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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT03997383
Registration number
NCT03997383
Ethics application status
Date submitted
24/06/2019
Date registered
25/06/2019
Date last updated
24/05/2024
Titles & IDs
Public title
APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
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Scientific title
APOLLO-B: A Phase 3, Randomized, Double-blind, Placebo-controlled Multicenter Study to Evaluate the Efficacy and Safety of Patisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
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Secondary ID [1]
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2019-001458-24
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Secondary ID [2]
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ALN-TTR02-011
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Neurological
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Other neurological disorders
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Metabolic and Endocrine
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Other metabolic disorders
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Metabolic and Endocrine
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Metabolic disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Placebo
Treatment: Drugs - Patisiran
Experimental: Patisiran - Participants will be administered multiple doses of patisiran in the double-blind and open-label extension period.
Placebo Comparator: Placebo - Participants will be administered multiple doses of placebo in the double-blind period. In the open-label extension period, participants will be administered multiple doses of patisiran.
Treatment: Drugs: Placebo
Normal saline (0.9% NaCl) matching volume of patisiran doses will be administered intravenously.
Treatment: Drugs: Patisiran
Patisiran will be administered by intravenous (IV) infusion.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Change From Baseline at Month 12 in Six-Minute Walk Test (6-MWT)
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Assessment method [1]
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Distance in meters walked in 6 minutes, longer distances indicate greater functional capacity. Missing 6MWT values due to non-COVID-19 death or inability to walk due to ATTR disease progression were imputed using the worst 10th percentile change observed in the DB period. Missing 6-MWT values due to other reasons are multiply imputed to create 100 complete datasets. The change from baseline is averaged across the 100 complete datasets.
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Timepoint [1]
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Baseline, Month 12
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Secondary outcome [1]
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Change From Baseline at Month 12 in Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) Score
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Assessment method [1]
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The KCCQ is a 23-item self-administered questionnaire quantifying 6 domains (symptoms, physical function, quality of life, social limitation, self-efficacy, and symptom stability) and 2 summary scores (clinical and overall summary [OS]). Scores are transformed to a range of 0-100, in which higher scores reflect better health status.
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Timepoint [1]
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Baseline, Month 12
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Secondary outcome [2]
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Composite Endpoint of All-Cause Mortality, Frequency of Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits) and Change From Baseline in 6-MWT Analyzed by Win Ratio
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Assessment method [2]
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The composite endpoint was analyzed using the stratified win ratio method, stratified by baseline tafamidis use. This method combines all-cause mortality, frequency of CV events (CV hospitalizations and HF visits) and change from baseline in 6-MWT in a hierarchical fashion. This method makes within-stratum pairwise comparisons for all patisiran-placebo participant pairs in a sequential manner (first mortality, then CV events, then 6-MWT), with later steps evaluated only in the case of a tie on the prior step. Within each stratum, the win ratio is the total number of 'winners' divided by the total number of 'losers' in the active group. A win ratio >1 represents a favorable outcome for patisiran.
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Timepoint [2]
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Up to Month 12
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Secondary outcome [3]
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Composite Endpoint of All-Cause Mortality and Frequency of All-Cause Hospitalizations and Urgent HF Visits in Participants Not on Tafamidis at Baseline
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Assessment method [3]
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The hazard rate of all-cause mortality and all-cause hospitalizations and urgent HF visits will be compared between treatment groups using an Andersen-Gill model. A hazard ratio <1 represents a favorable outcome for patisiran.
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Timepoint [3]
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Up to Month 12
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Secondary outcome [4]
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Composite Endpoint of All-cause Mortality and Frequency of All-cause Hospitalizations and Urgent HF Visits in All Participants
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Assessment method [4]
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The hazard rate of all-cause mortality and all-cause hospitalizations and urgent HF visits was compared between treatment groups using a modified Andersen-Gill model. A hazard ratio <1 represents a favorable outcome for patisiran.
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Timepoint [4]
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Up to Month 12
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Eligibility
Key inclusion criteria
- Documented diagnosis of ATTR amyloidosis with cardiomyopathy, classified as either
hereditary ATTR amyloidosis with cardiomyopathy or wild-type ATTR amyloidosis with
cardiomyopathy
- Medical history of heart failure with at least 1 prior hospitalization for heart
failure, or current clinical evidence (signs and symptoms of heart failure)
- Clinically stable with no cardiovascular related hospitalizations within 6 weeks of
study start
- Has never taken tafamidis before (tafamidis naïve) or currently on tafamidis for =6
months with evidence of disease progression while on tafamidis treatment
- Able to complete =150 m on the 6-minute walk test
- Screening N-terminal pro B-type natriuretic peptide (NT-proBNP), a blood marker of
heart failure severity, >300 ng/L and <8500 ng/L; in participants with permanent or
persistent atrial fibrillation, screening NT-proBNP> 600 ng/L and <8500 ng/L
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Minimum age
18
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Maximum age
85
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Known primary amyloidosis (AL) or leptomeningeal amyloidosis.
- Received prior TTR lowering treatment
- New York Heart Association heart failure classification of III and at high risk
- New York Heart Association heart failure classification of IV
- Neuropathy requiring cane or stick to walk, or is wheelchair bound
- Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m^2
- Abnormal liver function
- Has hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection
- Has non-amyloid disease that significantly affects ability to walk (e.g., severe
chronic obstructive pulmonary disease, severe arthritis, or peripheral vascular
disease affecting ambulation)
- Prior or planned heart, liver, or other organ transplant
- Other cardiomyopathy not related to ATTR amyloidosis
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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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
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
4/09/2019
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/03/2027
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Actual
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Sample size
Target
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Accrual to date
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Final
360
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Clinical Trial Site - Box Hill
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Recruitment hospital [2]
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Clinical Trial Site - Westmead
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Recruitment postcode(s) [1]
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- Box Hill
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Recruitment postcode(s) [2]
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- Westmead
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Recruitment outside Australia
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Alnylam Pharmaceuticals
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Address
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Ethics approval
Ethics application status
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Summary
Brief summary
The purpose of this study is to evaluate the efficacy and safety of patisiran in participants
with ATTR amyloidosis with cardiomyopathy.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT03997383
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Contacts
Principal investigator
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Medical Director
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Alnylam Pharmaceuticals
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03997383
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