The VITAL Amyloidosis Study, a Global Phase 3, Efficacy and Safety Study of NEOD001 in Patients With AL Amyloidosis (VITAL)
|Study Design:||Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled, 2-Arm, Efficacy and Safety Study of NEOD001 Plus Standard of Care Versus Placebo Plus Standard of Care in Subjects With Light Chain (AL) Amyloidosis|
- Time to composite of all-cause mortality or cardiac hospitalization [ Time Frame: From date of randomization until the date of death or cardiac hospitalization, whichever came first, assessed for up to approximately 42 months ]
- NT-proBNP best response [ Time Frame: Baseline to Month 9 ]
- Time to cardiac mortality or cardiac hospitalization [ Time Frame: Baseline to Month 9 ]
- Change in the 6 Minute Walk Test [ Time Frame: Baseline to Month 9 ]
- Change in the Short Form-36 Questionnaire [ Time Frame: Baseline to Month 9 ]
- Change in the Kansas City Cardiomyopathy Questionnaire [ Time Frame: Baseline to Month 9 ]
- Renal best response [ Time Frame: Baseline to Month 9 ]As assessed using Palladini et al, 2014 criteria
- Hepatic best response assessed using Comenzo et al, 2012 criteria [ Time Frame: Baseline to Month 9 ]As assessed using Comenzo et al, 2012 criteria
|Study Start Date:||February 2015|
|Estimated Study Completion Date:||February 2019|
|Estimated Primary Completion Date:||January 2018 (Final data collection date for primary outcome measure)|
24 mg/kg (maximum dose of 2500 mg) of NEOD001 administered once every 28 days.
NEOD001, is a humanized immunoglobulin G1 monoclonal antibody, which specifically targets misfolded light chain aggregates and amyloid deposits. NEOD001 is proposed for use to target the misfolded light chain protein in subjects with AL amyloidosis.
Placebo Comparator: Placebo
Placebo will be administered as a 250 mL bag of normal saline once every 28 days.
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 proteasome inhibitor-containing regimen, with the proteasome inhibitor 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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02312206
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