Primary Systemic (AL) AmyloidosisPhase 3VITALNCT02312206
The VITAL Amyloidosis Study, a Global Phase 3, Efficacy and Safety Study of NEOD001 in Patients With AL Amyloidosis
A Phase 3 study testing NEOD001 in people with Primary Systemic (AL) Amyloidosis, run by Prothena Biosciences Ltd. at 79 sites in 15 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Systemic (AL) Amyloidosis
- What is being tested?
- NEOD001
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 260 participants
- Where
- 79 sitesin 15 countries
- Study dates
- Feb 2015to May 2018
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
79 sites in 15 countries.
79 sites in 15 countries
United States32 sites
Winship Cancer Institute of Emory University
Atlanta, Georgia, 30322
John Hopkins University School of Medicine
Baltimore, Maryland, 21205
Tufts Medical Center
Boston, Massachusetts, 02111
Boston University School of Medicine
Boston, Massachusetts, 02118
University of North Carolina Chapel Hill Comprehensive Cancer Center
Chapel Hill, North Carolina, 27599-1651
Levine Cancer Institute
Charlotte, North Carolina, 28211
Radiant Research Northwestern University
Chicago, Illinois, 60611
University of Chicago Medicine
Chicago, Illinois, 60637
The Cleveland Clinic - Main Campus
Cleveland, Ohio, 44195
Colorado Blood Cancer Institute
Denver, Colorado, 80218
Karmanos Cancer Institute
Detroit, Michigan, 48201
City of Hope
Duarte, California, 91010
Duke University Medical Center
Durham, North Carolina, 27705
University of Texas; MD Anderson Cancer Center
Houston, Texas, 77030
Indiana University Cancer Center
Indianapolis, Indiana, 46202
Mayo Clinic
Jacksonville, Florida, 32224
Sylvester Cancer Center, University of Miami
Miami, Florida, 33312
Froedtert & Medical College of Wisconsin, Cancer Center-Froedtert Hospital
Milwaukee, Wisconsin, 53226
Vanderbilt University Medical Center
Nashville, Tennessee, 37232-5505
Ochsner Clinic Foundation
New Orleans, Louisiana
Columbia University - College of Physicians & Surgeons
New York, New York, 10032
Memorial Sloan-Kettering Cancer Center
New York, New York, 10065
Newark Beth Israel Medical Center
Newark, New Jersey, 07112
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104
University of Pittsburg - Hillman Cancer Center
Pittsburgh, Pennsylvania, 15232
Oregon Health & Science University
Portland, Oregon, 97239
Mayo Clinic
Rochester, Minnesota, 55905
Huntsman Cancer Institute
Salt Lake City, Utah, 84112
Mayo Clinic Arizona
Scottsdale, Arizona, 85259
University of Washington
Seattle, Washington, 98109
Washington University in Saint Louis
St Louis, Missouri, 63110
Stanford Cancer Institute (SCI)
Stanford, California, 94305
France8 sites
Hopital de la Cote de Nacre Avenue
Caen, 14033
Hopital Henri Mondor
Créteil, 94010
CHU Dijon - Hopital du Bocage
Dijon, 21079
Hôpital Dupuytren - CHU Limoges
Limoges, 87042
Hôpital Pitié-Salpêtrière
Paris, 75013
Hopitaux Lyon Sud
Pierre-Bénite, 69495
Service d'Hermatologie CHU de Poitiers
Poitiers, 86000
CHU Rennes, Service de Medecine Interne
Rennes
United Kingdom6 sites
University Hospitals Birmingham NHS Foundation Trust - Queen Elizabeth Hospital
Birmingham
University Hospitals Bristol NHS Foundation Trust - Bristol Haematology and Oncology Centre
Bristol
Leicester Royal Infirmary
Leicester
The Royal Free London NHS Foundation Trust-The Royal Free Hospital
London, NW3 2PF
NNUH NHS Foundation Trust - Norfolk and Norwich University Hospital
Norwich
Southampton General Hospital
Southampton
Spain5 sites
Hospital Universitari Germans Trias i Pujol
Badalona, 08916
Hospital Clinic de Barcelona
Barcelona, 08036
Hospital Universitario 12 de Octubre
Madrid, 28041
Hospital Universitario Puerta de Hierro - Maiadahonda
Madrid, 28222
Clínica Universidad de Navarra
Pamplona, Nevarra
Canada4 sites
University of Calgary-Southern Alberta Cancer Research Institute
Calgary, Alberta, T2N 4N2
Cross Cancer Institute
Edmonton, Alberta, T6G 122
Jewish General Hospital
Montreal, Quebec, H3T 1E2
Princess Margaret Cancer Research Centre
Toronto, Ontario, M5G 1X6
Germany4 sites
Charite-Universitatsmedizin
Berlin
Universitätsklinikum Essen
Essen, 45122
Universitatsklinikum Hamburg-Eppendorf (UKE)
Hamburg, 20246
Universitatsklinikum Heidelberg
Heidelberg, 69120
Israel4 sites
Hadassah University Medical Center
Jerusalem, 91120
Meir Medical Center
Kfar Saba, 44281
The Chaim Sheba Medical Center
Ramat Gan, 52621
Tel Aviv Sourasky Medical Center
Tel Aviv, 64239
Australia3 sites
Eastern Health (Box Hill Hospital)
Box Hill, 3128
Amyloidosis Clinic Cnr Darcy Road and Bridge St. Westmead
Sydney, 2145
The University of Queensland-Princess Alexandra Hospital (PAH)
Woolloongabba, 4102
Belgium3 sites
ZNA Stuivenberg
Antwerp, 2060
ULB Hopital Erasme
Brussels, 1070
UZ Gasthuisberg
Leuven, 3000
Italy3 sites
Policlinico S.Orsola Malpighi, Unita' Operativa di Ematologia
Bologna, 40138
Policlinica San Matteo
Pavia, 27100
Azienda Ospedaliera Sant'Andrea, U.O.S.
Rome, 00189
Greece2 sites
Alexandra General Hospital of Athens
Athens, 11528
University Hospital of Patras
Pátrai, 26504
Netherlands2 sites
University Medical Center Groningen
Groningen, 9713 GZ
Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX
Austria1 site
Universitatsklinik fur Innere Medizin 1 Kinische Abteilung fur Onkologie Aligemeines Krankenhaus - Universitatskliniken
Vienna, A-1090
About this study
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
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.
Read the 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.
- Conditions
- Primary Systemic (AL) Amyloidosis
- Being tested
- NEOD001, Placebo
- Medical subject headings
- Immunoglobulin Light-chain Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NEOD001Drug
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.
PlaceboOther
Placebo
| Study group | Role | What they receive |
|---|---|---|
| NEOD001 | Experimental | 24 mg/kg (maximum dose of 2500 mg) of NEOD001 administered once every 28 days. Receives: Drug: NEOD001 |
| Placebo | Placebo comparator | Placebo will be administered as a 250 mL bag of normal saline once every 28 days. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Randomization until the date of death or cardiac hospitalization, up to 32 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2014 (estimated)
- Study startFeb 2015
- Primary completionMay 2018
- Study completionMay 2018
Why it stopped: The VITAL study was terminated due to futility analysis
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary Systemic (AL) AmyloidosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 260 participants (actual)
Sponsor and investigators
- Lead sponsor
- Prothena Biosciences Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02312206
- Sponsor's protocol ID
- NEOD001-CL002
- Acronym
- VITAL
- Status last verified
- Oct 2023
- First submitted
- 2 Dec 2014
- Last updated
- 14 Nov 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
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.
PubMed 37366170
Study at a glance
From the registry record, last updated by the sponsor on 14 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02312206
- Acronym / sponsor ID
- VITAL / NEOD001-CL002
- Conditions
- Primary Systemic (AL) Amyloidosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The VITAL study was terminated due to futility analysis
- Enrolment
- 260 participants (actual)
- Lead sponsor
- Prothena Biosciences Ltd.Industry (pharma, biotech, devices)
- Study start
- Feb 2015
- Primary completion
- May 2018 (actual)
- Study completion
- May 2018 (actual)
- First posted
- 9 Dec 2014 (estimated)
- Results
- Posted 29 May 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| NEOD001 | Experimental | Drug: NEOD00124 mg/kg (maximum dose of 2500 mg) of NEOD001 administered once every 28 days. |
| Placebo | Placebo comparator | Other: PlaceboPlacebo will be administered as a 250 mL bag of normal saline once every 28 days. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Age ≥ 18 years
- Newly diagnosed, AL amyloidosis treatment naïve
- Bone marrow consistent with plasma cell dyscrasia
- Confirmed diagnosis of AL amyloidosis
- Cardiac involvement
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NEOD001NEOD001, 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. | Drug | NEOD001 |
| PlaceboPlacebo | Other | Placebo |
Primary outcome
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Nov 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 37366170Gertz 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.
Related trials for Primary Systemic (AL) Amyloidosis
Not yet recruitingPhase 3
Linvoseltamab Versus Daratumumab, Bortezomib, Cyclophosphamide, and Dexamethasone (Dara-Vcd) in Adult Participants With Newly Diagnosed Systemic Light Chain Amyloidosis
Newly Diagnosed Amyloidosis, Amyloidosis Stage I-IIIA
RecruitingPhase 2
Sonrotoclax Plus Dexamethasone With or Without Daratumumab Regimen in Patients With t(11;14) Primary AL Amyloidosis
AL Amyloidosis (AL), t(11;14) Positive
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
79 sites in 15 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing NEOD001 in people with Primary Systemic (AL) Amyloidosis, run by Prothena Biosciences Ltd. at 79 sites in 15 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 14 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 79 sites in United States, France, United Kingdom, Spain, Canada and 10 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 29 May 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02312206, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Nov 2023.