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

Phase 3
Recruiting
Conditions
ight Chain (AL) Amyloidosis
Registration Number
JPRN-jRCT2031220037
Lead Sponsor
ie Christie
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Recruiting
Sex
All
Target Recruitment
16
Inclusion 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.

Exclusion 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

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Time to all-cause mortality
Secondary Outcome Measures
NameTimeMethod
- Change from baseline to Month 9 in the Physical Component Summary (PCS) score of the SF-36v2<br>- Change from baseline to Month 9 in the 6MWT distance (meters)
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