9, 2025
• Study Start: July 1, 2025
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): June 1, 2027
• Last Updated: July 9, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share