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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/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
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
Secondary ID [1] 0 0
NEOD001-201
Universal Trial Number (UTN)
Trial acronym
PRONTO
Linked study record

Health condition
Health condition(s) or problem(s) studied:
AL Amyloidosis 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders
Metabolic and Endocrine 0 0 0 0
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
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
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Cardiac Response and Non-Response
Timepoint [1] 0 0
Baseline through 12 months of treatment
Secondary outcome [1] 0 0
SF-36v2 PCS Score
Timepoint [1] 0 0
Baseline to 12 months of treatment
Secondary outcome [2] 0 0
6MWT Distance
Timepoint [2] 0 0
Baseline to 12 months of treatment
Secondary outcome [3] 0 0
Number of Participants With Renal Best Response and Non-Response
Timepoint [3] 0 0
Baseline through 12 months of treatment
Secondary outcome [4] 0 0
NIS-LL Total Score
Timepoint [4] 0 0
Baseline to 12 months of treatment
Secondary outcome [5] 0 0
NT-proBNP Slope
Timepoint [5] 0 0
Baseline through 12 months of treatment
Secondary outcome [6] 0 0
Hepatic Best Response
Timepoint [6] 0 0
Baseline through 12 months of treatment

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
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 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

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 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/03/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/03/2018
Sample size
Target
Accrual to date
Final
129
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Westmead Hospital - Sydney
Recruitment hospital [2] 0 0
The University of Queensland - Princess Alexandra Hospital (PAH) - Woolloongabba
Recruitment hospital [3] 0 0
Eastern Health (Box Hill Hospital) - Box Hill
Recruitment postcode(s) [1] 0 0
2145 - Sydney
Recruitment postcode(s) [2] 0 0
4102 - Woolloongabba
Recruitment postcode(s) [3] 0 0
3128 - Box Hill
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Indiana
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Michigan
Country [8] 0 0
United States of America
State/province [8] 0 0
Minnesota
Country [9] 0 0
United States of America
State/province [9] 0 0
New York
Country [10] 0 0
United States of America
State/province [10] 0 0
North Carolina
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
Country [12] 0 0
United States of America
State/province [12] 0 0
Oregon
Country [13] 0 0
United States of America
State/province [13] 0 0
Pennsylvania
Country [14] 0 0
United States of America
State/province [14] 0 0
Tennessee
Country [15] 0 0
United States of America
State/province [15] 0 0
Texas
Country [16] 0 0
United States of America
State/province [16] 0 0
Washington
Country [17] 0 0
United States of America
State/province [17] 0 0
Wisconsin
Country [18] 0 0
Austria
State/province [18] 0 0
Vienna
Country [19] 0 0
France
State/province [19] 0 0
Limoges
Country [20] 0 0
France
State/province [20] 0 0
Paris
Country [21] 0 0
France
State/province [21] 0 0
Pierre-Benite Cedex
Country [22] 0 0
France
State/province [22] 0 0
Rennes Cedex 2
Country [23] 0 0
Germany
State/province [23] 0 0
Berlin
Country [24] 0 0
Germany
State/province [24] 0 0
Essen
Country [25] 0 0
Germany
State/province [25] 0 0
Hamburg
Country [26] 0 0
Germany
State/province [26] 0 0
Heidelberg
Country [27] 0 0
Greece
State/province [27] 0 0
Athens
Country [28] 0 0
Greece
State/province [28] 0 0
Patras
Country [29] 0 0
Israel
State/province [29] 0 0
Jerusalem
Country [30] 0 0
Italy
State/province [30] 0 0
Pavia
Country [31] 0 0
Spain
State/province [31] 0 0
Barcelona
Country [32] 0 0
Spain
State/province [32] 0 0
Majadahonda
Country [33] 0 0
United Kingdom
State/province [33] 0 0
Birmingham
Country [34] 0 0
United Kingdom
State/province [34] 0 0
London
Country [35] 0 0
United Kingdom
State/province [35] 0 0
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 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.
Trial website
https://clinicaltrials.gov/study/NCT02632786
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
Country 0 0
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/NCT02632786