Study of Eque-cel CAR-T Therapy in Newly Diagnosed Severe AL Amyloidosis
Launched by NANJING IASO BIOTECHNOLOGY CO., LTD. · Jul 3, 2025
Trial Information
Current as of July 23, 2025
Not yet recruiting
Keywords
ClinConnect Summary
This clinical trial is testing a new treatment called Eque-cel for adults who have just been diagnosed with a severe form of AL amyloidosis, a rare disease where abnormal proteins build up in organs like the heart, kidneys, or liver. The treatment uses a special type of therapy called CAR-T, which is designed to help the body’s immune system find and attack the harmful cells causing the disease. The main goals are to see if Eque-cel can help patients achieve a very good response to treatment and to understand how safe it is, including any side effects.
Adults 18 years and older with a recent diagnosis of severe AL amyloidosis and specific heart involvement might be eligible to join. Participants will have their blood cells collected to create a personalized Eque-cel therapy, receive a preparation treatment to get their body ready, and then get a single infusion of this therapy. After that, they will be carefully monitored for about 6 months, with follow-up visits for up to 15 years to track their health and response. This study is not yet recruiting, but it offers hope by testing a promising new approach for people facing this serious condition.
Gender
ALL
Eligibility criteria
- Inclusion Criteria:
- • 1. Subjects aged ≥18 years, regardless of gender.
- • 2. Performance status: Eastern Cooperative Oncology Group (ECOG) performance status score ≤2.
- • 3. 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.
- 4. Presence of measurable hematologic disease at screening, defined as at least one of the following:
- • 1. Difference in free light chains (dFLC) \>4 mg/dL, or
- • 2. Involved free light chain (iFLC) \>4 mg/dL with abnormal kappa/lambda (κ/λ) ratio, or
- • 3. Serum protein electrophoresis (SPEP) M protein \>0.5 g/dL.
- 5. 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:
- • 1. Immunohistochemistry/immunofluorescence,
- • 2. Mass spectrometry,
- • 3. Electron microscopy for characteristic appearance/immunoelectron microscopy.
- • 6. Presence of clonal plasma cells in bone marrow, detectable by light microscopy or flow cytometry.
- • 7. Presence of M protein in blood or urine, with exclusion of multiple myeloma, Waldenström macroglobulinemia, or other lymphoplasmacytic proliferative disorders.
- • 8. Organ involvement: At least one organ affected (kidney, heart, liver, nervous system, gastrointestinal tract, lung, soft tissue) as per consensus guidelines.
- • 9. Expected survival time ≥12 weeks.
- 10. Subjects must have adequate organ function, meeting all of the following laboratory criteria prior to enrollment:
- • 1. Complete blood count: Absolute neutrophil count (ANC) ≥1×10⁹/L; absolute lymphocyte count (ALC) ≥0.3×10⁹/L.
- • 2. Hemoglobin \>60 g/L (no red blood cell \[RBC\] transfusion within 7 days prior to testing; use of recombinant human erythropoietin allowed).
- • 3. Liver function: Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤2.5× upper limit of normal (ULN); total serum bilirubin ≤1.5× ULN.
- • 4. Renal function: Creatinine clearance (CrCl) ≥30 mL/min as calculated by the Cockcroft-Gault formula.
- • 5. Coagulation function: Fibrinogen ≥1.0 g/L; activated partial thromboplastin time (APTT) ≤1.5× ULN; prothrombin time (PT) ≤1.5× ULN.
- • 6. Blood oxygen saturation \>91%.
- • 7. Echocardiogram showing left ventricular ejection fraction (LVEF) ≥40%.
- • 8. Systolic blood pressure (SBP) ≥90 mmHg (oral vasopressor support allowed).
- • 11. Subjects and their spouses agree to use effective contraceptive methods (tools or medications) from the time of signing the informed consent form until one year after CAR-T cell infusion.
- • 12. Subjects voluntarily sign the informed consent form (ICF).
- Exclusion Criteria:
- 1. Subjects who have received any of the following treatments prior to enrollment:
- • 1. Gene therapy before enrollment;
- • 2. Live vaccine within 4 weeks before enrollment;
- • 3. Other interventional clinical trial drugs within 12 weeks before leukapheresis.
- • 2. 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.
- • 3. 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.
- • 4. 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).
- • 5. Receipt of any systemic therapy for AL amyloidosis within 14 days before PBMC collection.
- • 6. 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).
- • 7. Unresolved non-hematologic toxicity from prior therapy to baseline or ≤Grade 1 (per NCI-CTCAE v5.0, excluding alopecia and Grade 2 peripheral neuropathy).
- • 8. Known life-threatening allergic reaction, hypersensitivity, or intolerance to cellular products or their components.
- • 9. Subjects with suspected or confirmed symptoms of central nervous system (CNS) involvement by plasma cell tumors.
- • 10. Bone marrow plasma cells \>30% with clinical symptoms of multiple myeloma accompanied by osteolytic bone lesions.
- • 11. 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).
- • 12. 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.
- • 13. Subjects with hypertension that cannot be controlled by medication.
- • 14. Subjects who have received solid organ transplantation.
- • 15. Subjects with a history of central nervous system disorders (e.g., cerebral aneurysm, epilepsy, stroke, dementia, psychosis) or consciousness impairment.
- • 16. Subjects with unstable systemic diseases as judged by the investigator, including but not limited to severe liver, kidney, or metabolic diseases requiring medical treatment.
- • 17. Diagnosis of malignancy other than multiple myeloma within 5 years before screening, excluding adequately treated cervical carcinoma in situ, basal cell or squamous cell skin cancer, localized prostate cancer post-radical surgery, or ductal carcinoma in situ of the breast post-radical surgery.
- • 18. Participation in another interventional clinical trial within 1 month before signing the ICF.
- • 19. Women who are pregnant, breastfeeding, or planning to become pregnant.
- • 20. Subjects who do not agree to or have not completed the signing of the ICF.
- • 21. Other conditions deemed unsuitable for enrollment by the investigator.
About Nanjing Iaso Biotechnology Co., Ltd.
Nanjing Iaso Biotechnology Co., Ltd. is a leading biopharmaceutical company focused on the research, development, and commercialization of innovative therapeutics and diagnostic solutions. With a commitment to advancing healthcare, Iaso Biotechnology specializes in cutting-edge technologies and methodologies to address unmet medical needs in various therapeutic areas. The company leverages its expertise in biotechnology and a robust pipeline of clinical trials to deliver safe and effective treatments, aiming to enhance patient outcomes and contribute to the global medical community.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Patients applied
Trial Officials
Liu peng, Professor
Study Chair
Fudan University
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported