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

NCT ID: NCT02312206

Last Updated: 2023-11-14

Study Results

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE3

Total Enrollment

260 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-02-28

Study Completion Date

2018-05-31

Brief Summary

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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

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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

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Primary Systemic (AL) Amyloidosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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NEOD001

24 mg/kg (maximum dose of 2500 mg) of NEOD001 administered once every 28 days.

Group Type EXPERIMENTAL

NEOD001

Intervention Type DRUG

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

Placebo will be administered as a 250 mL bag of normal saline once every 28 days.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Placebo

Interventions

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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.

Intervention Type DRUG

Placebo

Placebo

Intervention Type OTHER

Eligibility Criteria

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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

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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Prothena Biosciences Ltd.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Mayo Clinic Arizona

Scottsdale, Arizona, United States

Site Status

City of Hope

Duarte, California, United States

Site Status

Stanford Cancer Institute (SCI)

Stanford, California, United States

Site Status

Colorado Blood Cancer Institute

Denver, Colorado, United States

Site Status

Mayo Clinic

Jacksonville, Florida, United States

Site Status

Sylvester Cancer Center, University of Miami

Miami, Florida, United States

Site Status

Winship Cancer Institute of Emory University

Atlanta, Georgia, United States

Site Status

Radiant Research Northwestern University

Chicago, Illinois, United States

Site Status

University of Chicago Medicine

Chicago, Illinois, United States

Site Status

Indiana University Cancer Center

Indianapolis, Indiana, United States

Site Status

Ochsner Clinic Foundation

New Orleans, Louisiana, United States

Site Status

John Hopkins University School of Medicine

Baltimore, Maryland, United States

Site Status

Tufts Medical Center

Boston, Massachusetts, United States

Site Status

Boston University School of Medicine

Boston, Massachusetts, United States

Site Status

Karmanos Cancer Institute

Detroit, Michigan, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

Washington University in Saint Louis

St Louis, Missouri, United States

Site Status

Newark Beth Israel Medical Center

Newark, New Jersey, United States

Site Status

Columbia University - College of Physicians & Surgeons

New York, New York, United States

Site Status

Memorial Sloan-Kettering Cancer Center

New York, New York, United States

Site Status

University of North Carolina Chapel Hill Comprehensive Cancer Center

Chapel Hill, North Carolina, United States

Site Status

Levine Cancer Institute

Charlotte, North Carolina, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

The Cleveland Clinic - Main Campus

Cleveland, Ohio, United States

Site Status

Oregon Health & Science University

Portland, Oregon, United States

Site Status

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

University of Pittsburg - Hillman Cancer Center

Pittsburgh, Pennsylvania, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

University of Texas; MD Anderson Cancer Center

Houston, Texas, United States

Site Status

Huntsman Cancer Institute

Salt Lake City, Utah, United States

Site Status

University of Washington

Seattle, Washington, United States

Site Status

Froedtert & Medical College of Wisconsin, Cancer Center-Froedtert Hospital

Milwaukee, Wisconsin, United States

Site Status

Eastern Health (Box Hill Hospital)

Box Hill, , Australia

Site Status

Amyloidosis Clinic Cnr Darcy Road and Bridge St. Westmead

Sydney, , Australia

Site Status

The University of Queensland-Princess Alexandra Hospital (PAH)

Woolloongabba, , Australia

Site Status

Universitatsklinik fur Innere Medizin 1 Kinische Abteilung fur Onkologie Aligemeines Krankenhaus - Universitatskliniken

Vienna, , Austria

Site Status

ZNA Stuivenberg

Antwerp, , Belgium

Site Status

ULB Hopital Erasme

Brussels, , Belgium

Site Status

UZ Gasthuisberg

Leuven, , Belgium

Site Status

University of Calgary-Southern Alberta Cancer Research Institute

Calgary, Alberta, Canada

Site Status

Cross Cancer Institute

Edmonton, Alberta, Canada

Site Status

Princess Margaret Cancer Research Centre

Toronto, Ontario, Canada

Site Status

Jewish General Hospital

Montreal, Quebec, Canada

Site Status

Rigshospitalet

Copenhagen, , Denmark

Site Status

Hopital de la Cote de Nacre Avenue

Caen, , France

Site Status

Hopital Henri Mondor

Créteil, , France

Site Status

CHU Dijon - Hopital du Bocage

Dijon, , France

Site Status

Hôpital Dupuytren - CHU Limoges

Limoges, , France

Site Status

Hôpital Pitié-Salpêtrière

Paris, , France

Site Status

Hopitaux Lyon Sud

Pierre-Bénite, , France

Site Status

Service d'Hermatologie CHU de Poitiers

Poitiers, , France

Site Status

CHU Rennes, Service de Medecine Interne

Rennes, , France

Site Status

Charite-Universitatsmedizin

Berlin, , Germany

Site Status

Universitätsklinikum Essen

Essen, , Germany

Site Status

Universitatsklinikum Hamburg-Eppendorf (UKE)

Hamburg, , Germany

Site Status

Universitatsklinikum Heidelberg

Heidelberg, , Germany

Site Status

Alexandra General Hospital of Athens

Athens, , Greece

Site Status

University Hospital of Patras

Pátrai, , Greece

Site Status

Hadassah University Medical Center

Jerusalem, , Israel

Site Status

Meir Medical Center

Kfar Saba, , Israel

Site Status

The Chaim Sheba Medical Center

Ramat Gan, , Israel

Site Status

Tel Aviv Sourasky Medical Center

Tel Aviv, , Israel

Site Status

Policlinico S.Orsola Malpighi, Unita' Operativa di Ematologia

Bologna, , Italy

Site Status

Policlinica San Matteo

Pavia, , Italy

Site Status

Azienda Ospedaliera Sant'Andrea, U.O.S.

Rome, , Italy

Site Status

University Medical Center Groningen

Groningen, , Netherlands

Site Status

Universitair Medisch Centrum Utrecht

Utrecht, , Netherlands

Site Status

MTZ Clinical Research Sp. z o.o.

Warsaw, , Poland

Site Status

Clínica Universidad de Navarra

Pamplona, Nevarra, Spain

Site Status

Hospital Universitari Germans Trias i Pujol

Badalona, , Spain

Site Status

Hospital Clinic de Barcelona

Barcelona, , Spain

Site Status

Hospital Universitario 12 de Octubre

Madrid, , Spain

Site Status

Hospital Universitario Puerta de Hierro - Maiadahonda

Madrid, , Spain

Site Status

University Hospitals Birmingham NHS Foundation Trust - Queen Elizabeth Hospital

Birmingham, , United Kingdom

Site Status

University Hospitals Bristol NHS Foundation Trust - Bristol Haematology and Oncology Centre

Bristol, , United Kingdom

Site Status

Leicester Royal Infirmary

Leicester, , United Kingdom

Site Status

The Royal Free London NHS Foundation Trust-The Royal Free Hospital

London, , United Kingdom

Site Status

NNUH NHS Foundation Trust - Norfolk and Norwich University Hospital

Norwich, , United Kingdom

Site Status

Southampton General Hospital

Southampton, , United Kingdom

Site Status

Countries

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United States Australia Austria Belgium Canada Denmark France Germany Greece Israel Italy Netherlands Poland Spain United Kingdom

References

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Gertz MA, Cohen AD, Comenzo RL, Kastritis E, Landau HJ, Libby EN, Liedtke M, Sanchorawala V, Schonland S, Wechalekar A, Zonder JA, Palladini G, Walling J, Guthrie S, Nie C, Karp C, Jin Y, Kinney GG, Merlini G. Birtamimab plus standard of care in light-chain amyloidosis: the phase 3 randomized placebo-controlled VITAL trial. Blood. 2023 Oct 5;142(14):1208-1218. doi: 10.1182/blood.2022019406.

Reference Type DERIVED
PMID: 37366170 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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NEOD001-CL002

Identifier Type: -

Identifier Source: org_study_id

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