A Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients With AL Amyloidosis

Study Identifier:
CAEL101-203
ClinicalTrials.gov Identifier:
NCT04304144
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Study Complete

Available Documents

ProtocolStatistical Analysis Plan

Study Details

Medical Condition
  • AL Amyloidosis
Study Drug
  • Drug: CAEL-101
  • Drug: SoC: cyclophosphamide, bortezomib, and Dexamethasone (CyBorD)
  • Drug: Daratumumab
Date
Mar 2020 - Nov 2023
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: N/A
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • 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.
Exclusion Criteria
  • 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)
Sex
Female & Male
Age
N/A

Study Details

Medical Condition
  • AL Amyloidosis
Study Drug
  • Drug: CAEL-101
  • Drug: SoC: cyclophosphamide, bortezomib, and Dexamethasone (CyBorD)
  • Drug: Daratumumab
Date
Mar 2020 - Nov 2023
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: N/A years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • 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.
Exclusion Criteria
  • 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)

Protocol 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 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 .

Trial Locations

Location
Status
Location
Research Site
Stanford, California, United States, 94305
Status
N/A
Location
Research Site
Detroit, Michigan, United States, 48201
Status
N/A
Location
Research Site
Cleveland, Ohio, United States, 44195
Status
N/A