Study of Eque-cel CAR-T Therapy in Newly Diagnosed Severe AL Amyloidosis
ConditionAL Amyloidosis
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorNanjing IASO Biotechnology Co., Ltd.
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Difference in free light chains (dFLC) >4 mg/dL, or
Disqualifiers
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.
Trial design
Design model
Single group
Treatments tested in this trial
Eque-cel CAR-T Therapy
Biological/VaccineParticipants 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.
Treatment groups
17 Participants
are divided into 1 treatment groupGroup A: Eque-cel ArmExperimental treatment 1 intervention
Trial outcomes
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.
Time frame
From Eque-cel infusion to Day 90 post-infusionSecondary outcomes
1
Complete Hematologic Response Rate (CHR)
The proportion and time to achieving complete hematologic response as defined by the international consensus criteria for amyloidosis.
Time frame
From Eque-cel infusion to 24 months2
Major Organ Deterioration-Progression-Free Survival (MOD-PFS)
This composite endpoint is defined as the time from randomization to the first occurrence of any of the following events (whichever comes first)： 1. Death; 2. Clinical manifestation of heart failure: defined as dyspnea at rest (for at least 3 consecutive days) solely due to amyloid cardiomyopathy, or the need for heart transplantation, left ventricular assist device (LVAD), or intra-aortic balloon pump (IABP); 3. Clinical manifestation of renal failure: defined as progression to end-stage renal disease (requiring hemodialysis or kidney transplantation); hematologic progression as per consensus criteria: from CHR, any detectable monoclonal protein or abnormal free light chain ratio (ratio must double); from PR, a 50% increase in serum M protein to \>0.5 g/dL or a 50% increase in urine M protein to \>200 mg/day (measurable peak required); a 50% increase in free light chains to \>100 mg/L.
Time frame
From the time of Eque-cel infusion up to 24 months3
Progression-Free Survival (PFS)
Defined as the time from screening/enrollment to the first documentation of hematologic disease progression, organ (cardiac, renal, or hepatic) progression, or death from any cause (whichever occurs first), based on laboratory results and determined by international consensus criteria.
Time frame
From the time of Eque-cel infusion up to 24 months4
Organ Response Rate (OrRR)
The proportion of patients with baseline organ involvement achieving a response in each respective organ within 24 months.
Time frame
From the time of Eque-cel infusion up to 24 monthsLocations
Sponsors and contacts
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Source ClinicalTrials.gov
This trial is not recruiting at the moment. You can still explore other options: