APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
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)
2 other identifiers
interventional
360
22 countries
90
Brief Summary
The purpose of this study is to evaluate the efficacy and safety of patisiran in participants with ATTR amyloidosis with cardiomyopathy.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3
Started Sep 2019
Longer than P75 for phase_3
90 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 24, 2019
CompletedFirst Posted
Study publicly available on registry
June 25, 2019
CompletedStudy Start
First participant enrolled
September 4, 2019
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 20, 2022
CompletedResults Posted
Study results publicly available
October 18, 2023
CompletedStudy Completion
Last participant's last visit for all outcomes
December 24, 2025
CompletedApril 20, 2026
March 1, 2026
2.8 years
June 24, 2019
June 20, 2023
March 30, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change From Baseline at Month 12 in Six-Minute Walk Test (6-MWT)
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.
Baseline, Month 12
Secondary Outcomes (4)
Change From Baseline at Month 12 in Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) Score
Baseline, Month 12
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
Up to Month 12
Composite Endpoint of All-Cause Mortality and Frequency of All-Cause Hospitalizations and Urgent HF Visits in Participants Not on Tafamidis at Baseline
Up to Month 12
Composite Endpoint of All-cause Mortality and Frequency of All-cause Hospitalizations and Urgent HF Visits in All Participants
Up to Month 12
Study Arms (2)
Patisiran
EXPERIMENTALParticipants will be administered multiple doses of patisiran in the double-blind and open-label extension period.
Placebo
PLACEBO COMPARATORParticipants 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.
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (90)
Clinical Trial Site
Los Angeles, California, 90048, United States
Clinical Trial Site
Chicago, Illinois, 60637, United States
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Skokie, Illinois, 60076, United States
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Iowa City, Iowa, 52242, United States
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Kansas City, Kansas, 66160, United States
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Baltimore, Maryland, 21224, United States
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Boston, Massachusetts, 02118, United States
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Burlington, Massachusetts, 10805, United States
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Rochester, Minnesota, 55905, United States
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St Louis, Missouri, 63110, United States
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New York, New York, 10029, United States
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New York, New York, 10032, United States
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Cleveland, Ohio, 44195, United States
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Philadelphia, Pennsylvania, 19104, United States
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Philadelphia, Pennsylvania, 19140, United States
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Pittsburgh, Pennsylvania, 15212, United States
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Nashville, Tennessee, 37232, United States
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Dallas, Texas, 75246, United States
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Norfolk, Virginia, 23507, United States
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Buenos Aires, C1039AAO, Argentina
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Buenos Aires, C1428AQK, Argentina
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Córdoba, X5000KEH, Argentina
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Rosario, S2000DSR, Argentina
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Rosario, S2000DTC, Argentina
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Rosario, S2000PBJ, Argentina
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San Juan Bautista, B1888AAE, Argentina
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Santa Fe, S3000FWO, Argentina
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Box Hill, 3128, Australia
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Westmead, 2145, Australia
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Woolloongabba, 4021, Australia
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Aalst, 9300, Belgium
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Hasselt, 3500, Belgium
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Liège, 4000, Belgium
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Roeselare, 8800, Belgium
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Porto Alegre, 90035-030, Brazil
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Ribeirão Preto, 14026-900, Brazil
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Ribeirão Preto, 14048-900, Brazil
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Rio de Janeiro, 22280-000, Brazil
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São Paulo, 04012-909, Brazil
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São Paulo, 05403-000, Brazil
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Sofia, 1680, Bulgaria
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Sofia, 1784, Bulgaria
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Stara Zagora, 6000, Bulgaria
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Providencia/ Santiago, 7500587, Chile
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Brno, 656 91, Czechia
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Prague, 120 00, Czechia
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Prague, 128 08, Czechia
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Prague, 140 21, Czechia
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Aarhus N, 8200, Denmark
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Copenhagen, 2100, Denmark
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Odense C, 5000, Denmark
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Clichy, 92110, France
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Créteil, 94010, France
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Rennes, 35033, France
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Toulouse, 31059, France
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Lai Chi Kok, 999077, Hong Kong
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Bologna, 40138, Italy
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Florence, 50134, Italy
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Messina, 98125, Italy
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Pavia, 27100, Italy
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Bunkyō City, 113-8655, Japan
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Fukuoka, 812-8582, Japan
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Kumamoto, 860-8556, Japan
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Kurume, 830-0011, Japan
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Matsumoto, 390-8621, Japan
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Nagoya, 466-8560, Japan
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Suita, 565-8565, Japan
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Mexico City, 14080, Mexico
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Groningen, 9713 GZ, Netherlands
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Maastricht, 6229 HX, Netherlands
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Christchurch, 8011, New Zealand
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Hamilton, 3204, New Zealand
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Gdansk, 80-382, Poland
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Katowice, 40-635, Poland
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Lódz, 90-127, Poland
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Warsaw, 01-192, Poland
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Warsaw, 04-628, Poland
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Porto, 4099-001, Portugal
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Senhora da Hora, 4464-513, Portugal
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Viseu, 3504-509, Portugal
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Seoul, 3080, South Korea
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Stockholm, 17164, Sweden
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Taipei, 11217, Taiwan
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Bellshill, ML4 3NJ, United Kingdom
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Birmingham, B15 2SQ, United Kingdom
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Cardiff, CF15 9SS, United Kingdom
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Hexham, NE46 1QJ, United Kingdom
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London, NW3 2PF, United Kingdom
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London, SE1 1YR, United Kingdom
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Manchester, M15 6SE, United Kingdom
Related Publications (2)
Mesquita CT, Schwartzmann P, Correia EB, Simoes MV, Biolo A, Duque DR, Jay PY, Fernandes F. Patisiran Treatment in the Brazilian Subpopulation of the Phase 3 APOLLO-B Study in Transthyretin Amyloidosis with Cardiomyopathy: Post Hoc Analysis. Arq Bras Cardiol. 2025 Mar;122(4):e20240568. doi: 10.36660/abc.20240568. English, Portuguese.
PMID: 40243852DERIVEDMaurer MS, Kale P, Fontana M, Berk JL, Grogan M, Gustafsson F, Hung RR, Gottlieb RL, Damy T, Gonzalez-Duarte A, Sarswat N, Sekijima Y, Tahara N, Taylor MS, Kubanek M, Donal E, Palecek T, Tsujita K, Tang WHW, Yu WC, Obici L, Simoes M, Fernandes F, Poulsen SH, Diemberger I, Perfetto F, Solomon SD, Di Carli M, Badri P, White MT, Chen J, Yureneva E, Sweetser MT, Jay PY, Garg PP, Vest J, Gillmore JD; APOLLO-B Trial Investigators. Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis. N Engl J Med. 2023 Oct 26;389(17):1553-1565. doi: 10.1056/NEJMoa2300757.
PMID: 37888916DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Chief Medical Officer
- Organization
- Alnylam Pharmaceuticals Inc.
Study Officials
- STUDY DIRECTOR
Medical Director
Alnylam Pharmaceuticals
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 24, 2019
First Posted
June 25, 2019
Study Start
September 4, 2019
Primary Completion
June 20, 2022
Study Completion
December 24, 2025
Last Updated
April 20, 2026
Results First Posted
October 18, 2023
Record last verified: 2026-03
Data Sharing
- IPD Sharing
- Will share
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 US and/or the EU. Access to data may be declined where there is likelihood a patient could be identified or other feasibility issue, where there is a potential conflict of interest, a planned business activities or an actual or potential competitive risk. Data will be provided contingent upon the approval of a research proposal and the execution of a data sharing agreement. Timeframes for data access may vary and can take up to 6 months or more. Requests for access to data can be submitted via the website www.vivli.org. Questions can also be directed to datasharing@alnylam.com.