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

October 31, 2023 updated by: Prothena Biosciences Ltd.

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

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

260

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Box Hill, Australia, 3128
        • Eastern Health (Box Hill Hospital)
      • Sydney, Australia, 2145
        • Amyloidosis Clinic Cnr Darcy Road and Bridge St. Westmead
      • Woolloongabba, Australia, 4102
        • The University of Queensland-Princess Alexandra Hospital (PAH)
  • Austria
      • Wien, Austria, A-1090
        • Universitatsklinik fur Innere Medizin 1 Kinische Abteilung fur Onkologie Aligemeines Krankenhaus - Universitatskliniken
  • Belgium
      • Antwerpen, Belgium, 2060
        • ZNA Stuivenberg
      • Brussel, Belgium, 1070
        • ULB Hôpital Erasme
      • Leuven, Belgium, 3000
        • UZ Gasthuisberg
  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T2N 4N2
        • University of Calgary-Southern Alberta Cancer Research Institute
      • Edmonton, Alberta, Canada, T6G 122
        • Cross Cancer Institute
    • Ontario
      • Toronto, Ontario, Canada, M5G 1X6
        • Princess Margaret Cancer Research Centre
    • Quebec
      • Montreal, Quebec, Canada, H3T 1E2
        • Jewish General Hospital
  • Denmark
      • Copenhagen, Denmark
        • Rigshospitalet
  • France
      • Caen Cedex 9, France, 14033
        • Hopital de la Cote de Nacre Avenue
      • Creteil, France, 94010
        • Hôpital Henri Mondor
      • Dijon, France, 21079
        • Chu Dijon - Hopital Du Bocage
      • Limoges, France, 87042
        • Hôpital Dupuytren - CHU Limoges
      • Paris, France, 75013
        • Hôpital Pitié-Salpêtrière
      • Pierre-Benite Cedex, France, 69495
        • Hopitaux Lyon Sud
      • Poitiers, France, 86000
        • Service d'Hermatologie CHU de Poitiers
      • Rennes Cedex 2, France
        • CHU Rennes, Service de Medecine Interne
  • Germany
      • Berlin, Germany
        • Charité-Universitätsmedizin
      • Essen, Germany, 45122
        • Universitätsklinikum Essen
      • Hamburg, Germany, 20246
        • Universitätsklinikum Hamburg-Eppendorf (UKE)
      • Heidelberg, Germany, 69120
        • Universitätsklinikum Heidelberg
  • Greece
      • Athens, Greece, 11528
        • Alexandra General Hospital of Athens
      • Patra, Greece, 26504
        • University Hospital of Patras
  • Israel
      • Jerusalem, Israel, 91120
        • Hadassah University Medical Center
      • Kfar Saba, Israel, 44281
        • Meir Medical Center
      • Ramat Gan, Israel, 52621
        • The Chaim Sheba Medical Center
      • Tel Aviv, Israel, 64239
        • Tel Aviv Sourasky Medical Center
  • Italy
      • Bologna, Italy, 40138
        • Policlinico S.Orsola Malpighi, Unita' Operativa di Ematologia
      • Pavia, Italy, 27100
        • Policlinica San Matteo
      • Rome, Italy, 00189
        • Azienda Ospedaliera Sant'Andrea, U.O.S.
  • Netherlands
      • Groningen, Netherlands, 9713 GZ
        • University Medical Center Groningen
      • Utrecht, Netherlands, 3584 CX
        • Universitair Medisch Centrum Utrecht
  • Poland
      • Warszawa, Poland, 02-106
        • MTZ Clinical Research Sp. z o.o.
  • Spain
      • Badalona, Spain, 08916
        • Hospital Universitari Germans Trias i Pujol
      • Barcelona, Spain, 08036
        • Hospital Clinic De Barcelona
      • Madrid, Spain, 28041
        • Hospital Universitario 12 de Octubre
      • Madrid, Spain, 28222
        • Hospital Universitario Puerta de Hierro - Maiadahonda
    • Nevarra
      • Pamplona, Nevarra, Spain
        • Clinica Universidad de Navarra
  • United Kingdom
      • Birmingham, United Kingdom
        • University Hospitals Birmingham NHS Foundation Trust - Queen Elizabeth Hospital
      • Bristol, United Kingdom
        • University Hospitals Bristol NHS Foundation Trust - Bristol Haematology And Oncology Centre
      • Leicester, United Kingdom
        • Leicester Royal Infirmary
      • London, United Kingdom, NW3 2PF
        • The Royal Free London NHS Foundation Trust-The Royal Free Hospital
      • Norwich, United Kingdom
        • NNUH NHS Foundation Trust - Norfolk and Norwich University Hospital
      • Southampton, United Kingdom
        • Southampton General Hospital
  • United States
    • Arizona
      • Scottsdale, Arizona, United States, 85259
        • Mayo Clinic Arizona
    • California
      • Duarte, California, United States, 91010
        • City of Hope
      • Stanford, California, United States, 94305
        • Stanford Cancer Institute (SCI)
    • Colorado
      • Denver, Colorado, United States, 80218
        • Colorado Blood Cancer Institute
    • Florida
      • Jacksonville, Florida, United States, 32224
        • Mayo Clinic
      • Miami, Florida, United States, 33312
        • Sylvester Cancer Center, University of Miami
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Winship Cancer Institute of Emory University
    • Illinois
      • Chicago, Illinois, United States, 60637
        • University of Chicago Medicine
      • Chicago, Illinois, United States, 60611
        • Radiant Research Northwestern University
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Indiana University Cancer Center
    • Louisiana
      • New Orleans, Louisiana, United States
        • Ochsner Clinic Foundation
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • John Hopkins University School of Medicine
    • Massachusetts
      • Boston, Massachusetts, United States, 02111
        • Tufts Medical Center
      • Boston, Massachusetts, United States, 02118
        • Boston University School of Medicine
    • Michigan
      • Detroit, Michigan, United States, 48201
        • Karmanos Cancer Institute
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Mayo Clinic
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University in Saint Louis
    • New Jersey
      • Newark, New Jersey, United States, 07112
        • Newark Beth Israel Medical Center
    • New York
      • New York, New York, United States, 10065
        • Memorial Sloan-Kettering Cancer Center
      • New York, New York, United States, 10032
        • Columbia University - College of Physicians & Surgeons
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599-1651
        • University of North Carolina Chapel Hill Comprehensive Cancer Center
      • Charlotte, North Carolina, United States, 28211
        • Levine Cancer Institute
      • Durham, North Carolina, United States, 27705
        • Duke University Medical Center
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • The Cleveland Clinic - Main Campus
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health & Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Hospital of the University of Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15232
        • University of Pittsburg - Hillman Cancer Center
    • Tennessee
      • Nashville, Tennessee, United States, 37232-5505
        • Vanderbilt University Medical Center
    • Texas
      • Houston, Texas, United States, 77030
        • University of Texas; MD Anderson Cancer Center
    • Utah
      • Salt Lake City, Utah, United States, 84112
        • Huntsman Cancer Institute
    • Washington
      • Seattle, Washington, United States, 98109
        • University of Washington
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Froedtert & Medical College of Wisconsin, Cancer Center-Froedtert Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

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

Key Exclusion Criteria:

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: NEOD001
24 mg/kg (maximum dose of 2500 mg) of NEOD001 administered once every 28 days.
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.
Placebo Comparator: Placebo
Placebo will be administered as a 250 mL bag of normal saline once every 28 days.
Other: Placebo
Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Time to Composite of All-cause Mortality or Cardiac Hospitalization
Time Frame: 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
Randomization until the date of death or cardiac hospitalization, up to 32 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Prothena Biosciences Ltd.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

February 1, 2015

Primary Completion (Actual)

May 1, 2018

Study Completion (Actual)

May 1, 2018

Study Registration Dates

First Submitted

December 2, 2014

First Submitted That Met QC Criteria

December 5, 2014

First Posted (Estimated)

December 9, 2014

Study Record Updates

Last Update Posted (Estimated)

November 14, 2023

Last Update Submitted That Met QC Criteria

October 31, 2023

Last Verified

October 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NEOD001-CL002

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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