Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02632786
Registration number
NCT02632786
Ethics application status
Date submitted
9/12/2015
Date registered
17/12/2015
Date last updated
5/04/2019
Titles & IDs
Public title
The PRONTO Study, a Global Phase 2b Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis
Query!
Scientific title
A Phase 2b, Randomized, Double-blind, Placebo-controlled Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis Who Have Persistent Cardiac Dysfunction
Query!
Secondary ID [1]
0
0
NEOD001-201
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
PRONTO
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
AL Amyloidosis
0
0
Query!
Condition category
Condition code
Metabolic and Endocrine
0
0
0
0
Query!
Other metabolic disorders
Query!
Inflammatory and Immune System
0
0
0
0
Query!
Other inflammatory or immune system disorders
Query!
Metabolic and Endocrine
0
0
0
0
Query!
Metabolic disorders
Query!
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Other human genetics and inherited disorders
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Treatment: Drugs - NEOD001
Treatment: Drugs - Placebo
Experimental: NEOD001 - Study Drug given IV every 28 days at 24mg/kg
Placebo Comparator: Placebo - Placebo
Treatment: Drugs: NEOD001
NEOD001 is a monoclonal antibody directed at soluble and insoluble light chain aggregates
Treatment: Drugs: Placebo
Saline Bag
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Number of Participants With Cardiac Response and Non-Response
Query!
Assessment method [1]
0
0
N-terminal pro-brain natriuretic peptide (NT-proBNP ) best response (Response or Non-Response [Stable, Progression]) from baseline through 12 months of treatment. Cardiac best response, as assessed by NT-proBNP alone, is defined as the most favorable category among response (ie, decrease in NT-proBNP from baseline of >30% and >300 ng/L), stable (ie, neither response nor progression), and progression (ie, increase in NT-proBNP from baseline of >30% and >300 ng/L) across all visits after the first infusion of study drug up to and through the end of the study. Subjects are considered non-responders until a response is achieved. Non-response is defined as either stable or progression.
Query!
Timepoint [1]
0
0
Baseline through 12 months of treatment
Query!
Secondary outcome [1]
0
0
SF-36v2 PCS Score
Query!
Assessment method [1]
0
0
Change in Short Form-36 (SF-36 version 2) questionnaire Physical Component Summary [PCS] Score. PCS scores are calculated based on responses to specific Short Form-36 (version 2) questions using a weight scoring method. The lower the PCS score the more disability, the higher the score the less disability. A score of 50 is the mean in the US General Population and the standard deviation is 10. Minimum is 0 and maximum value is 100.
Query!
Timepoint [1]
0
0
Baseline to 12 months of treatment
Query!
Secondary outcome [2]
0
0
6MWT Distance
Query!
Assessment method [2]
0
0
Change in 6 Minute Walk Test (6MWT) Distance (meters)
Query!
Timepoint [2]
0
0
Baseline to 12 months of treatment
Query!
Secondary outcome [3]
0
0
Number of Participants With Renal Best Response and Non-Response
Query!
Assessment method [3]
0
0
Proteinuria and estimated Glomerular Filtration Rate (eGFR) response (Response or Non-Response [Stable, Progression]) from baseline through 12 months of treatment in subjects with renal involvement. Renal best response, as assessed by proteinuria, is defined as the most favorable category among response (ie, =30% decrease from baseline or <0.5 g/24 hours postbaseline result if subject does not meet criteria for progression), stable (ie, neither response nor progression), and progression (ie, =25% decrease in eGFR from baseline) across all visits after the first infusion of study drug up to and through the end of the study. Subjects are considered non-responders until a response is achieved. Assessments that qualify as both a response and progression are counted as progression.
Non-response is defined as either stable or progression.
Query!
Timepoint [3]
0
0
Baseline through 12 months of treatment
Query!
Secondary outcome [4]
0
0
NIS-LL Total Score
Query!
Assessment method [4]
0
0
Change in Neuropathy Impairment Score-Lower Limb (NIS-LL) Total Score in subjects with peripheral nerve involvement. NIS-LL is a scoring system graduated from 0 points to a maximum of 88 points (the absence of all motor, sensory, and reflex activity in the lower extremities). The scale is an additive of all deficits (64 potential points for muscle strength, 8 points for reflexes, and 16 points for sensory function) in the lower extremities. A score of 0 is normal and score of 88 is total impairment.
Query!
Timepoint [4]
0
0
Baseline to 12 months of treatment
Query!
Secondary outcome [5]
0
0
NT-proBNP Slope
Query!
Assessment method [5]
0
0
Rate of change in NT-proBNP (ng/L per infusion). Estimates of the intercept, slope, SE, and associated 95% CI for each treatment group, and the NEOD001 and placebo group difference comparisons are estimated using a general linear mixed effects model. The model fits a random intercept and slope for each subject and includes fixed effects for treatment group, time, treatment group by time interaction, IWRS stratification factors (hematologic response to first-line therapy: CR/VGPR, PR and NT-proBNP <1800 ng/L, =1800 ng/L), and an unstructured covariance structure to model the within-subject errors. Time is represented in months as a continuous variable and includes all scheduled time points, including baseline. The p-value is associated with the visit by treatment group interaction term.
Query!
Timepoint [5]
0
0
Baseline through 12 months of treatment
Query!
Secondary outcome [6]
0
0
Hepatic Best Response
Query!
Assessment method [6]
0
0
Alkaline Phosphatase response (Response or Non-Response [Stable, Progression]) from baseline through 12 months of treatment in subjects with hepatic involvement
Query!
Timepoint [6]
0
0
Baseline through 12 months of treatment
Query!
Eligibility
Key inclusion criteria
1. Age =18 years
2. Confirmed diagnosis of systemic AL amyloidosis
3. =1 prior systemic plasma cell dyscrasia therapy with at least a partial hematologic
response
4. Cardiac involvement
5. NT-proBNP =650
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key exclusion criteria
1. Non-AL amyloidosis
2. Meets the International Myeloma Working Group (IMWG) definition of Multiple Myeloma
3. NT-proBNP >5000
4. Received Plasma cell directed chemotherapy within 6 months
5. Received autologous stem cell transplant (ASCT) within 12 months
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Randomised controlled trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Blinded (masking used)
Query!
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
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 2
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
1/03/2016
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
1/03/2018
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
129
Query!
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Query!
Recruitment hospital [1]
0
0
Westmead Hospital - Sydney
Query!
Recruitment hospital [2]
0
0
The University of Queensland - Princess Alexandra Hospital (PAH) - Woolloongabba
Query!
Recruitment hospital [3]
0
0
Eastern Health (Box Hill Hospital) - Box Hill
Query!
Recruitment postcode(s) [1]
0
0
2145 - Sydney
Query!
Recruitment postcode(s) [2]
0
0
4102 - Woolloongabba
Query!
Recruitment postcode(s) [3]
0
0
3128 - Box Hill
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
California
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Colorado
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Florida
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Illinois
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Indiana
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Massachusetts
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
Michigan
Query!
Country [8]
0
0
United States of America
Query!
State/province [8]
0
0
Minnesota
Query!
Country [9]
0
0
United States of America
Query!
State/province [9]
0
0
New York
Query!
Country [10]
0
0
United States of America
Query!
State/province [10]
0
0
North Carolina
Query!
Country [11]
0
0
United States of America
Query!
State/province [11]
0
0
Ohio
Query!
Country [12]
0
0
United States of America
Query!
State/province [12]
0
0
Oregon
Query!
Country [13]
0
0
United States of America
Query!
State/province [13]
0
0
Pennsylvania
Query!
Country [14]
0
0
United States of America
Query!
State/province [14]
0
0
Tennessee
Query!
Country [15]
0
0
United States of America
Query!
State/province [15]
0
0
Texas
Query!
Country [16]
0
0
United States of America
Query!
State/province [16]
0
0
Washington
Query!
Country [17]
0
0
United States of America
Query!
State/province [17]
0
0
Wisconsin
Query!
Country [18]
0
0
Austria
Query!
State/province [18]
0
0
Vienna
Query!
Country [19]
0
0
France
Query!
State/province [19]
0
0
Limoges
Query!
Country [20]
0
0
France
Query!
State/province [20]
0
0
Paris
Query!
Country [21]
0
0
France
Query!
State/province [21]
0
0
Pierre-Benite Cedex
Query!
Country [22]
0
0
France
Query!
State/province [22]
0
0
Rennes Cedex 2
Query!
Country [23]
0
0
Germany
Query!
State/province [23]
0
0
Berlin
Query!
Country [24]
0
0
Germany
Query!
State/province [24]
0
0
Essen
Query!
Country [25]
0
0
Germany
Query!
State/province [25]
0
0
Hamburg
Query!
Country [26]
0
0
Germany
Query!
State/province [26]
0
0
Heidelberg
Query!
Country [27]
0
0
Greece
Query!
State/province [27]
0
0
Athens
Query!
Country [28]
0
0
Greece
Query!
State/province [28]
0
0
Patras
Query!
Country [29]
0
0
Israel
Query!
State/province [29]
0
0
Jerusalem
Query!
Country [30]
0
0
Italy
Query!
State/province [30]
0
0
Pavia
Query!
Country [31]
0
0
Spain
Query!
State/province [31]
0
0
Barcelona
Query!
Country [32]
0
0
Spain
Query!
State/province [32]
0
0
Majadahonda
Query!
Country [33]
0
0
United Kingdom
Query!
State/province [33]
0
0
Birmingham
Query!
Country [34]
0
0
United Kingdom
Query!
State/province [34]
0
0
London
Query!
Country [35]
0
0
United Kingdom
Query!
State/province [35]
0
0
Southampton
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Query!
Name
Prothena Biosciences Ltd.
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief summary
This is a global, multicenter, Phase 2b, randomized, double-blind, placebo-controlled,
two-arm, parallel-group efficacy and safety study of NEOD001 as a single agent administered
intravenously in adults with AL amyloidosis who had a hematologic response to previous
treatment for their amyloidosis (e.g., chemotherapy, autologous stem cell transplant [ASCT])
and have persistent cardiac dysfunction.
Query!
Trial website
https://clinicaltrials.gov/ct2/show/NCT02632786
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02632786
Download to PDF