A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled, Efficacy and Safety Study of Birtamimab Plus Standard of Care vs. Placebo Plus Standard of Care in Mayo Stage IV Subjects with Light Chain (AL) Amyloidosis

Cancer Categories

Hematologic (Blood Cancers)

Karmanos Trial ID

2022-021

NCT ID

NCT04973137

Age Group

Adult

Scope

National

Phase

Phase III

Principal Investigator

Jeffrey
Zonder, M.D.
Oncology - Hematology, Oncology - Medical View Profile

Objective:

Primary Objective:

To evaluate the efficacy of birtamimab plus standard of care compared to placebo plus standard of care when administered intravenously in Mayo Stage IV subjects with AL amyloidosis by assessing time to all-cause mortality.

Secondary Objectives:

To evaluate birtamimab plus standard of care compared to placebo plus standard of care on the following:
- Change from baseline to Month 9 in health-related quality of life using the Short Form-36 questionnaire Version 2 (SF-36v2)
- Change from baseline to Month 9 in the 6-Minute Walk Test (6MWT) distance

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Eligibility

Inclusion Criteria:

Aged ≥18 years and legal age of consent according to local regulations
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 ≥0.025 ng/mL or high sensitivity cardiac troponin T≥40ng/L and dFLC ≥18 mg/dL
Planned first-line chemotherapy contains bortezomib administered subcutaneously weekly
Must not have discontinued treatment in Double-blind Phase
WOCBP must have a negative pregnancy test and must agree to use highly effective contraception through 90 days following last study drug administration
Male subjects must be surgically sterile or agree to use highly effective contraception through 90 days following last study drug administration
Ability to understand and willingness to sign an ICF prior to initiating the OLE Phase

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 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
Any medical condition or clinically significant abnormality on physical, neurological, laboratory, vital signs, or ECG examination that precludes treatment with birtamimab or participation in the study, in the medical judgment of the Investigator
Symptomatic orthostatic hypotension that in the medical judgment of the Investigator would interfere with subject's ability to safely receive treatment or complete study assessments
History of Grade ≥3 infusion-related AEs during the Double-blind Phase or hypersensitivity to birtamimab
Unable or unwilling to adhere to the study-specified procedures and restrictions
Planning to use any other investigational treatment during the study

Locations

Karmanos Cancer Institute - Detroit Headquarters

4100 John R
Detroit, MI 48201
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Phone: 800-527-6266

Karmanos Cancer Institute at Weisberg Cancer Center - Farmington Hills

31995 Northwestern Hwy
Farmington Hills, MI 48334
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Phone: 800-527-6266

Applicable Disease Site

Therapies, Drugs, Devices