Back to resultsThe VITAL Amyloidosis Study, a Global Phase 3, Efficacy and Safety Study of NEOD001 in Patients With AL Amyloidosis (VITAL)
Primary Purpose
Primary Systemic (AL) Amyloidosis
Status
Terminated
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
NEOD001
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Primary Systemic (AL) Amyloidosis
Eligibility Criteria
Key Inclusion Criteria:
- 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 Exclusion Criteria:
- 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
Sites / Locations
- Mayo Clinic Arizona
- City of Hope
- Stanford Cancer Institute (SCI)
- Colorado Blood Cancer Institute
- Mayo Clinic
- Sylvester Cancer Center, University of Miami
- Winship Cancer Institute of Emory University
- Radiant Research Northwestern University
- University of Chicago Medicine
- Indiana University Cancer Center
- Ochsner Clinic Foundation
- John Hopkins University School of Medicine
- Tufts Medical Center
- Boston University School of Medicine
- Karmanos Cancer Institute
- Mayo Clinic
- Washington University in Saint Louis
- Newark Beth Israel Medical Center
- Columbia University - College of Physicians & Surgeons
- Memorial Sloan-Kettering Cancer Center
- University of North Carolina Chapel Hill Comprehensive Cancer Center
- Levine Cancer Institute
- Duke University Medical Center
- The Cleveland Clinic - Main Campus
- Oregon Health & Science University
- Hospital of the University of Pennsylvania
- University of Pittsburg - Hillman Cancer Center
- Vanderbilt University Medical Center
- University of Texas; MD Anderson Cancer Center
- Huntsman Cancer Institute
- University of Washington
- Froedtert & Medical College of Wisconsin, Cancer Center-Froedtert Hospital
- Eastern Health (Box Hill Hospital)
- Amyloidosis Clinic Cnr Darcy Road and Bridge St. Westmead
- The University of Queensland-Princess Alexandra Hospital (PAH)
- Universitatsklinik fur Innere Medizin 1 Kinische Abteilung fur Onkologie Aligemeines Krankenhaus - Universitatskliniken
- ZNA Stuivenberg
- ULB Hopital Erasme
- UZ Gasthuisberg
- University of Calgary-Southern Alberta Cancer Research Institute
- Cross Cancer Institute
- Princess Margaret Cancer Research Centre
- Jewish General Hospital
- Rigshospitalet
- Hopital de la Cote de Nacre Avenue
- Hopital Henri Mondor
- CHU Dijon - Hopital du Bocage
- Hôpital Dupuytren - CHU Limoges
- Hôpital Pitié-Salpêtrière
- Hopitaux Lyon Sud
- Service d'Hermatologie CHU de Poitiers
- CHU Rennes, Service de Medecine Interne
- Charite-Universitatsmedizin
- Universitätsklinikum Essen
- Universitatsklinikum Hamburg-Eppendorf (UKE)
- Universitatsklinikum Heidelberg
- Alexandra General Hospital of Athens
- University Hospital of Patras
- Hadassah University Medical Center
- Meir Medical Center
- The Chaim Sheba Medical Center
- Tel Aviv Sourasky Medical Center
- Policlinico S.Orsola Malpighi, Unita' Operativa di Ematologia
- Policlinica San Matteo
- Azienda Ospedaliera Sant'Andrea, U.O.S.
- University Medical Center Groningen
- Universitair Medisch Centrum Utrecht
- MTZ Clinical Research Sp. z o.o.
- Clínica Universidad de Navarra
- Hospital Universitari Germans Trias i Pujol
- Hospital Clinic de Barcelona
- Hospital Universitario 12 de Octubre
- Hospital Universitario Puerta de Hierro - Maiadahonda
- University Hospitals Birmingham NHS Foundation Trust - Queen Elizabeth Hospital
- University Hospitals Bristol NHS Foundation Trust - Bristol Haematology and Oncology Centre
- Leicester Royal Infirmary
- The Royal Free London NHS Foundation Trust-The Royal Free Hospital
- NNUH NHS Foundation Trust - Norfolk and Norwich University Hospital
- Southampton General Hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
NEOD001
Placebo
Arm Description
24 mg/kg (maximum dose of 2500 mg) of NEOD001 administered once every 28 days.
Placebo will be administered as a 250 mL bag of normal saline once every 28 days.
Outcomes
Primary Outcome Measures
Time to Composite of All-cause Mortality or Cardiac Hospitalization
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
Full Information
NCT ID
NCT02312206
First Posted
December 2, 2014
Last Updated
May 28, 2019
Sponsor
Prothena Biosciences Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT02312206
Brief Title
The VITAL Amyloidosis Study, a Global Phase 3, Efficacy and Safety Study of NEOD001 in Patients With AL Amyloidosis
Acronym
VITAL
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
Study Type
Interventional
2. Study Status
Record Verification Date
May 2019
Overall Recruitment Status
Terminated
Why Stopped
The VITAL study was terminated due to futility analysis
Study Start Date
February 2015 (undefined)
Primary Completion Date
May 2018 (Actual)
Study Completion Date
May 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Prothena Biosciences Ltd.
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Systemic (AL) Amyloidosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
260 (Actual)
8. Arms, Groups, and Interventions
Arm Title
NEOD001
Arm Type
Experimental
Arm Description
24 mg/kg (maximum dose of 2500 mg) of NEOD001 administered once every 28 days.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo will be administered as a 250 mL bag of normal saline once every 28 days.
Intervention Type
Drug
Intervention Name(s)
NEOD001
Intervention Description
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.
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
Placebo
Primary Outcome Measure Information:
Title
Time to Composite of All-cause Mortality or Cardiac Hospitalization
Description
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
Time Frame
Randomization until the date of death or cardiac hospitalization, up to 32 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria:
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 Exclusion Criteria:
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
Facility Information:
Facility Name
Mayo Clinic Arizona
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85259
Country
United States
Facility Name
City of Hope
City
Duarte
State/Province
California
ZIP/Postal Code
91010
Country
United States
Facility Name
Stanford Cancer Institute (SCI)
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
Colorado Blood Cancer Institute
City
Denver
State/Province
Colorado
ZIP/Postal Code
80218
Country
United States
Facility Name
Mayo Clinic
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32224
Country
United States
Facility Name
Sylvester Cancer Center, University of Miami
City
Miami
State/Province
Florida
ZIP/Postal Code
33312
Country
United States
Facility Name
Winship Cancer Institute of Emory University
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Radiant Research Northwestern University
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
University of Chicago Medicine
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60637
Country
United States
Facility Name
Indiana University Cancer Center
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Ochsner Clinic Foundation
City
New Orleans
State/Province
Louisiana
Country
United States
Facility Name
John Hopkins University School of Medicine
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
Tufts Medical Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02111
Country
United States
Facility Name
Boston University School of Medicine
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02118
Country
United States
Facility Name
Karmanos Cancer Institute
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Facility Name
Mayo Clinic
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Washington University in Saint Louis
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Newark Beth Israel Medical Center
City
Newark
State/Province
New Jersey
ZIP/Postal Code
07112
Country
United States
Facility Name
Columbia University - College of Physicians & Surgeons
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Memorial Sloan-Kettering Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Facility Name
University of North Carolina Chapel Hill Comprehensive Cancer Center
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599-1651
Country
United States
Facility Name
Levine Cancer Institute
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28211
Country
United States
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27705
Country
United States
Facility Name
The Cleveland Clinic - Main Campus
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
Oregon Health & Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Hospital of the University of Pennsylvania
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
University of Pittsburg - Hillman Cancer Center
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15232
Country
United States
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232-5505
Country
United States
Facility Name
University of Texas; MD Anderson Cancer Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Huntsman Cancer Institute
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84112
Country
United States
Facility Name
University of Washington
City
Seattle
State/Province
Washington
ZIP/Postal Code
98109
Country
United States
Facility Name
Froedtert & Medical College of Wisconsin, Cancer Center-Froedtert Hospital
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Facility Name
Eastern Health (Box Hill Hospital)
City
Box Hill
ZIP/Postal Code
3128
Country
Australia
Facility Name
Amyloidosis Clinic Cnr Darcy Road and Bridge St. Westmead
City
Sydney
ZIP/Postal Code
2145
Country
Australia
Facility Name
The University of Queensland-Princess Alexandra Hospital (PAH)
City
Woolloongabba
ZIP/Postal Code
4102
Country
Australia
Facility Name
Universitatsklinik fur Innere Medizin 1 Kinische Abteilung fur Onkologie Aligemeines Krankenhaus - Universitatskliniken
City
Wien
ZIP/Postal Code
A-1090
Country
Austria
Facility Name
ZNA Stuivenberg
City
Antwerpen
ZIP/Postal Code
2060
Country
Belgium
Facility Name
ULB Hopital Erasme
City
Brussel
ZIP/Postal Code
1070
Country
Belgium
Facility Name
UZ Gasthuisberg
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
University of Calgary-Southern Alberta Cancer Research Institute
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T2N 4N2
Country
Canada
Facility Name
Cross Cancer Institute
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T6G 122
Country
Canada
Facility Name
Princess Margaret Cancer Research Centre
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X6
Country
Canada
Facility Name
Jewish General Hospital
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H3T 1E2
Country
Canada
Facility Name
Rigshospitalet
City
Copenhagen
Country
Denmark
Facility Name
Hopital de la Cote de Nacre Avenue
City
Caen Cedex 9
ZIP/Postal Code
14033
Country
France
Facility Name
Hopital Henri Mondor
City
Creteil
ZIP/Postal Code
94010
Country
France
Facility Name
CHU Dijon - Hopital du Bocage
City
Dijon
ZIP/Postal Code
21079
Country
France
Facility Name
Hôpital Dupuytren - CHU Limoges
City
Limoges
ZIP/Postal Code
87042
Country
France
Facility Name
Hôpital Pitié-Salpêtrière
City
Paris
ZIP/Postal Code
75013
Country
France
Facility Name
Hopitaux Lyon Sud
City
Pierre-Benite Cedex
ZIP/Postal Code
69495
Country
France
Facility Name
Service d'Hermatologie CHU de Poitiers
City
Poitiers
ZIP/Postal Code
86000
Country
France
Facility Name
CHU Rennes, Service de Medecine Interne
City
Rennes Cedex 2
Country
France
Facility Name
Charite-Universitatsmedizin
City
Berlin
Country
Germany
Facility Name
Universitätsklinikum Essen
City
Essen
ZIP/Postal Code
45122
Country
Germany
Facility Name
Universitatsklinikum Hamburg-Eppendorf (UKE)
City
Hamburg
ZIP/Postal Code
20246
Country
Germany
Facility Name
Universitatsklinikum Heidelberg
City
Heidelberg
ZIP/Postal Code
69120
Country
Germany
Facility Name
Alexandra General Hospital of Athens
City
Athens
ZIP/Postal Code
11528
Country
Greece
Facility Name
University Hospital of Patras
City
Patra
ZIP/Postal Code
26504
Country
Greece
Facility Name
Hadassah University Medical Center
City
Jerusalem
ZIP/Postal Code
91120
Country
Israel
Facility Name
Meir Medical Center
City
Kfar Saba
ZIP/Postal Code
44281
Country
Israel
Facility Name
The Chaim Sheba Medical Center
City
Ramat Gan
ZIP/Postal Code
52621
Country
Israel
Facility Name
Tel Aviv Sourasky Medical Center
City
Tel Aviv
ZIP/Postal Code
64239
Country
Israel
Facility Name
Policlinico S.Orsola Malpighi, Unita' Operativa di Ematologia
City
Bologna
ZIP/Postal Code
40138
Country
Italy
Facility Name
Policlinica San Matteo
City
Pavia
ZIP/Postal Code
27100
Country
Italy
Facility Name
Azienda Ospedaliera Sant'Andrea, U.O.S.
City
Rome
ZIP/Postal Code
00189
Country
Italy
Facility Name
University Medical Center Groningen
City
Groningen
ZIP/Postal Code
9713 GZ
Country
Netherlands
Facility Name
Universitair Medisch Centrum Utrecht
City
Utrecht
ZIP/Postal Code
3584 CX
Country
Netherlands
Facility Name
MTZ Clinical Research Sp. z o.o.
City
Warszawa
ZIP/Postal Code
02-106
Country
Poland
Facility Name
Clínica Universidad de Navarra
City
Pamplona
State/Province
Nevarra
Country
Spain
Facility Name
Hospital Universitari Germans Trias i Pujol
City
Badalona
ZIP/Postal Code
08916
Country
Spain
Facility Name
Hospital Clinic de Barcelona
City
Barcelona
ZIP/Postal Code
08036
Country
Spain
Facility Name
Hospital Universitario 12 de Octubre
City
Madrid
ZIP/Postal Code
28041
Country
Spain
Facility Name
Hospital Universitario Puerta de Hierro - Maiadahonda
City
Madrid
ZIP/Postal Code
28222
Country
Spain
Facility Name
University Hospitals Birmingham NHS Foundation Trust - Queen Elizabeth Hospital
City
Birmingham
Country
United Kingdom
Facility Name
University Hospitals Bristol NHS Foundation Trust - Bristol Haematology and Oncology Centre
City
Bristol
Country
United Kingdom
Facility Name
Leicester Royal Infirmary
City
Leicester
Country
United Kingdom
Facility Name
The Royal Free London NHS Foundation Trust-The Royal Free Hospital
City
London
ZIP/Postal Code
NW3 2PF
Country
United Kingdom
Facility Name
NNUH NHS Foundation Trust - Norfolk and Norwich University Hospital
City
Norwich
Country
United Kingdom
Facility Name
Southampton General Hospital
City
Southampton
Country
United Kingdom
12. IPD Sharing Statement
Learn more about this trial
The VITAL Amyloidosis Study, a Global Phase 3, Efficacy and Safety Study of NEOD001 in Patients With AL Amyloidosis
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