APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

NCT ID: NCT03997383

Last Updated: 2026-01-12

Study Results

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Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

360 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-09-12

Study Completion Date

2027-03-15

Brief Summary

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The purpose of this study is to evaluate the efficacy and safety of patisiran in participants with ATTR amyloidosis with cardiomyopathy.

Detailed Description

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Conditions

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Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Patisiran

Participants will be administered multiple doses of patisiran in the double-blind and open-label extension period.

Group Type EXPERIMENTAL

Patisiran

Intervention Type DRUG

Patisiran will be administered by intravenous (IV) infusion.

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.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Normal saline (0.9% NaCl) matching volume of patisiran doses will be administered intravenously.

Patisiran

Intervention Type DRUG

Patisiran will be administered by intravenous (IV) infusion.

Interventions

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Placebo

Normal saline (0.9% NaCl) matching volume of patisiran doses will be administered intravenously.

Intervention Type DRUG

Patisiran

Patisiran will be administered by intravenous (IV) infusion.

Intervention Type DRUG

Other Intervention Names

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ALN-TTR02 patisiran-lipid nanoparticle (LNP)

Eligibility Criteria

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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

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
Minimum Eligible Age

18 Years

Maximum Eligible Age

85 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alnylam Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Alnylam Pharmaceuticals

Locations

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Clinical Trial Site

Los Angeles, California, United States

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Chicago, Illinois, United States

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Skokie, Illinois, United States

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Iowa City, Iowa, United States

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Kansas City, Kansas, United States

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Baltimore, Maryland, United States

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Boston, Massachusetts, United States

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Burlington, Massachusetts, United States

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Rochester, Minnesota, United States

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St Louis, Missouri, United States

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New York, New York, United States

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New York, New York, United States

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Cleveland, Ohio, United States

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Philadelphia, Pennsylvania, United States

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Philadelphia, Pennsylvania, United States

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Pittsburgh, Pennsylvania, United States

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Nashville, Tennessee, United States

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Dallas, Texas, United States

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Norfolk, Virginia, United States

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Buenos Aires, Buenos Aires, Argentina

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San Juan Bautista, Buenos Aires, Argentina

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Córdoba, Córdoba Province, Argentina

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Rosario, Santa Fe Province, Argentina

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Rosario, Santa Fe Province, Argentina

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Rosario, Santa Fe Province, Argentina

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Santa Fe, Santa Fe Province, Argentina

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Buenos Aires, , Argentina

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Westmead, New South Wales, Australia

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Woolloongabba, Queensland, Australia

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Box Hill, Victoria, Australia

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Liège, LIE, Belgium

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Hasselt, LIM, Belgium

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Aalst, OVL, Belgium

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Roeselare, WVL, Belgium

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Porto Alegre, Rio Grande do Sul, Brazil

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Ribeirão Preto, São Paulo, Brazil

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Ribeirão Preto, São Paulo, Brazil

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São Paulo, São Paulo, Brazil

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Rio de Janeiro, , Brazil

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São Paulo, , Brazil

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Sofia, Sofia-Grad, Bulgaria

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Sofia, , Bulgaria

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Stara Zagora, , Bulgaria

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Santiago, , Chile

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Prague, Praha, Hlavní Mesto, Czechia

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Brno, , Czechia

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Prague, , Czechia

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Prague, , Czechia

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Aarhus N, Aarhus, Denmark

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Copenhagen, , Denmark

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Odense C, , Denmark

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Clichy, Paris, France

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Créteil, Val-de-Marne, France

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Rennes, , France

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Toulouse, , France

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Lai Chi Kok, , Hong Kong

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Bologna, Emilia-Romagna, Italy

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Messina, Sicily, Italy

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Florence, , Italy

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Pavia, , Italy

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Nagoya, Aichi-ken, Japan

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Kurume, Fukuoka, Japan

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Matsumoto, Nagano, Japan

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Suita, Osaka, Japan

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Bunkyo-ku, Tokyo, Japan

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Fukuoka, , Japan

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Kumamoto, , Japan

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Mexico City, , Mexico

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Groningen, , Netherlands

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Maastricht, , Netherlands

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Christchurch, , New Zealand

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Hamilton, , New Zealand

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Lódz, Lódzkie, Poland

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Warsaw, Masovian Voivodeship, Poland

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Warsaw, Masovian Voivodeship, Poland

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Gdansk, Pomeranian Voivodeship, Poland

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Katowice, Silesian Voivodeship, Poland

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Porto, , Portugal

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Senhora da Hora, , Portugal

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Viseu, , Portugal

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Seoul, , South Korea

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Stockholm, , Sweden

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Taipei, , Taiwan

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Bellshill, Lanarkshire, United Kingdom

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London, London, City of, United Kingdom

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Hexham, Northumberland, United Kingdom

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Birmingham, West Midlands, United Kingdom

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Cardiff, , United Kingdom

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London, , United Kingdom

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Manchester, , United Kingdom

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Countries

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United States Argentina Australia Belgium Brazil Bulgaria Chile Czechia Denmark France Hong Kong Italy Japan Mexico Netherlands New Zealand Poland Portugal South Korea Sweden Taiwan United Kingdom

References

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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.

Reference Type DERIVED
PMID: 40243852 (View on PubMed)

Maurer 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.

Reference Type DERIVED
PMID: 37888916 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2019-001458-24

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

ALN-TTR02-011

Identifier Type: -

Identifier Source: org_study_id

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