The VITAL Amyloidosis Study, a Global Phase 3, Efficacy and Safety Study of NEOD001 in Patients With AL Amyloidosis
- Conditions
- Primary Systemic (AL) Amyloidosis
- Interventions
- Other: Placebo
- Registration Number
- NCT02312206
- Lead Sponsor
- Prothena Biosciences Ltd.
- 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
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 260
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- 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
Trial Locations
- Locations (78)
CHU Dijon - Hopital du Bocage
🇫🇷Dijon, France
Stanford Cancer Institute (SCI)
🇺🇸Stanford, California, United States
Hopital de la Cote de Nacre Avenue
🇫🇷Caen Cedex 9, France
Hopital Henri Mondor
🇫🇷Creteil, France
Charite-Universitatsmedizin
🇩🇪Berlin, Germany
Washington University in Saint Louis
🇺🇸Saint Louis, Missouri, United States
Hôpital Pitié-Salpêtrière
🇫🇷Paris, France
Azienda Ospedaliera Sant'Andrea, U.O.S.
🇮🇹Rome, Italy
Service d'Hermatologie CHU de Poitiers
🇫🇷Poitiers, France
Hospital Universitari Germans Trias i Pujol
🇪🇸Badalona, Spain
Universitätsklinikum Essen
🇩🇪Essen, Germany
Eastern Health (Box Hill Hospital)
🇦🇺Box Hill, Australia
Levine Cancer Institute
🇺🇸Charlotte, North Carolina, United States
The University of Queensland-Princess Alexandra Hospital (PAH)
🇦🇺Woolloongabba, Australia
Columbia University - College of Physicians & Surgeons
🇺🇸New York, New York, United States
Hopitaux Lyon Sud
🇫🇷Pierre-Benite Cedex, France
CHU Rennes, Service de Medecine Interne
🇫🇷Rennes Cedex 2, France
University Hospitals Bristol NHS Foundation Trust - Bristol Haematology and Oncology Centre
🇬🇧Bristol, United Kingdom
Policlinica San Matteo
🇮🇹Pavia, Italy
Hôpital Dupuytren - CHU Limoges
🇫🇷Limoges, France
Universitatsklinikum Heidelberg
🇩🇪Heidelberg, Germany
Meir Medical Center
🇮🇱Kfar Saba, Israel
Clínica Universidad de Navarra
🇪🇸Pamplona, Nevarra, Spain
Hospital Universitario Puerta de Hierro - Maiadahonda
🇪🇸Madrid, Spain
Policlinico S.Orsola Malpighi, Unita' Operativa di Ematologia
🇮🇹Bologna, Italy
Hospital Universitario 12 de Octubre
🇪🇸Madrid, Spain
Hospital Clinic de Barcelona
🇪🇸Barcelona, Spain
The Royal Free London NHS Foundation Trust-The Royal Free Hospital
🇬🇧London, United Kingdom
NNUH NHS Foundation Trust - Norfolk and Norwich University Hospital
🇬🇧Norwich, United Kingdom
Winship Cancer Institute of Emory University
🇺🇸Atlanta, Georgia, United States
John Hopkins University School of Medicine
🇺🇸Baltimore, Maryland, United States
University of Chicago Medicine
🇺🇸Chicago, Illinois, United States
Tufts Medical Center
🇺🇸Boston, Massachusetts, United States
The Cleveland Clinic - Main Campus
🇺🇸Cleveland, Ohio, United States
Hospital of the University of Pennsylvania
🇺🇸Philadelphia, Pennsylvania, United States
University of Pittsburg - Hillman Cancer Center
🇺🇸Pittsburgh, Pennsylvania, United States
University of Texas; MD Anderson Cancer Center
🇺🇸Houston, Texas, United States
Princess Margaret Cancer Research Centre
🇨🇦Toronto, Ontario, Canada
Sylvester Cancer Center, University of Miami
🇺🇸Miami, Florida, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
Indiana University Cancer Center
🇺🇸Indianapolis, Indiana, United States
Duke University Medical Center
🇺🇸Durham, North Carolina, United States
Huntsman Cancer Institute
🇺🇸Salt Lake City, Utah, United States
City of Hope
🇺🇸Duarte, California, United States
Radiant Research Northwestern University
🇺🇸Chicago, Illinois, United States
Boston University School of Medicine
🇺🇸Boston, Massachusetts, United States
Memorial Sloan-Kettering Cancer Center
🇺🇸New York, New York, United States
Amyloidosis Clinic Cnr Darcy Road and Bridge St. Westmead
🇦🇺Sydney, Australia
Hadassah University Medical Center
🇮🇱Jerusalem, Israel
The Chaim Sheba Medical Center
🇮🇱Ramat Gan, Israel
MTZ Clinical Research Sp. z o.o.
🇵🇱Warszawa, Poland
University Hospitals Birmingham NHS Foundation Trust - Queen Elizabeth Hospital
🇬🇧Birmingham, United Kingdom
Leicester Royal Infirmary
🇬🇧Leicester, United Kingdom
Southampton General Hospital
🇬🇧Southampton, United Kingdom
Rigshospitalet
🇩🇰Copenhagen, Denmark
Universitatsklinikum Hamburg-Eppendorf (UKE)
🇩🇪Hamburg, Germany
University of Washington
🇺🇸Seattle, Washington, United States
University Medical Center Groningen
🇳🇱Groningen, Netherlands
ZNA Stuivenberg
🇧🇪Antwerpen, Belgium
Cross Cancer Institute
🇨🇦Edmonton, Alberta, Canada
University of Calgary-Southern Alberta Cancer Research Institute
🇨🇦Calgary, Alberta, Canada
Jewish General Hospital
🇨🇦Montreal, Quebec, Canada
University Hospital of Patras
🇬🇷Patra, Greece
Tel Aviv Sourasky Medical Center
🇮🇱Tel Aviv, Israel
Universitair Medisch Centrum Utrecht
🇳🇱Utrecht, Netherlands
Newark Beth Israel Medical Center
🇺🇸Newark, New Jersey, United States
Alexandra General Hospital of Athens
🇬🇷Athens, Greece
Universitatsklinik fur Innere Medizin 1 Kinische Abteilung fur Onkologie Aligemeines Krankenhaus - Universitatskliniken
🇦🇹Wien, Austria
ULB Hopital Erasme
🇧🇪Brussel, Belgium
UZ Gasthuisberg
🇧🇪Leuven, Belgium
Mayo Clinic Arizona
🇺🇸Scottsdale, Arizona, United States
Colorado Blood Cancer Institute
🇺🇸Denver, Colorado, United States
Froedtert & Medical College of Wisconsin, Cancer Center-Froedtert Hospital
🇺🇸Milwaukee, Wisconsin, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
Karmanos Cancer Institute
🇺🇸Detroit, Michigan, United States
Ochsner Clinic Foundation
🇺🇸New Orleans, Louisiana, United States
Oregon Health & Science University
🇺🇸Portland, Oregon, United States
University of North Carolina Chapel Hill Comprehensive Cancer Center
🇺🇸Chapel Hill, North Carolina, United States