A Study to Evaluate the Efficacy and Safety of Birtamimab in Mayo Stage IV Patients With AL Amyloidosis
A Phase 3 study to evaluate the efficacy and safety of birtamimab plus standard of care compared to placebo plus standard of care in patients with AL amyloidosis in Mayo Stage IV.
- Light Chain (AL) Amyloidosis
- Eligible Ages
- Over 18 Years
- Eligible Genders
- Accepts Healthy Volunteers
- Aged ≥18 years - Newly diagnosed and AL amyloidosis treatment-naïve with cardiac involvement - Confirmed diagnosis of AL amyloidosis - Confirmed Mayo Stage IV AL Amyloidosis as defined by NT-proBNP ≥1800 pg/mL and Troponin-T >0.03 ng/mL and dFLC ≥18 mg/dL - Planned first-line chemotherapy contains bortezomib administered subcutaneously weekly.
- Non-AL amyloidosis. - NT-proBNP >8500 pg/mL. - Meets the International Myeloma Working Group (IMWG) definition of multiple myeloma - Subject is eligible for and plans to undergo ASCT or organ transplant during the study. - Myocardial infarction, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or ECG evidence of acute ischemia, within 6 months prior to the Month 1-Day 1 Visit. - Severe valvular stenosis (e.g., aortic or mitral stenosis with a valve area <1.0 cm2) or severe congenital heart disease. - ECG evidence of acute ischemia or active conduction system abnormalities - Prior treatment with hematopoietic growth factors, transfusions of blood or blood products within 1 week of Month 1-Day 1. - Prior radiotherapy within 4 weeks of Month 1-Day 1. - Prior treatment with plasma cell-directed chemotherapy, birtamimab, daratumumab, 11- 1F4, anti-serum amyloid P antibody, doxycycline for amyloid, or other investigational treatment directed at amyloid . - Waldenström's macroglobulinemia and/or immunoglobulin M monoclonal gammopathy
- Phase 3
- Study Type
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Birtamimab plus Standard of Care Chemotherapy
Placebo plus Standard of Care Chemotherapy
- Prothena Biosciences Ltd.
Study ContactAlma Araneta
This is a Phase 3 multicenter, global, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of birtamimab in Mayo Stage IV patients with AL amyloidosis. The primary objective is to evaluate the efficacy of birtamimab by assessing time to all-cause mortality. All patients will receive bortezomib-containing chemotherapy regimen as standard of care. Approximately 150 newly diagnosed Mayo Stage IV patients with AL amyloidosis will be enrolled and randomized in a 2:1 ratio to birtamimab or placebo. The minimum planned treatment time for each patient is expected to be at least 9 months or until the patient's death. It is planned that all patients will continue their double-blind treatment until the last patient completes 9 months of treatment or has a mortality event.