The VITAL Amyloidosis Study, a Global Phase 3, Efficacy and Safety Study of NEOD001 in Patients With AL Amyloidosis

Phase 3

Terminated

Brief Summary: This is a multi-center, international, randomized, double-blind, placebo-controlled, two-arm efficacy and safety study in subjects newly diagnosed with AL amyloidosis. Subjects will remain on-study until study completion, which will occur when all primary endpoint events (all-cause mortality or cardiac hospitalizations) have been reached.

Detailed Description: This is a multi-center, international, randomized, double-blind, placebo-controlled, two-arm efficacy and safety study in subjects newly diagnosed with AL amyloidosis. Approximately 236 subjects will be enrolled in \~60 centers, with approximately 118 subjects per arm.

This is an event driven trial, therefore subjects will remain on-study until study completion, which will occur when all primary endpoint events (all-cause mortality or cardiac hospitalizations) have been reached. All subjects who discontinue will be followed until the last event is adjudicated. The estimated overall study duration is approximately 42 months, including the enrollment and treatment periods

Study drug will be administered once every 28 days as a 60-120 minute IV infusion.

First-line chemotherapy must be a bortezomib-containing regimen, with bortezomib administered weekly. The number of cycles of first-line chemotherapy that are administered are at the discretion of the Investigator, and subsequent chemotherapy regimens may be prescribed as per standard of care at the Investigator's discretion.

An independent Data Monitoring Committee (DMC) will review data on a regular basis.

Recruitment & Eligibility

Inclusion Criteria

Age ≥ 18 years
Newly diagnosed, AL amyloidosis treatment naïve
Bone marrow consistent with plasma cell dyscrasia
Confirmed diagnosis of AL amyloidosis
Cardiac involvement
Planned first-line chemotherapy contains a proteasome-inhibiting agent administered weekly
Adequate bone marrow reserve, hepatic and renal function

Key

Exclusion Criteria

Non-AL amyloidosis
Meets diagnostic criteria for symptomatic multiple myeloma
Subject is eligible for and plans to undergo ASCT
History of Grade ≥ 3 infusion-associated AEs or hypersensitivity to another monoclonal antibody, or known hypersensitivity to diphenhydramine or acetaminophen

Arm && Interventions

Group	Intervention	Description
NEOD001	NEOD001	24 mg/kg (maximum dose of 2500 mg) of NEOD001 administered once every 28 days.
Placebo	Placebo	Placebo will be administered as a 250 mL bag of normal saline once every 28 days.

Primary Outcome Measures

Name	Time	Method
Time to Composite of All-cause Mortality or Cardiac Hospitalization	Randomization until the date of death or cardiac hospitalization, up to 32 months	Time to all-cause mortality death occurring after the first infusion of study drug or cardiac hospitalization as adjudicated by the CEC occurring at least 91 days after first infusion of study drug through last subject last visit, whichever came first

Secondary Outcome Measures

Name	Time	Method

Locations (78): Mayo Clinic Arizona
🇺🇸
Scottsdale, Arizona, United States
City of Hope
🇺🇸
Duarte, California, United States
Stanford Cancer Institute (SCI)
🇺🇸
Stanford, California, United States
Colorado Blood Cancer Institute
🇺🇸
Denver, Colorado, United States
Mayo Clinic
🇺🇸
Rochester, Minnesota, United States
Sylvester Cancer Center, University of Miami
🇺🇸
Miami, Florida, United States
Winship Cancer Institute of Emory University
🇺🇸
Atlanta, Georgia, United States
Radiant Research Northwestern University
🇺🇸
Chicago, Illinois, United States
University of Chicago Medicine
🇺🇸
Chicago, Illinois, United States
Indiana University Cancer Center
🇺🇸
Indianapolis, Indiana, United States
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Mayo Clinic Arizona
🇺🇸Scottsdale, Arizona, United States