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A Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients With AL Amyloidosis
Trial ID: NCT04304144
AL amyloidosis begins in the bone marrow where abnormal proteins misfold and create free light chains that cannot be broken down. These free light chains bind together to form amyloid fibrils that build up in the extracellular space of organs, affecting the kidneys, heart, liver, spleen, nervous system and digestive tract. The primary purpose of this study is to determine the recommended dose of CAEL-101 to facilitate progression of further clinical trials and evaluate safety and tolerability of CAEL-101 in combination with the standard of care (SoC) cyclophosphamide-bortezomib-dexamethasone (CyBorD) chemotherapy and daratumumab .
CAEL101-203: A Phase 2, Open-label, Multicenter Dose Selection Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients With AL Amyloidosis
Key Inclusion Criteria:
Each patient must meet the following criteria to be enrolled in this study.
1. AL amyloidosis Mayo stage I, II or IIIa
2. For Part A only, measurable hematologic disease defined by at least one of the
1. involved/uninvolved free light chain difference (dFLC) > 5mg/dL or
2. free light chain (FLC) > 5mg/dL with abnormal Kappa/Lambda ratio or
3. serum protein electrophoresis (SPEP) m- spike > 0.5 g/dL Patients with confirmed
AL amyloid diagnosis without measurable disease may be enrolled with consultation
and approval by the Sponsor Medical Monitor or their designee.
3. a. For Part A, currently on and continuing OR planned to start concurrent chemotherapy
with CyBorD administered weekly as SoC. b. For Part B, currently on and continuing OR
planned to start concurrent chemotherapy with CyBorD and daratumumab administered as
Key Exclusion Criteria:
Patients who meet any of the following criteria will not be permitted entry to the study.
1. Any form of secondary, hereditary, senile, localized, dialysis-related or leukocyte
chemotactic factor 2-related (ALECT2) amyloidosis
2. Meets the International Myeloma Working Group (IMWG) definition of multiple myeloma.
Patients with signs and/or symptoms attributable ONLY to amyloidosis and who do NOT
meet IMWG definition of smoldering myeloma may be enrolled upon approval of the
3. Supine systolic blood pressure < 90 mmHg or symptomatic orthostatic hypotension,
defined as a decrease in systolic blood pressure upon standing of > 20 mmHg despite
medical management (e.g., midodrine, fludrocortisones) in the absence of volume
4. Receiving dialysis
5. Myocardial infarction, uncontrolled angina, severe uncontrolled ventricular
arrhythmias, or percutaneous cardiac intervention with recent stent, coronary artery
bypass grafting or major cerebrovascular accident within 6 months prior to screening
6. Left ventricular ejection fraction (LVEF) < 45 percent by echocardiogram or multigated
acquisition scan (MUGA)
drug: SoC: cyclophosphamide, bortezomib, and Dexamethasone (CyBorD)
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