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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02312206




Registration number
NCT02312206
Ethics application status
Date submitted
2/12/2014
Date registered
9/12/2014
Date last updated
14/11/2023

Titles & IDs
Public title
The VITAL Amyloidosis Study, a Global Phase 3, Efficacy and Safety Study of NEOD001 in Patients With AL Amyloidosis
Scientific 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
Secondary ID [1] 0 0
NEOD001-CL002
Universal Trial Number (UTN)
Trial acronym
VITAL
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Primary Systemic (AL) Amyloidosis 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - NEOD001
Other interventions - Placebo

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

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


Treatment: Drugs: 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.

Other interventions: Placebo
Placebo
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Time to Composite of All-cause Mortality or Cardiac Hospitalization
Timepoint [1] 0 0
Randomization until the date of death or cardiac hospitalization, up to 32 months

Eligibility
Key inclusion criteria
Key

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

Key
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/02/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/05/2018
Sample size
Target
Accrual to date
Final
260
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Eastern Health (Box Hill Hospital) - Box Hill
Recruitment hospital [2] 0 0
Amyloidosis Clinic Cnr Darcy Road and Bridge St. Westmead - Sydney
Recruitment hospital [3] 0 0
The University of Queensland-Princess Alexandra Hospital (PAH) - Woolloongabba
Recruitment postcode(s) [1] 0 0
3128 - Box Hill
Recruitment postcode(s) [2] 0 0
2145 - Sydney
Recruitment postcode(s) [3] 0 0
4102 - Woolloongabba
Recruitment outside Australia
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United States of America
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Arizona
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California
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Colorado
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Florida
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Georgia
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Illinois
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Indiana
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Louisiana
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Maryland
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Massachusetts
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Michigan
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New York
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North Carolina
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Ohio
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Oregon
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Texas
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Utah
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Washington
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Wisconsin
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Austria
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Wien
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Belgium
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Antwerpen
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Leuven
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Ontario
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Copenhagen
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Berlin
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Germany
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Essen
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Germany
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Hamburg
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Germany
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Heidelberg
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Greece
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Athens
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Greece
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Patra
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Israel
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Jerusalem
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Israel
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Kfar Saba
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Israel
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Ramat Gan
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Israel
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Tel Aviv
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Italy
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Bologna
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Italy
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Pavia
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Rome
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Groningen
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Utrecht
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Poland
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Nevarra
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Badalona
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Norwich
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Southampton

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Prothena Biosciences Ltd.
Address
Country

Ethics approval
Ethics application status

Summary
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 website
https://clinicaltrials.gov/study/NCT02312206
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
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Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT02312206