NCT02312206

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.

Trial Health

68
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
260

participants targeted

Target at P50-P75 for phase_3

Timeline
Completed

Started Feb 2015

Typical duration for phase_3

Geographic Reach
14 countries

78 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

December 2, 2014

Completed
7 days until next milestone

First Posted

Study publicly available on registry

December 9, 2014

Completed
2 months until next milestone

Study Start

First participant enrolled

February 1, 2015

Completed
3.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2018

Completed
1.1 years until next milestone

Results Posted

Study results publicly available

May 29, 2019

Completed
Last Updated

November 14, 2023

Status Verified

October 1, 2023

Enrollment Period

3.2 years

First QC Date

December 2, 2014

Results QC Date

May 3, 2019

Last Update Submit

October 31, 2023

Conditions

Outcome Measures

Primary Outcomes (1)

  • 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

    Randomization until the date of death or cardiac hospitalization, up to 32 months

Study Arms (2)

NEOD001

EXPERIMENTAL

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

Drug: NEOD001

Placebo

PLACEBO COMPARATOR

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

Other: Placebo

Interventions

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.

NEOD001
PlaceboOTHER

Placebo

Placebo

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (79)

Mayo Clinic Arizona

Scottsdale, Arizona, 85259, United States

Location

City of Hope

Duarte, California, 91010, United States

Location

Stanford Cancer Institute (SCI)

Stanford, California, 94305, United States

Location

Colorado Blood Cancer Institute

Denver, Colorado, 80218, United States

Location

Mayo Clinic

Jacksonville, Florida, 32224, United States

Location

Sylvester Cancer Center, University of Miami

Miami, Florida, 33312, United States

Location

Winship Cancer Institute of Emory University

Atlanta, Georgia, 30322, United States

Location

Radiant Research Northwestern University

Chicago, Illinois, 60611, United States

Location

University of Chicago Medicine

Chicago, Illinois, 60637, United States

Location

Indiana University Cancer Center

Indianapolis, Indiana, 46202, United States

Location

Ochsner Clinic Foundation

New Orleans, Louisiana, United States

Location

John Hopkins University School of Medicine

Baltimore, Maryland, 21205, United States

Location

Tufts Medical Center

Boston, Massachusetts, 02111, United States

Location

Boston University School of Medicine

Boston, Massachusetts, 02118, United States

Location

Karmanos Cancer Institute

Detroit, Michigan, 48201, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Washington University in Saint Louis

St Louis, Missouri, 63110, United States

Location

Newark Beth Israel Medical Center

Newark, New Jersey, 07112, United States

Location

Columbia University - College of Physicians & Surgeons

New York, New York, 10032, United States

Location

Memorial Sloan-Kettering Cancer Center

New York, New York, 10065, United States

Location

University of North Carolina Chapel Hill Comprehensive Cancer Center

Chapel Hill, North Carolina, 27599-1651, United States

Location

Levine Cancer Institute

Charlotte, North Carolina, 28211, United States

Location

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

The Cleveland Clinic - Main Campus

Cleveland, Ohio, 44195, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

University of Pittsburg - Hillman Cancer Center

Pittsburgh, Pennsylvania, 15232, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232-5505, United States

Location

University of Texas; MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Huntsman Cancer Institute

Salt Lake City, Utah, 84112, United States

Location

University of Washington

Seattle, Washington, 98109, United States

Location

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

Milwaukee, Wisconsin, 53226, United States

Location

Eastern Health (Box Hill Hospital)

Box Hill, 3128, Australia

Location

Amyloidosis Clinic Cnr Darcy Road and Bridge St. Westmead

Sydney, 2145, Australia

Location

The University of Queensland-Princess Alexandra Hospital (PAH)

Woolloongabba, 4102, Australia

Location

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

Vienna, A-1090, Austria

Location

ZNA Stuivenberg

Antwerp, 2060, Belgium

Location

ULB Hopital Erasme

Brussels, 1070, Belgium

Location

UZ Gasthuisberg

Leuven, 3000, Belgium

Location

University of Calgary-Southern Alberta Cancer Research Institute

Calgary, Alberta, T2N 4N2, Canada

Location

Cross Cancer Institute

Edmonton, Alberta, T6G 122, Canada

Location

Princess Margaret Cancer Research Centre

Toronto, Ontario, M5G 1X6, Canada

Location

Jewish General Hospital

Montreal, Quebec, H3T 1E2, Canada

Location

Rigshospitalet

Copenhagen, Denmark

Location

Hopital de la Cote de Nacre Avenue

Caen, 14033, France

Location

Hopital Henri Mondor

Créteil, 94010, France

Location

CHU Dijon - Hopital du Bocage

Dijon, 21079, France

Location

Hôpital Dupuytren - CHU Limoges

Limoges, 87042, France

Location

Hôpital Pitié-Salpêtrière

Paris, 75013, France

Location

Hopitaux Lyon Sud

Pierre-Bénite, 69495, France

Location

Service d'Hermatologie CHU de Poitiers

Poitiers, 86000, France

Location

CHU Rennes, Service de Medecine Interne

Rennes, France

Location

Charite-Universitatsmedizin

Berlin, Germany

Location

Universitätsklinikum Essen

Essen, 45122, Germany

Location

Universitatsklinikum Hamburg-Eppendorf (UKE)

Hamburg, 20246, Germany

Location

Universitatsklinikum Heidelberg

Heidelberg, 69120, Germany

Location

Alexandra General Hospital of Athens

Athens, 11528, Greece

Location

University Hospital of Patras

Pátrai, 26504, Greece

Location

Hadassah University Medical Center

Jerusalem, 91120, Israel

Location

Meir Medical Center

Kfar Saba, 44281, Israel

Location

The Chaim Sheba Medical Center

Ramat Gan, 52621, Israel

Location

Tel Aviv Sourasky Medical Center

Tel Aviv, 64239, Israel

Location

Policlinico S.Orsola Malpighi, Unita' Operativa di Ematologia

Bologna, 40138, Italy

Location

Policlinica San Matteo

Pavia, 27100, Italy

Location

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

Rome, 00189, Italy

Location

University Medical Center Groningen

Groningen, 9713 GZ, Netherlands

Location

Universitair Medisch Centrum Utrecht

Utrecht, 3584 CX, Netherlands

Location

MTZ Clinical Research Sp. z o.o.

Warsaw, 02-106, Poland

Location

Clínica Universidad de Navarra

Pamplona, Nevarra, Spain

Location

Hospital Universitari Germans Trias i Pujol

Badalona, 08916, Spain

Location

Hospital Clinic de Barcelona

Barcelona, 08036, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

Location

Hospital Universitario Puerta de Hierro - Maiadahonda

Madrid, 28222, Spain

Location

University Hospitals Birmingham NHS Foundation Trust - Queen Elizabeth Hospital

Birmingham, United Kingdom

Location

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

Bristol, United Kingdom

Location

Leicester Royal Infirmary

Leicester, United Kingdom

Location

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

London, NW3 2PF, United Kingdom

Location

NNUH NHS Foundation Trust - Norfolk and Norwich University Hospital

Norwich, United Kingdom

Location

Southampton General Hospital

Southampton, United Kingdom

Location

Related Publications (1)

  • 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.

MeSH Terms

Conditions

Immunoglobulin Light-chain Amyloidosis

Interventions

birtamimab

Condition Hierarchy (Ancestors)

Neoplasms, Plasma CellNeoplasms by Histologic TypeNeoplasmsAmyloidosisProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic DiseasesLymphoproliferative DisordersImmunoproliferative DisordersImmune System DiseasesParaproteinemias

Results Point of Contact

Title
Wendy Curlin
Organization
Prothena Biosciences

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 2, 2014

First Posted

December 9, 2014

Study Start

February 1, 2015

Primary Completion

May 1, 2018

Study Completion

May 1, 2018

Last Updated

November 14, 2023

Results First Posted

May 29, 2019

Record last verified: 2023-10

Locations