AmyloidosisPhase 3ATTRibute-CMNCT03860935
Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy
A Phase 3 study testing acoramidis in people with Amyloidosis and Amyloid Cardiomyopathy, run by Eidos Therapeutics, a BridgeBio company at 104 sites in 18 countries.
Trial summary
Phase 3 efficacy and safety study to evaluate acoramidis (AG10) HCl 800 mg administered orally twice a day compared to placebo in subjects with symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM). Read more about this study
- Who is it for?
- Ages 18 to 90 years
- Conditions the study is for
- Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis
- What is being tested?
- acoramidis
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 632 participants
- Where
- 104 sitesin 18 countries
- Study dates
- 19 Mar 2019to 11 May 2023
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 90 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Have an established diagnosis of ATTR-CM with either wild-type TTR or variant TTR genotype
- Have a history of heart failure evidenced by at least one prior hospitalization for heart failure or clinical evidence of heart failure without prior heart failure hospitalization manifested by signs or symptoms of volume overload or elevated intracardiac pressures or heart failure symptoms that required or require ongoing treatment with a diuretic.
- New York Heart Association (NYHA) Class I-III symptoms due to ATTR cardiomyopathy.
- On stable doses of cardiovascular medical therapy
- Completed ≥150 m on the 6MWT on 2 tests that are within 15% of total distance walked prior to randomization
- Biomarkers of myocardial wall stress, NT-proBNP level ≥300 pg/mL at screening
- Have left ventricular wall (interventricular septum or left ventricular posterior wall) thickness ≥12 mm
Who cannot join
- Had acute myocardial infarction, acute coronary syndrome or coronary revascularization, or experienced stroke or transient ischemic attack within 90 days prior to screening
- Has hemodynamic instability
- Likely to undergo heart transplantation within a year of screening
- Confirmed diagnosis of primary (light chain) amyloidosis
- Biomarkers of myocardial wall stress, NT-proBNP level ≥8500 pg/mL at screening
- Measure of kidney function, eGFR by MDRD formula <15 mL/min/1.73 m2
- Current treatment with marketed drug products and other investigational agents for the treatment of ATTR-CM
- Current treatment with calcium channel blockers with conduction system effects (e.g. verapamil, diltiazem). The use of dihydropyridine calcium channel blockers is allowed. The use of digitalis will only be allowed if required for management of atrial fibrillation with rapid ventricular response
Trial locations and contacts
104 sites in 18 countries.
104 sites in 18 countries
United States40 sites
Piedmont Heart Institute Athens
Athens, Georgia, 30606
Emory Heart and Vascular Center
Atlanta, Georgia, 30322
University of Colorado Hospital - Anschutz Medical Campus
Aurora, Colorado, 80045
MedStar Medical Group Cardiology at Franklin Square Medical Center
Baltimore, Maryland, 21218
Boston University School of Medicine
Boston, Massachusetts, 02118
Saint Elizabeth's Medical Center
Boston, Massachusetts, 02135
University of North Carolina - Chapel Hill
Chapel Hill, North Carolina, 27514
The Medical University of South Carolina
Charleston, South Carolina, 29425
Northwestern University
Chicago, Illinois, 60611
The University of Chicago Medical Center
Chicago, Illinois, 60637
Cleveland Clinic
Cleveland, Ohio, 44195
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
Duke University Health System
Durham, North Carolina, 27710
NorthShore University Health System
Evanston, Illinois, 60201
Prisma Health - Greenville Memorial Hospital
Greenville, South Carolina, 29605
Indiana University
Indianapolis, Indiana, 46202
Saint Luke's Hospital - Kansas City
Kansas City, Missouri, 64111
Cedars-Sinai Medical Center
Los Angeles, California, 90048
North Shore University Hospital
Manhasset, New York, 11030
University of Miami - Sylvester Comprehensive Cancer Center
Miami, Florida, 33136
Yale School of Medicine
New Haven, Connecticut, 06473
New York University Langone Health
New York, New York, 10010
Mount Sinai Hospital
New York, New York, 10029
Columbia University Medical Center
New York, New York, 10034
Newark Beth Israel Medical Center
Newark, New Jersey, 07112
Penn Presbyterian Medical Center
Philadelphia, Pennsylvania, 19104
Allegheny General Hospital
Pittsburgh, Pennsylvania, 15212
University of Pittsburgh
Pittsburgh, Pennsylvania, 15213
Oregon Health and Science University
Portland, Oregon, 97239
Virginia Commonwealth University Medical Center
Richmond, Virginia, 23298
Carilion Clinic Roanoke Heart Institute
Roanoke, Virginia, 24014
Mayo Clinic
Rochester, Minnesota, 55905
Laurelton Heart Specialist
Rosedale, New York, 11422
University of Utah
Salt Lake City, Utah, 84132
Pacific Heart Institute
Santa Monica, California, 90404
University of Washington School of Medicine
Seattle, Washington, 98195
Providence Sacred Heart Medical Center
Spokane, Washington, 99204
Washington University School of Medicine
St Louis, Missouri, 63110
Montefiore Medical Center
The Bronx, New York, 10461
MedStar Washington Hospital Center
Washington D.C., District of Columbia, 20010
Canada11 sites
University of Calgary
Calgary, Alberta, T2N 4Z6
Halifax Infirmary
Halifax, Nova Scotia, B3H 3A7
Centre Hospitalier de L'Universite de Montreal - Hôpital Notre-Dame
Montreal, Quebec, H2X 3H8
Hôpital du Sacré-Coeur de Montréal
Montreal, Quebec, H4J 1C5
Montreal Heart Institute
Montreal
Institut Universitaire de Cardiologie et de Pneumologie de Québec
Québec, Quebec, G1V 4G5
Hôpital régional de Rimouski
Rimouski, Quebec, G5L 5T1
Toronto Heart Centre
Toronto, Ontario, M4P 1E4
University of Toronto
Toronto, Ontario, M5G 2N2
University of British Columbia
Vancouver, British Columbia, V5Z 1M9
CancerCare Manitoba - St. Boniface
Winnipeg, Manitoba
Spain9 sites
Hospital Universitari Germans Trias i Pujol
Barcelona
Hospital Juan Ramón Jiménez
Huelva, 21005
Hospital Juan Ramón Jiménez
Huelva
Clinica Universidad de Navarra Madrid
Madrid
Hospital Universitario Puerta de Hierro
Madrid
Hospital Son Llàtzer
Palma de Mallorca
Clínica Universidad de Navarra
Pamplona, Navarre
Hospital Clínico Universitario de Santiago de Compostela
Santiago de Compostela
Hospital Clínico Universitario de Valencia
Valencia
Italy7 sites
Ospedale San Donato
Arezzo
Azienda Ospedaliero Universitaria di Bologna Policlinico Sant'Orsola-Malpighi
Bologna
Azienda Ospedaliero - Universitaria Careggi
Florence
Fondazione IRCCS Policlinico San Matteo
Pavia, 27100
Fondazione Toscana Gabriele Monasterio per la Ricerca Medica e di Sanità Pubblica
Pisa
Ospedale degli Infermi
Rimini
Universita degli Studi di Roma La Sapienza - Umberto I Policlinico di Roma
Roma
Australia6 sites
Royal Adelaide Hospital
Adelaide
Box Hill Hospital
Box Hill
Royal Hobart Hospital
Hobart
Fiona Stanley Hospital
Murdoch
Saint Vincent's Hospital Sydney
Sydney, 2010
Princess Alexandra Hospital
Woolloongabba
Belgium5 sites
Onze-Lieve-Vrouw Ziekenhuis Aalst
Aalst
Algemeen Ziekenhuis Sint-Jan Brugge-Oostende
Bruges, West Vlaanderen
Ziekenhuis Oost-Limburg - Campus Sint-Jan
Genk, Limburg
Jessa Ziekenhuis - Campus Virga Jesse
Hasselt, Limburg
Universitair Ziekenhuis Leuven
Leuven
Brazil5 sites
Santa Casa de Misericordia - Porto Alegre
Porto Alegre, Rio Grande do Sul, 90020-090
Instituto de Cardiologia do Rio Grande do Sul
Porto Alegre
CAPED - Centro Avançado de Pesquisa e Estudos para o Diagnóstico
Ribeirão Preto, São Paulo, 14026-900
Hospital Cárdio Pulmonar
Salvador, Estado de Bahia
INCOR
São Paulo, São Paulo
Czechia3 sites
St. Anne´s University Hospital
Brno-střed
General University Hospital in Prague
New Town
Institute for Clinical and Experimental Medicine
Prague
Netherlands3 sites
Universitair Medisch Centrum Groningen
Groningen
Maastricht Universitair Medisch Centrum
Maastricht, Limburg, 6229 HX
Universitair Medisch Centrum Utrecht
Utrecht
Ireland2 sites
Mater Misericordiae University Hospital
Dublin
Saint Vincents University Hospital
Dublin
Israel2 sites
Hadassah University Hospital Ein Kerem
Jerusalem
The Chaim Sheba Medical Center
Tel Litwinsky
New Zealand2 sites
Waikato Hospital
Hamilton, Waikato Region, 3240
Middlemore Hospital
Otahuhu, Auckland, 1640
Portugal2 sites
Centro Hospitalar de Lisboa Norte EPE- Hospital Santa Maria
Lisbon
Centro Hospitalar do Porto
Porto, 4099-001
South Korea2 sites
Seoul National University Bundang Hospital
Seongnam-si, Gyeonggi-do, 13620
Seoul National University Hospital
Seoul, Gyeonggi-do, 13620
United Kingdom2 sites
Royal Free Hospital
London, England, NW3 2PF
Richmond Pharmacology
London, SE1 1YR
About this study
Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy and Safety of AG10 in Subjects With Symptomatic Transthyretin Amyloid Cardiomyopathy (ATTRibute-CM Trial)
Phase 3 efficacy and safety study to evaluate acoramidis (AG10) HCl 800 mg administered orally twice a day compared to placebo in subjects with symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM).
Read the detailed description
Transthyretin amyloid cardiomyopathy (ATTR-CM) is an underdiagnosed condition believed to affect more than 400,000 people worldwide. In ATTR-CM, the accumulation of transthyretin (TTR) amyloid results in thickening and stiffening of the heart, which often leads to heart failure or even death.
There are two forms of ATTR-CM:
- Wild Type* This form of the condition primarily develops in older individuals who do not carry gene mutations.
- Hereditary* This form of the condition comes from gene mutations passed down in families.
In this study we are researching the investigational drug acoramidis HCl 800 mg administered orally twice a day. Through the study, we want to evaluate the efficacy and safety of acoramidis in patients with ATTR-CM versus placebo.
This is a 30 month, randomized, double-blind, placebo-controlled study. This means that, during the 30 month study, investigators conducting the research and study participants will not know whether the study participant is receiving acoramidis or placebo.
The primary outcomes of the study are:
- The impact of acoramidis versus placebo on the change in distance walked on the 6 minute walk test (6MWT) after 12 months of treatment compared to baseline.
- The impact of acoramidis versus placebo on the hierarchical combination of All-Cause mortality, cumulative frequency of cardiovascular-related hospitalizations, change from baseline in NT-proBNP, and change in from baseline in 6MWT over a 30-month fixed treatment duration.
At the end of 30 months, participants may be eligible to receive investigational acoramidis, and there is no placebo. This is called an "open label extension." This separate study may help us better understand the safety related to taking acoramidis over a longer period of time.
- Conditions
- Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies, Heart Diseases
- Being tested
- acoramidis, Placebo Oral Tablet
- Keywords
- Amyloidosis, ATTR-CM, Transthyretin, Amyloid, TTR
- Medical subject headings
- Amyloidosis, Amyloid Neuropathies, Familial, Amyloidosis, Hereditary, Transthyretin-Related, Cardiomyopathies, Heart Diseases, Alzheimer Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
acoramidisDrug
Also called AG10, ALXN2060
TTR stabilizer administered orally twice daily (BID)
Placebo Oral TabletDrug
Non-active control administered orally twice daily (BID)
| Study group | Role | What they receive |
|---|---|---|
| acoramidis HCl 800 mg | Experimental | Subjects will receive acoramidis HCl 800 mg twice daily. 6MWT primary outcome will be assessed at the end of 12 months. The hierarchical combination of All-Cause mortality, cumulative frequency of cardiovascular-related hospitalizations, change from baseline in NT-proBNP levels, and change from baseline in distance walked on the 6MWT will be assessed after 30 months of treatment. Receives: Drug: acoramidis |
| Placebo | Placebo comparator | Subjects will receive placebo to match twice daily. 6MWT primary outcome will be assessed at the end of 12 months. The hierarchical combination of All-Cause mortality, cumulative frequency of cardiovascular-related hospitalizations, change from baseline in NT-proBNP levels, and change from baseline in distance walked on the 6MWT will be assessed after 30 months of treatment. Receives: Drug: Placebo Oral Tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
A Hierarchical Combination of All-Cause Mortality, Cumulative Frequency of CV-related Hospitalization, Change From Baseline in NT-proBNP and Change From Baseline in 6MWT at the Last Available Visit Where Both Subjects Had Non-missing Assessments.
The endpoint was analyzed using Finkelstein-Schoenfeld method. The method combines all-cause mortality, cumulative frequency of CV-related hospitalizations, change from baseline in NT-proBNP and change from baseline in 6MWT in a hierarchical fashion. The method compares every participant with every other participant within strata, assigning a +1 to the "better" participant and a -1 to the "worse" participant and 0 if they are "tied". Participants who had heart transplantation or implantation of a cardiac mechanical assist device were handled in the same manner as death. 'Win' represents a participant doing better based on hierarchical comparison. The reported unit is the total percent of "wins" for each treatment group from performing such a hierarchical comparison across stratification factors in the study.
Measured: Baseline up to Month 30
Secondary outcomes (4)
Change From Baseline to Month 30 in the Distance Walked During the 6 Minute Walk Test (6MWT)
6MWT measures the total distance that a participant could walk in 6 minutes. Participants were asked to perform the test at a pace that was comfortable to them, with as many breaks as they needed.
Measured: Month 30
Change From Baseline to Month 30 of the Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS)
KCCQ is a 23-item participant-completed questionnaire that assesses health status and health-related quality of life in participants with heart failure. Eight domain scores were calculated for the KCCQ: Physical limitation, Social limitation, Quality of life, Self-efficacy, Symptom stability, Symptom frequency, Symptom burden, and Total symptoms (calculated as the mean of Symptom frequency and Symptom burden scores). The summary score of Overall Summary (calculated as mean of Physical limitation, Social limitation, Total symptoms, and Quality of life scores) was calculated. Domain and summary scores were scaled to range from 0 (minimum) to 100 (maximum); higher scores represent better health status.
Measured: Month 30
Change From Baseline to Month 30 in Serum TTR (Prealbumin) Level
Serum TTR (Prealbumin) is an in vivo biomarker of stabilization.
Measured: Month 30
All-cause Mortality by Month 30, Including Death Due to Any Cause, Heart Transplant or Cardiac Mechanical Assist Device (CMAD)
Number of deaths due to any cause was analyzed. Participants who had heart transplantation or implantation of a CMAD were handled in the same manner as death.
Measured: Baseline up to Month 30
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Mar 2019
- Study start19 Mar 2019
- Primary completion11 May 2023
- Study completion11 May 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (16)Papers the sponsor has linked to this study.
- Other trials for AmyloidosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 632 participants (actual)
Sponsor and investigators
- Lead sponsor
- Eidos Therapeutics, a BridgeBio company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03860935
- Sponsor's protocol ID
- AG10-301
- Other IDs
- 2018-004280-32, 2018
- Acronym
- ATTRibute-CM
- Status last verified
- Jun 2024
- First submitted
- 27 Feb 2019
- Last updated
- 27 Jun 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Ilonze OJ, Judge DP, Masri A, Soman P, Cappelli F, Khouri MG, Sarswat N, Hanna M, Liu Y, Murtagh G, Xiong K, Castano A, Tamby JF, Fox JC, Sinha U, Griffin JM, Gillmore JD, Januzzi JL Jr. Natriuretic Peptide and Transthyretin and Their Relationship With Clinical Outcomes: Results From ATTRIBUTE-CM, a Randomized Controlled Trial. J Am Heart Assoc. 2026 Aug 30:e050566. doi: 10.1161/JAHA.126.050566. Online ahead of print.
PubMed 42669902Pfister R, Michel L, Schulze M, Vogtlander K, Ciaccia A, Merz M, Hengstenberg C. ACO-SWITCH: Rationale and Design of a Phase 4 Study Evaluating Serum Transthyretin in ATTR-CM After Switching from Tafamidis to Acoramidis. Cardiol Ther. 2026 Aug 20. doi: 10.1007/s40119-026-00466-4. Online ahead of print.
PubMed 42622768Judge DP, Alexander KM, Cappelli F, Fontana M, Garcia-Pavia P, Grogan M, Hanna M, Masri A, Obici L, Soman P, Cao X, Lystig T, Tamby JF, Siddhanti S, Castano A, Katz L, Fox JC, Mahaffey KW, Gillmore JD. Effect of acoramidis on risk of death and heart problem-related hospitalizations in people with transthyretin amyloid cardiomyopathy: a plain language summary. Future Cardiol. 2026 Aug 5:1-10. doi: 10.1080/14796678.2026.2702094. Online ahead of print.
PubMed 42554365Sherrod CF 4th, Fontana M, Gillmore JD, Kastritis E, Mitchell JD, Krejci J, Tauras JM, van der Meer P, Chen C, Falvey H, Siddhanti S, Tamby JF, Fox JC, Cappelli F, Masri A, Garcia-Pavia P, Hanna M, Sperry BW. Effect of Acoramidis on Heart Failure-Related Health Status: A Secondary Analysis of the ATTRibute-CM Randomized Clinical Trial. JAMA Cardiol. 2026 Jul 29:e262413. doi: 10.1001/jamacardio.2026.2413. Online ahead of print.
PubMed 42525404Testani JM, Judge DP, Borlaug BA, Cherney D, Cox ZL, Gillmore JD, Lewis JB, Adler SH, Cao X, Castano A, Fox JC, Katz L, Mathur V, Xiong K, Butler J, Masri A. Effects of Acoramidis on Kidney Function in Transthyretin Amyloid Cardiomyopathy. Circ Heart Fail. 2026 Jul 2:e014656. doi: 10.1161/CIRCHEARTFAILURE.126.014656. Online ahead of print.
PubMed 42389794Judge DP, Ji AX, Graef IA, Grogan M, Soman P, Cappelli F, Fontana M, Masri A, Gillmore JD, Garcia-Pavia P, Cao X, Fox JC, Sinha U. Differential Transthyretin Stabilization in Patients With Wild Type and Variant Transthyretin Amyloidosis. JACC CardioOncol. 2026 Jun;8(3):299-313. doi: 10.1016/j.jaccao.2026.04.006.
PubMed 42307157Fontana M, Cappelli F, Garcia-Pavia P, Judge DP, Obici L, Masri A, Agostinho JR, Tschope C, Vogtlander K, Ciaccia A, Zazula A, Zeitler S, Tamby JF, Fox JC, Gillmore JD, Damy T. Outpatient worsening heart failure in transthyretin amyloid cardiomyopathy: Findings from ATTRibute-CM. Eur J Heart Fail. 2026 May 29:xuag177. doi: 10.1093/ejhf/xuag177. Online ahead of print.
PubMed 42216490Tahara N, Raizner A, Chen Y, Mercuri MF, Minamisawa M; Phase 3 Japan Study Investigators. Efficacy of Acoramidis in Adults With Transthyretin Amyloid Cardiomyopathy: Propensity Score Comparison Between a Single-Arm Open-Label Phase 3 Trial in Japan and a Global Double-Blind Phase 3 Trial. Circ Rep. 2026 Apr 23;8(5):797-806. doi: 10.1253/circrep.CR-25-0304. eCollection 2026 May 8.
PubMed 42110872Gillmore JD, Judge DP, Cappelli F, Fontana M, Garcia-Pavia P, Gibbs S, Grogan M, Hanna M, Hoffman J, Masri A, Maurer MS, Nativi-Nicolau J, Obici L, Poulsen SH, Rockhold F, Shah KB, Soman P, Garg J, Chiswell K, Xu H, Cao X, Lystig T, Sinha U, Fox JC; ATTRibute-CM Investigators. A plain language review of the ATTRibute-CM study: efficacy and safety of acoramidis in transthyretin amyloid cardiomyopathy. Future Cardiol. 2025 Dec;21(12):1017-1025. doi: 10.1080/14796678.2025.2591426. Epub 2025 Dec 12.
PubMed 41384357Alexander KM, Davis MK, Akinboboye O, Berk J, Bhatt K, Cappelli F, Cuddy SAM, Fontana M, Garcia-Pavia P, Gillmore JD, Griffin JM, Grodin JL, Judge DP, Khouri MG, Lam K, Masri A, Maurer MS, Obici L, Ruberg FL, Sarswat N, Shah K, Soman P, Stern L, Wright R, Xiong K, Cao X, Lystig T, Tamby JF, Castano A, Katz L, Sinha U, Fox JC, Solomon SD, Grogan M. Efficacy of Acoramidis in Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy: Results From ATTRibute-CM and Its Open-Label Extension. JAMA Cardiol. 2026 Jan 1;11(1):57-67. doi: 10.1001/jamacardio.2025.4477.
PubMed 41205147Dasgupta N, Poulsen SH, Emdin M, Ambardekar AV, Shah KB, Hennum L, Marwah R, Allison M, Shivanna P, Siddhanti S, Tamby JF, Falvey H, Grodin JL. Contemporary Oral Medication Use and Frequency in Patients with Transthyretin Amyloid Cardiomyopathy. Am J Cardiovasc Drugs. 2025 Nov;25(6):829-839. doi: 10.1007/s40256-025-00752-x. Epub 2025 Aug 1.
PubMed 40750720Maurer MS, Judge DP, Gillmore JD, Garcia-Pavia P, Masri A, Cappelli F, Alexander KM, Sarswat N, Grogan M, Ambardekar AV, Ducharme A, Poulsen SH, Lam K, Obici L, Soman P, Rao S, Tamby JF, Castano A, Fox JC, Adam B, Chepyala SR, Poland B, Sinha U, Fontana M. Early Increase in Serum Transthyretin by Acoramidis Independently Predicts Improved Survival in TTR Amyloid Cardiomyopathy. J Am Coll Cardiol. 2025 May 27;85(20):1911-1923. doi: 10.1016/j.jacc.2025.03.542.
PubMed 40398971Judge DP, Alexander KM, Cappelli F, Fontana M, Garcia-Pavia P, Gibbs SDJ, Grogan M, Hanna M, Masri A, Maurer MS, Obici L, Soman P, Cao X, Lystig T, Tamby JF, Siddhanti S, Castano A, Katz L, Fox JC, Mahaffey KW, Gillmore JD. Efficacy of Acoramidis on All-Cause Mortality and Cardiovascular Hospitalization in Transthyretin Amyloid Cardiomyopathy. J Am Coll Cardiol. 2025 Mar 18;85(10):1003-1014. doi: 10.1016/j.jacc.2024.11.042.
PubMed 40074465Soman P, Khouri MG, Lenihan D, Reyentovich A, Sperry BW, Sowalsky K, Bai Y, Du J, Katz L, Siddhanti S, Fox JC. Comparison of in-clinic assessment of 6MWT by conventional method and using wearable sensors for patients with ATTR-CM. Future Cardiol. 2025 Feb;21(2):75-81. doi: 10.1080/14796678.2025.2457881. Epub 2025 Jan 29.
PubMed 39878480Gillmore JD, Judge DP, Cappelli F, Fontana M, Garcia-Pavia P, Gibbs S, Grogan M, Hanna M, Hoffman J, Masri A, Maurer MS, Nativi-Nicolau J, Obici L, Poulsen SH, Rockhold F, Shah KB, Soman P, Garg J, Chiswell K, Xu H, Cao X, Lystig T, Sinha U, Fox JC; ATTRibute-CM Investigators. Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy. N Engl J Med. 2024 Jan 11;390(2):132-142. doi: 10.1056/NEJMoa2305434.
PubMed 38197816