Open-label Phase 1b/2 study with primary objective of this study is to evaluate the safety, tolerability and efficacy of AZD0120 in participants with light chain (AL) amyloidosis.
Confirmed histopathological diagnosis of AL amyloidosis
One or more organs currently or historically impacted by AL amyloidosis according to consensus guidelines
Measurable hematologic disease: dFLC > 20 mg/L or serum M-protein > 5g/L
Relapsed or refractory disease with a need for additional therapy after at least 1 line of anti-plasma cell-directed therapy.
Have any other form of amyloidosis other than AL amyloidosis
Mayo Stage IIIb AL amyloidosis
Oxygen saturation < 95% on room air
Systolic blood pressure <100mmHg
Alexion Pharmaceuticals, Inc.
Lead sponsor