NCT02312206

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

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

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 260 (actual)
• Sponsor: Prothena Biosciences Ltd. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Primary Systemic (AL) Amyloidosis

Interventions

Type	Name	Description
DRUG	NEOD001	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.
OTHER	Placebo	Placebo

Timeline

• Start date: 2015-02-01
• Primary completion: 2018-05-01
• Completion: 2018-05-01
• First posted: 2014-12-09
• Last updated: 2023-11-14
• Results posted: 2019-05-29

Locations

79 sites across 15 countries: United States, Australia, Austria, Belgium, Canada, Denmark, France, Germany, Greece, Israel, Italy, Netherlands, Poland, Spain, United Kingdom