A Study to Evaluate the Efficacy and Safety of Birtamimab in Mayo Stage IV Patients With AL Amyloidosis

• Status: Recruiting
• Trial end date: 2024-06-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Prothena Biosciences Ltd.

Criteria

Key Inclusion Criteria:

• 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.

Key Exclusion Criteria:

• 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