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

JRCT ID: jRCT2031220037

Registered date:23/04/2022

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Light Chain (AL) Amyloidosis
• Date of first enrollment: 23/01/2023
• Target sample size: 16
• Countries of recruitment: Australia,Japan,Canada,Japan,Czechia,Japan,Denmark,Japan,France,Japan,Germany,Japan,Greece,Japan,Hungary,Japan,Ireland,Japan,Israel,Japan,Italy,Japan,Republic of Korea,Japan,Netherlands,Japan,Poland,Japan,Portugal,Japan,Spain,Japan,Taiwan,Japan,Turkey,Japan,United Kingdom,Japan,United States,Japan
• Study type: Interventional

Intervention(s)

- Birtamimab group:
Intravenous administration of 24 mg/kg birtamimab every 28 days. Bortezomib-containing chemotherapy regimen (e.g. cyclophosphamide, bortezomib, and dexamethasone (CyBorD)) according to the institutional standard of care is co-administered as Standard of Care Chemotherapy
- Placebo group:
Intravenous 0.9% Saline administration as a placebo every 28 days. Bortezomib-containing chemotherapy regimen (e.g. cyclophosphamide, bortezomib, and dexamethasone (CyBorD)) according to the institutional standard of care is co-administered as Standard of Care Chemotherapy

Outcome(s)

• Primary Outcome: Time to all-cause mortality

Secondary Outcome

- Change from baseline to Month 9 in the Physical Component Summary (PCS) score of the SF-36v2
- Change from baseline to Month 9 in the 6MWT distance (meters)

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

- Aged >=18 years and legal age of consent according to local regulations
- Newly diagnosed and AL amyloidosis treatment naive 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 >=0.025 ng/mL or high sensitivity cardiac troponin and dFLC >=18 mg/dL
- Planned first-line chemotherapy contains bortezomib administered subcutaneously weekly.

Exclude criteria

- Non-AL amyloidosis.
- NT-proBNP >8500 pg/mL.
- Meets the International Myeloma Working Group (IMWG) definition of multiple myeloma except for malignancy biomarker of involved /uninvolved serum free light chain ratio >=100
- 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 cm^2) 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 .
- Waldenstrom's macroglobulinemia and/or immunoglobulin M monoclonal gammopathy

Related Information

• Primary Sponsor: Nie Christie
• Secondary ID(s): NCT04973137,2021-000037-14