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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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 Chai

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 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. 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.


Sponsor: Prothena Therapeutics Ltd.

Current Primary Outcome: 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 ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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


Original Secondary Outcome: Same as current

Information By: Prothena Therapeutics Ltd.

Dates:
Date Received: December 2, 2014
Date Started: February 2015
Date Completion: August 2018
Last Updated: November 9, 2016
Last Verified: November 2016