Status:
COMPLETED
A Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients With AL Amyloidosis
Lead Sponsor:
Alexion Pharmaceuticals, Inc.
Conditions:
AL Amyloidosis
Eligibility:
All Genders
Phase:
PHASE2
Brief Summary
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 bui...
Detailed Description
This is a multicenter, open-label, sequential cohort, dose-selection study of CAEL-101 in Mayo Stage I, Stage II and Stage IIIa AL amyloidosis patients. CAEL-101 will be administered in combination wi...
Eligibility Criteria
Inclusion
- Key
- Each patient must meet the following criteria to be enrolled in this study.
- AL amyloidosis Mayo stage I, II or IIIa
- For Part A only, measurable hematologic disease defined by at least one of the following:
- involved/uninvolved free light chain difference (dFLC) \> 5mg/dL or
- free light chain (FLC) \> 5mg/dL with abnormal Kappa/Lambda ratio or
- 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.
- 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 SoC.
- Key
Exclusion
- Patients who meet any of the following criteria will not be permitted entry to the study.
- Any form of secondary, hereditary, senile, localized, dialysis-related or leukocyte chemotactic factor 2-related (ALECT2) amyloidosis
- 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 medical monitor.
- 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 depletion
- Receiving dialysis
- 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
- Left ventricular ejection fraction (LVEF) \< 45 percent by echocardiogram or multigated acquisition scan (MUGA)
Key Trial Info
Start Date :
March 18 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 14 2023
Estimated Enrollment :
25 Patients enrolled
Trial Details
Trial ID
NCT04304144
Start Date
March 18 2020
End Date
November 14 2023
Last Update
March 5 2025
Active Locations (3)
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1
Research Site
Stanford, California, United States, 94305
2
Research Site
Detroit, Michigan, United States, 48201
3
Research Site
Cleveland, Ohio, United States, 44195