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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03997383




Registration number
NCT03997383
Ethics application status
Date submitted
24/06/2019
Date registered
25/06/2019

Titles & IDs
Public title
APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
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)
Secondary ID [1] 0 0
2019-001458-24
Secondary ID [2] 0 0
ALN-TTR02-011
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline at Month 12 in Six-Minute Walk Test (6-MWT)
Timepoint [1] 0 0
Baseline, Month 12
Secondary outcome [1] 0 0
Change From Baseline at Month 12 in Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) Score
Timepoint [1] 0 0
Baseline, Month 12
Secondary outcome [2] 0 0
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
Timepoint [2] 0 0
Up to Month 12
Secondary outcome [3] 0 0
Composite Endpoint of All-Cause Mortality and Frequency of All-Cause Hospitalizations and Urgent HF Visits in Participants Not on Tafamidis at Baseline
Timepoint [3] 0 0
Up to Month 12
Secondary outcome [4] 0 0
Composite Endpoint of All-cause Mortality and Frequency of All-cause Hospitalizations and Urgent HF Visits in All Participants
Timepoint [4] 0 0
Up to Month 12

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

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
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
4/09/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/03/2027
Actual
Sample size
Target
Accrual to date
Final
360
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Clinical Trial Site - Box Hill
Recruitment hospital [2] 0 0
Clinical Trial Site - Westmead
Recruitment postcode(s) [1] 0 0
- Box Hill
Recruitment postcode(s) [2] 0 0
- Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
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United States of America
State/province [2] 0 0
Illinois
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United States of America
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Kansas
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United States of America
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Maryland
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United States of America
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Massachusetts
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Minnesota
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Missouri
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United States of America
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New York
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United States of America
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Ohio
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United States of America
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Pennsylvania
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United States of America
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Tennessee
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United States of America
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Texas
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Argentina
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Córdoba
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Argentina
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Rosario
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Belgium
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Aalst
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Belgium
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Hasselt
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Belgium
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Liège
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Belgium
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Roeselare
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Brazil
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Porto Alegre
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Brazil
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Ribeirão Preto
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Brazil
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Rio De Janeiro
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Brazil
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São Paulo
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Bulgaria
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Sofia
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Chile
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Santiago
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Czechia
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Brno
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Czechia
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Prague
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Czechia
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Praha 2
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Czechia
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Praha
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Denmark
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Aarhus
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Denmark
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Copenhagen
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Denmark
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Odense
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France
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Créteil
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France
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Rennes
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France
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Toulouse
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Hong Kong
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Lai Chi Kok
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Italy
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Bologna
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Italy
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Firenze
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Italy
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Messina
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Italy
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Pavia
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Japan
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Fukuoka
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Japan
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Kumamoto
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Japan
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Kurume
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Japan
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Matsumoto
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Japan
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Nagoya
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Japan
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Osaka
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Japan
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Tokyo
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Korea, Republic of
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Seoul
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Mexico
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Mexico City
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Netherlands
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Groningen
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Netherlands
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Maastricht
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New Zealand
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Christchurch
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New Zealand
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Hamilton
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Portugal
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Viseu
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Sweden
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Stockholm
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Taiwan
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Taipei
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United Kingdom
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Birmingham
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Cardiff
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Glasgow
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London
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Manchester
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United Kingdom
State/province [61] 0 0
Stockton-on-Tees

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alnylam Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Alnylam Pharmaceuticals
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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Access to Anonymized individual participant data that support these results is made available 12 months after study completion and not less than 12 months after the product and indication have been approved in the United States (US) and/or the European Union (EU).

Data will be provided contingent upon the approval of a research proposal and the execution of a data sharing agreement. Requests for access to data can be submitted via the website www.vivli.org.
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT03997383